{"id":3766,"date":"2025-11-24T12:54:19","date_gmt":"2025-11-24T11:54:19","guid":{"rendered":"https:\/\/www.technoscience.it\/?page_id=3766"},"modified":"2025-11-24T13:07:40","modified_gmt":"2025-11-24T12:07:40","slug":"studi-su-malattie-rare-modelli-endpoint-e-sostenibilita-clinica","status":"publish","type":"page","link":"https:\/\/www.technoscience.it\/en\/servizi\/biotecnologie-e-innovazione\/biopharma-terapie\/studi-su-malattie-rare-modelli-endpoint-e-sostenibilita-clinica\/","title":{"rendered":"Rare-Disease Studies: Models, Endpoints and Clinical Sustainability"},"content":{"rendered":"\t\t<div data-elementor-type=\"wp-page\" data-elementor-id=\"3766\" class=\"elementor elementor-3766\" data-elementor-post-type=\"page\">\n\t\t\t\t\t\t<section class=\"elementor-section elementor-top-section elementor-element elementor-element-3f8e8db elementor-section-boxed elementor-section-height-default elementor-section-height-default\" data-id=\"3f8e8db\" data-element_type=\"section\" data-e-type=\"section\" data-settings=\"{&quot;background_background&quot;:&quot;classic&quot;}\">\n\t\t\t\t\t\t<div class=\"elementor-container elementor-column-gap-extended\">\n\t\t\t\t\t<div class=\"elementor-column elementor-col-50 elementor-top-column elementor-element elementor-element-a07f2dc\" data-id=\"a07f2dc\" data-element_type=\"column\" data-e-type=\"column\">\n\t\t\t<div class=\"elementor-widget-wrap elementor-element-populated\">\n\t\t\t\t\t\t<div class=\"elementor-element elementor-element-c9662aa elementor-widget elementor-widget-theme-post-featured-image elementor-widget-image\" data-id=\"c9662aa\" data-element_type=\"widget\" data-e-type=\"widget\" data-widget_type=\"theme-post-featured-image.default\">\n\t\t\t\t<div class=\"elementor-widget-container\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<img fetchpriority=\"high\" decoding=\"async\" width=\"800\" height=\"800\" src=\"https:\/\/www.technoscience.it\/wp-content\/uploads\/2025\/11\/studi-clinici-su-malattie-rare-1024x1024.jpg\" class=\"attachment-large size-large wp-image-3780\" alt=\"Multidisciplinary team designing rare-disease clinical studies, reviewing study models, endpoints, patient registries and real-world data\" srcset=\"https:\/\/www.technoscience.it\/wp-content\/uploads\/2025\/11\/studi-clinici-su-malattie-rare-1024x1024.jpg 1024w, https:\/\/www.technoscience.it\/wp-content\/uploads\/2025\/11\/studi-clinici-su-malattie-rare-300x300.jpg 300w, https:\/\/www.technoscience.it\/wp-content\/uploads\/2025\/11\/studi-clinici-su-malattie-rare-150x150.jpg 150w, https:\/\/www.technoscience.it\/wp-content\/uploads\/2025\/11\/studi-clinici-su-malattie-rare-768x768.jpg 768w, https:\/\/www.technoscience.it\/wp-content\/uploads\/2025\/11\/studi-clinici-su-malattie-rare-1536x1536.jpg 1536w, https:\/\/www.technoscience.it\/wp-content\/uploads\/2025\/11\/studi-clinici-su-malattie-rare-12x12.jpg 12w, https:\/\/www.technoscience.it\/wp-content\/uploads\/2025\/11\/studi-clinici-su-malattie-rare.jpg 1920w\" sizes=\"(max-width: 800px) 100vw, 800px\" title=\"Rare-Disease Clinical Studies \u2013 Models, Endpoints and Clinical Sustainability\" \/>\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t\t<\/div>\n\t\t<\/div>\n\t\t\t\t<div class=\"elementor-column elementor-col-50 elementor-top-column elementor-element elementor-element-ffd87b2\" data-id=\"ffd87b2\" data-element_type=\"column\" data-e-type=\"column\">\n\t\t\t<div class=\"elementor-widget-wrap elementor-element-populated\">\n\t\t\t\t\t\t<div class=\"elementor-element elementor-element-5d5f4e1 elementor-widget elementor-widget-heading\" data-id=\"5d5f4e1\" data-element_type=\"widget\" data-e-type=\"widget\" data-widget_type=\"heading.default\">\n\t\t\t\t<div class=\"elementor-widget-container\">\n\t\t\t\t\t<h1 class=\"elementor-heading-title elementor-size-default\">Studi su malattie rare: modelli, endpoint e sostenibilit\u00e0 clinica<\/h1>\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t<div class=\"elementor-element elementor-element-9ab876a elementor-widget-divider--view-line elementor-widget elementor-widget-divider\" data-id=\"9ab876a\" data-element_type=\"widget\" data-e-type=\"widget\" data-widget_type=\"divider.default\">\n\t\t\t\t<div class=\"elementor-widget-container\">\n\t\t\t\t\t\t\t<div class=\"elementor-divider\">\n\t\t\t<span class=\"elementor-divider-separator\">\n\t\t\t\t\t\t<\/span>\n\t\t<\/div>\n\t\t\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t<div class=\"elementor-element elementor-element-06b7306 elementor-widget elementor-widget-text-editor\" data-id=\"06b7306\" data-element_type=\"widget\" data-e-type=\"widget\" data-widget_type=\"text-editor.default\">\n\t\t\t\t<div class=\"elementor-widget-container\">\n\t\t\t\t\t\t\t\t\t<p>Le malattie rare obbligano a ripensare il modo in cui si fa evidenza. I numeri sono piccoli, l\u2019eterogeneit\u00e0 \u00e8 alta, la storia naturale \u00e8 spesso incompleta. Eppure proprio qui si misura la seriet\u00e0 di un programma <strong>BioPharma<\/strong>: nella capacit\u00e0 di costruire <strong>modelli di studio<\/strong> credibili, scegliere <strong>endpoint<\/strong> che dicano qualcosa di vero per pazienti e regolatori, e dimostrare <strong>sostenibilit\u00e0 clinica<\/strong> oltre l\u2019annuncio iniziale. In Technoscience affrontiamo le rare con una regola semplice: \u201cmeno pazienti\u201d non significa \u201cmeno metodo\u201d. Significa metodo diverso, pi\u00f9 attento, pi\u00f9 trasparente.<\/p>\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t\t<\/div>\n\t\t<\/div>\n\t\t\t\t\t<\/div>\n\t\t<\/section>\n\t\t\t\t<section class=\"elementor-section elementor-top-section elementor-element elementor-element-b9d5dbe elementor-section-boxed elementor-section-height-default elementor-section-height-default\" data-id=\"b9d5dbe\" data-element_type=\"section\" data-e-type=\"section\" data-settings=\"{&quot;background_background&quot;:&quot;classic&quot;}\">\n\t\t\t\t\t\t<div class=\"elementor-container elementor-column-gap-extended\">\n\t\t\t\t\t<div class=\"elementor-column elementor-col-50 elementor-top-column elementor-element elementor-element-bf15449\" data-id=\"bf15449\" data-element_type=\"column\" data-e-type=\"column\">\n\t\t\t<div class=\"elementor-widget-wrap elementor-element-populated\">\n\t\t\t\t\t\t<div class=\"elementor-element elementor-element-7787d0e elementor-position-inline-start elementor-tablet-position-inline-start elementor-view-default elementor-mobile-position-block-start elementor-widget elementor-widget-icon-box\" data-id=\"7787d0e\" data-element_type=\"widget\" data-e-type=\"widget\" data-widget_type=\"icon-box.default\">\n\t\t\t\t<div class=\"elementor-widget-container\">\n\t\t\t\t\t\t\t<div class=\"elementor-icon-box-wrapper\">\n\n\t\t\t\t\t\t<div class=\"elementor-icon-box-icon\">\n\t\t\t\t<span  class=\"elementor-icon\">\n\t\t\t\t<svg aria-hidden=\"true\" class=\"e-font-icon-svg e-fas-fingerprint\" viewBox=\"0 0 512 512\" xmlns=\"http:\/\/www.w3.org\/2000\/svg\"><path d=\"M256.12 245.96c-13.25 0-24 10.74-24 24 1.14 72.25-8.14 141.9-27.7 211.55-2.73 9.72 2.15 30.49 23.12 30.49 10.48 0 20.11-6.92 23.09-17.52 13.53-47.91 31.04-125.41 29.48-224.52.01-13.25-10.73-24-23.99-24zm-.86-81.73C194 164.16 151.25 211.3 152.1 265.32c.75 47.94-3.75 95.91-13.37 142.55-2.69 12.98 5.67 25.69 18.64 28.36 13.05 2.67 25.67-5.66 28.36-18.64 10.34-50.09 15.17-101.58 14.37-153.02-.41-25.95 19.92-52.49 54.45-52.34 31.31.47 57.15 25.34 57.62 55.47.77 48.05-2.81 96.33-10.61 143.55-2.17 13.06 6.69 25.42 19.76 27.58 19.97 3.33 26.81-15.1 27.58-19.77 8.28-50.03 12.06-101.21 11.27-152.11-.88-55.8-47.94-101.88-104.91-102.72zm-110.69-19.78c-10.3-8.34-25.37-6.8-33.76 3.48-25.62 31.5-39.39 71.28-38.75 112 .59 37.58-2.47 75.27-9.11 112.05-2.34 13.05 6.31 25.53 19.36 27.89 20.11 3.5 27.07-14.81 27.89-19.36 7.19-39.84 10.5-80.66 9.86-121.33-.47-29.88 9.2-57.88 28-80.97 8.35-10.28 6.79-25.39-3.49-33.76zm109.47-62.33c-15.41-.41-30.87 1.44-45.78 4.97-12.89 3.06-20.87 15.98-17.83 28.89 3.06 12.89 16 20.83 28.89 17.83 11.05-2.61 22.47-3.77 34-3.69 75.43 1.13 137.73 61.5 138.88 134.58.59 37.88-1.28 76.11-5.58 113.63-1.5 13.17 7.95 25.08 21.11 26.58 16.72 1.95 25.51-11.88 26.58-21.11a929.06 929.06 0 0 0 5.89-119.85c-1.56-98.75-85.07-180.33-186.16-181.83zm252.07 121.45c-2.86-12.92-15.51-21.2-28.61-18.27-12.94 2.86-21.12 15.66-18.26 28.61 4.71 21.41 4.91 37.41 4.7 61.6-.11 13.27 10.55 24.09 23.8 24.2h.2c13.17 0 23.89-10.61 24-23.8.18-22.18.4-44.11-5.83-72.34zm-40.12-90.72C417.29 43.46 337.6 1.29 252.81.02 183.02-.82 118.47 24.91 70.46 72.94 24.09 119.37-.9 181.04.14 246.65l-.12 21.47c-.39 13.25 10.03 24.31 23.28 24.69.23.02.48.02.72.02 12.92 0 23.59-10.3 23.97-23.3l.16-23.64c-.83-52.5 19.16-101.86 56.28-139 38.76-38.8 91.34-59.67 147.68-58.86 69.45 1.03 134.73 35.56 174.62 92.39 7.61 10.86 22.56 13.45 33.42 5.86 10.84-7.62 13.46-22.59 5.84-33.43z\"><\/path><\/svg>\t\t\t\t<\/span>\n\t\t\t<\/div>\n\t\t\t\n\t\t\t\t\t\t<div class=\"elementor-icon-box-content\">\n\n\t\t\t\t\t\t\t\t\t<h3 class=\"elementor-icon-box-title\">\n\t\t\t\t\t\t<span  >\n\t\t\t\t\t\t\tPrima del trial: storia naturale e registri che valgono\t\t\t\t\t\t<\/span>\n\t\t\t\t\t<\/h3>\n\t\t\t\t\n\t\t\t\t\n\t\t\t<\/div>\n\t\t\t\n\t\t<\/div>\n\t\t\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t<div class=\"elementor-element elementor-element-a418f6d elementor-widget-divider--view-line elementor-widget elementor-widget-divider\" data-id=\"a418f6d\" data-element_type=\"widget\" data-e-type=\"widget\" data-widget_type=\"divider.default\">\n\t\t\t\t<div class=\"elementor-widget-container\">\n\t\t\t\t\t\t\t<div class=\"elementor-divider\">\n\t\t\t<span class=\"elementor-divider-separator\">\n\t\t\t\t\t\t<\/span>\n\t\t<\/div>\n\t\t\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t<div class=\"elementor-element elementor-element-edabc6c elementor-widget elementor-widget-text-editor\" data-id=\"edabc6c\" data-element_type=\"widget\" data-e-type=\"widget\" data-widget_type=\"text-editor.default\">\n\t\t\t\t<div class=\"elementor-widget-container\">\n\t\t\t\t\t\t\t\t\t<p>Nelle rare, il tempo speso a <strong>capire la storia naturale<\/strong> non \u00e8 tempo perso: \u00e8 il fondamento di qualsiasi valutazione di efficacia. Un registro ben costruito non \u00e8 un elenco di nomi; \u00e8 una struttura che definisce <strong>criteri diagnostici<\/strong>, <strong>fenotipi<\/strong>, <strong>tempi di progressione<\/strong>, <strong>eventi chiave<\/strong> e <strong>misure ripetibili<\/strong>. Senza questa mappa, l\u2019effetto di una terapia rischia di confondersi con l\u2019oscillazione naturale della malattia. Per questo integriamo registri clinici, biobanche, dati di telemedicina e, dove pertinente, misure digitali continue: \u00e8 l\u2019unico modo per dare contesto a numeri che, da soli, non possono essere \u201cgrandi\u201d.<\/p>\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t<div class=\"elementor-element elementor-element-8dc073d elementor-position-inline-start elementor-tablet-position-inline-start elementor-view-default elementor-mobile-position-block-start elementor-widget elementor-widget-icon-box\" data-id=\"8dc073d\" data-element_type=\"widget\" data-e-type=\"widget\" data-widget_type=\"icon-box.default\">\n\t\t\t\t<div class=\"elementor-widget-container\">\n\t\t\t\t\t\t\t<div class=\"elementor-icon-box-wrapper\">\n\n\t\t\t\t\t\t<div class=\"elementor-icon-box-icon\">\n\t\t\t\t<span  class=\"elementor-icon\">\n\t\t\t\t<svg aria-hidden=\"true\" class=\"e-font-icon-svg e-fas-filter\" viewBox=\"0 0 512 512\" xmlns=\"http:\/\/www.w3.org\/2000\/svg\"><path d=\"M487.976 0H24.028C2.71 0-8.047 25.866 7.058 40.971L192 225.941V432c0 7.831 3.821 15.17 10.237 19.662l80 55.98C298.02 518.69 320 507.493 320 487.98V225.941l184.947-184.97C520.021 25.896 509.338 0 487.976 0z\"><\/path><\/svg>\t\t\t\t<\/span>\n\t\t\t<\/div>\n\t\t\t\n\t\t\t\t\t\t<div class=\"elementor-icon-box-content\">\n\n\t\t\t\t\t\t\t\t\t<h3 class=\"elementor-icon-box-title\">\n\t\t\t\t\t\t<span  >\n\t\t\t\t\t\t\tModelli di studio: quando i classici non bastano\t\t\t\t\t\t<\/span>\n\t\t\t\t\t<\/h3>\n\t\t\t\t\n\t\t\t\t\n\t\t\t<\/div>\n\t\t\t\n\t\t<\/div>\n\t\t\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t<div class=\"elementor-element elementor-element-3a3d6fb elementor-widget-divider--view-line elementor-widget elementor-widget-divider\" data-id=\"3a3d6fb\" data-element_type=\"widget\" data-e-type=\"widget\" data-widget_type=\"divider.default\">\n\t\t\t\t<div class=\"elementor-widget-container\">\n\t\t\t\t\t\t\t<div class=\"elementor-divider\">\n\t\t\t<span class=\"elementor-divider-separator\">\n\t\t\t\t\t\t<\/span>\n\t\t<\/div>\n\t\t\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t<div class=\"elementor-element elementor-element-bffc321 elementor-widget elementor-widget-text-editor\" data-id=\"bffc321\" data-element_type=\"widget\" data-e-type=\"widget\" data-widget_type=\"text-editor.default\">\n\t\t\t\t<div class=\"elementor-widget-container\">\n\t\t\t\t\t\t\t\t\t<p>La randomizzazione resta l\u2019oro della prova, ma nelle rare non sempre \u00e8 praticabile o etica. Da qui l\u2019uso accorto di <strong>disegni adattativi<\/strong> (con regole pre-specificate), <strong>master protocol<\/strong> e <strong>platform trial<\/strong> quando la biologia collega pi\u00f9 sottotipi; in alternativa, <strong>controlli esterni<\/strong> derivati da registri di qualit\u00e0, con matching trasparente e limiti dichiarati. In alcune condizioni ha senso ricorrere a <strong>N-of-1<\/strong> o <strong>crossover<\/strong> per misurare l\u2019effetto intra-paziente. Dove i segnali sono complessi e le serie piccole, strumenti <strong>bayesiani<\/strong> aiutano a integrare conoscenza pregressa e dato corrente, ma solo se le assunzioni sono esplicitate e condivise col regolatore. La parola chiave \u00e8 una: <strong>pre-specificazione<\/strong>. Qualsiasi flessibilit\u00e0 non dichiarata prima diventa fragilit\u00e0 dopo.<\/p>\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t\t<\/div>\n\t\t<\/div>\n\t\t\t\t<div class=\"elementor-column elementor-col-50 elementor-top-column elementor-element elementor-element-99f9daa\" data-id=\"99f9daa\" data-element_type=\"column\" data-e-type=\"column\">\n\t\t\t<div class=\"elementor-widget-wrap elementor-element-populated\">\n\t\t\t\t\t\t<div class=\"elementor-element elementor-element-a7fcec9 elementor-widget elementor-widget-wp-widget-tag_cloud\" data-id=\"a7fcec9\" data-element_type=\"widget\" data-e-type=\"widget\" data-widget_type=\"wp-widget-tag_cloud.default\">\n\t\t\t\t<div class=\"elementor-widget-container\">\n\t\t\t\t\t<h5>Esplora i temi<\/h5><nav aria-label=\"Esplora i temi\"><div class=\"tagcloud\"><a href=\"https:\/\/www.technoscience.it\/en\/news\/tag\/cardiologia\/\" class=\"tag-cloud-link tag-link-191 tag-link-position-1\" style=\"font-size: 15.225806451613pt;\" aria-label=\"Cardiologia (5 items)\">Cardiologia<\/a>\n<a href=\"https:\/\/www.technoscience.it\/en\/news\/tag\/dieta-mediterranea\/\" class=\"tag-cloud-link tag-link-137 tag-link-position-2\" style=\"font-size: 10.709677419355pt;\" aria-label=\"dieta mediterranea (2 items)\">dieta mediterranea<\/a>\n<a href=\"https:\/\/www.technoscience.it\/en\/news\/tag\/digital-health\/\" class=\"tag-cloud-link tag-link-188 tag-link-position-3\" style=\"font-size: 16.279569892473pt;\" aria-label=\"Digital Health (6 items)\">Digital Health<\/a>\n<a href=\"https:\/\/www.technoscience.it\/en\/news\/tag\/ecosistema-dellinnovazione\/\" class=\"tag-cloud-link tag-link-185 tag-link-position-4\" style=\"font-size: 21.548387096774pt;\" aria-label=\"Ecosistema dell\u2019innovazione (15 items)\">Ecosistema dell\u2019innovazione<\/a>\n<a href=\"https:\/\/www.technoscience.it\/en\/news\/tag\/flebologia\/\" class=\"tag-cloud-link tag-link-181 tag-link-position-5\" style=\"font-size: 12.516129032258pt;\" aria-label=\"Flebologia (3 items)\">Flebologia<\/a>\n<a href=\"https:\/\/www.technoscience.it\/en\/news\/tag\/formazione-avanzata\/\" class=\"tag-cloud-link tag-link-182 tag-link-position-6\" style=\"font-size: 17.784946236559pt;\" aria-label=\"Formazione avanzata (8 items)\">Formazione avanzata<\/a>\n<a href=\"https:\/\/www.technoscience.it\/en\/news\/tag\/iba\/\" class=\"tag-cloud-link tag-link-198 tag-link-position-7\" style=\"font-size: 8pt;\" aria-label=\"IBA (1 item)\">IBA<\/a>\n<a href=\"https:\/\/www.technoscience.it\/en\/news\/tag\/industria-4-0\/\" class=\"tag-cloud-link tag-link-193 tag-link-position-8\" style=\"font-size: 10.709677419355pt;\" aria-label=\"Industria 4.0 (2 items)\">Industria 4.0<\/a>\n<a href=\"https:\/\/www.technoscience.it\/en\/news\/tag\/innovazione\/\" class=\"tag-cloud-link tag-link-64 tag-link-position-9\" style=\"font-size: 20.795698924731pt;\" aria-label=\"innovazione (13 items)\">innovazione<\/a>\n<a href=\"https:\/\/www.technoscience.it\/en\/news\/tag\/intelligenza-artificiale\/\" class=\"tag-cloud-link tag-link-39 tag-link-position-10\" style=\"font-size: 14.021505376344pt;\" aria-label=\"intelligenza artificiale (4 items)\">intelligenza artificiale<\/a>\n<a href=\"https:\/\/www.technoscience.it\/en\/news\/tag\/international-biotech-academy\/\" class=\"tag-cloud-link tag-link-199 tag-link-position-11\" style=\"font-size: 8pt;\" aria-label=\"International Biotech Academy (1 item)\">International Biotech Academy<\/a>\n<a href=\"https:\/\/www.technoscience.it\/en\/news\/tag\/invitalia\/\" class=\"tag-cloud-link tag-link-155 tag-link-position-12\" style=\"font-size: 8pt;\" aria-label=\"Invitalia (1 item)\">Invitalia<\/a>\n<a href=\"https:\/\/www.technoscience.it\/en\/news\/tag\/life-sciences\/\" class=\"tag-cloud-link tag-link-183 tag-link-position-13\" style=\"font-size: 22pt;\" aria-label=\"Life Sciences (16 items)\">Life Sciences<\/a>\n<a href=\"https:\/\/www.technoscience.it\/en\/news\/tag\/medicina-rigenerativa\/\" class=\"tag-cloud-link tag-link-129 tag-link-position-14\" style=\"font-size: 14.021505376344pt;\" aria-label=\"medicina rigenerativa (4 items)\">medicina rigenerativa<\/a>\n<a href=\"https:\/\/www.technoscience.it\/en\/news\/tag\/medicina-traslazionale\/\" class=\"tag-cloud-link tag-link-192 tag-link-position-15\" style=\"font-size: 15.225806451613pt;\" aria-label=\"Medicina traslazionale (5 items)\">Medicina traslazionale<\/a>\n<a href=\"https:\/\/www.technoscience.it\/en\/news\/tag\/networking-scientifico\/\" class=\"tag-cloud-link tag-link-195 tag-link-position-16\" style=\"font-size: 10.709677419355pt;\" aria-label=\"Networking scientifico (2 items)\">Networking scientifico<\/a>\n<a href=\"https:\/\/www.technoscience.it\/en\/news\/tag\/nutraceutica\/\" class=\"tag-cloud-link tag-link-186 tag-link-position-17\" style=\"font-size: 10.709677419355pt;\" aria-label=\"Nutraceutica (2 items)\">Nutraceutica<\/a>\n<a href=\"https:\/\/www.technoscience.it\/en\/news\/tag\/nutrizione-e-prevenzione\/\" class=\"tag-cloud-link tag-link-196 tag-link-position-18\" style=\"font-size: 10.709677419355pt;\" aria-label=\"Nutrizione e prevenzione (2 items)\">Nutrizione e prevenzione<\/a>\n<a href=\"https:\/\/www.technoscience.it\/en\/news\/tag\/pnrr\/\" class=\"tag-cloud-link tag-link-122 tag-link-position-19\" style=\"font-size: 14.021505376344pt;\" aria-label=\"PNRR (4 items)\">PNRR<\/a>\n<a href=\"https:\/\/www.technoscience.it\/en\/news\/tag\/ricerca-applicata\/\" class=\"tag-cloud-link tag-link-121 tag-link-position-20\" style=\"font-size: 20.795698924731pt;\" aria-label=\"Ricerca applicata (13 items)\">Ricerca applicata<\/a>\n<a href=\"https:\/\/www.technoscience.it\/en\/news\/tag\/rome-future-week\/\" class=\"tag-cloud-link tag-link-190 tag-link-position-21\" style=\"font-size: 10.709677419355pt;\" aria-label=\"Rome Future Week (2 items)\">Rome Future Week<\/a>\n<a href=\"https:\/\/www.technoscience.it\/en\/news\/tag\/rome-technopole\/\" class=\"tag-cloud-link tag-link-117 tag-link-position-22\" style=\"font-size: 10.709677419355pt;\" aria-label=\"Rome Technopole (2 items)\">Rome Technopole<\/a>\n<a href=\"https:\/\/www.technoscience.it\/en\/news\/tag\/sistema-invitalia\/\" class=\"tag-cloud-link tag-link-156 tag-link-position-23\" style=\"font-size: 8pt;\" aria-label=\"Sistema Invitalia (1 item)\">Sistema Invitalia<\/a>\n<a href=\"https:\/\/www.technoscience.it\/en\/news\/tag\/sostenibilita\/\" class=\"tag-cloud-link tag-link-184 tag-link-position-24\" style=\"font-size: 16.279569892473pt;\" aria-label=\"Sostenibilit\u00e0 (6 items)\">Sostenibilit\u00e0<\/a>\n<a href=\"https:\/\/www.technoscience.it\/en\/news\/tag\/startup-e-pmi\/\" class=\"tag-cloud-link tag-link-197 tag-link-position-25\" style=\"font-size: 12.516129032258pt;\" aria-label=\"Startup e PMI (3 items)\">Startup e PMI<\/a>\n<a href=\"https:\/\/www.technoscience.it\/en\/news\/tag\/tecnologie-indossabili\/\" class=\"tag-cloud-link tag-link-194 tag-link-position-26\" style=\"font-size: 12.516129032258pt;\" aria-label=\"Tecnologie indossabili (3 items)\">Tecnologie indossabili<\/a>\n<a href=\"https:\/\/www.technoscience.it\/en\/news\/tag\/telemedicina\/\" class=\"tag-cloud-link tag-link-187 tag-link-position-27\" style=\"font-size: 14.021505376344pt;\" aria-label=\"Telemedicina (4 items)\">Telemedicina<\/a>\n<a href=\"https:\/\/www.technoscience.it\/en\/news\/tag\/trasferimento-tecnologico\/\" class=\"tag-cloud-link tag-link-88 tag-link-position-28\" style=\"font-size: 17.784946236559pt;\" aria-label=\"Trasferimento tecnologico (8 items)\">Trasferimento tecnologico<\/a>\n<a href=\"https:\/\/www.technoscience.it\/en\/news\/tag\/trasformazione-digitale\/\" class=\"tag-cloud-link tag-link-189 tag-link-position-29\" style=\"font-size: 10.709677419355pt;\" aria-label=\"Trasformazione digitale (2 items)\">Trasformazione digitale<\/a><\/div>\n<\/nav>\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t\t<\/div>\n\t\t<\/div>\n\t\t\t\t\t<\/div>\n\t\t<\/section>\n\t\t\t\t<section class=\"elementor-section elementor-top-section elementor-element elementor-element-f5f07ea elementor-section-boxed elementor-section-height-default elementor-section-height-default\" data-id=\"f5f07ea\" data-element_type=\"section\" data-e-type=\"section\" data-settings=\"{&quot;background_background&quot;:&quot;classic&quot;}\">\n\t\t\t\t\t\t<div class=\"elementor-container elementor-column-gap-extended\">\n\t\t\t\t\t<div class=\"elementor-column elementor-col-100 elementor-top-column elementor-element elementor-element-688beaa\" data-id=\"688beaa\" data-element_type=\"column\" data-e-type=\"column\">\n\t\t\t<div class=\"elementor-widget-wrap elementor-element-populated\">\n\t\t\t\t\t\t<div class=\"elementor-element elementor-element-21171d4 elementor-position-inline-start elementor-tablet-position-inline-start elementor-view-default elementor-mobile-position-block-start elementor-widget elementor-widget-icon-box\" data-id=\"21171d4\" data-element_type=\"widget\" data-e-type=\"widget\" data-widget_type=\"icon-box.default\">\n\t\t\t\t<div class=\"elementor-widget-container\">\n\t\t\t\t\t\t\t<div class=\"elementor-icon-box-wrapper\">\n\n\t\t\t\t\t\t<div class=\"elementor-icon-box-icon\">\n\t\t\t\t<span  class=\"elementor-icon\">\n\t\t\t\t<svg aria-hidden=\"true\" class=\"e-font-icon-svg e-fas-hand-holding-medical\" viewBox=\"0 0 576 512\" xmlns=\"http:\/\/www.w3.org\/2000\/svg\"><path d=\"M159.88,175.82h64v64a16,16,0,0,0,16,16h64a16,16,0,0,0,16-16v-64h64a16,16,0,0,0,16-16v-64a16,16,0,0,0-16-16h-64v-64a16,16,0,0,0-16-16h-64a16,16,0,0,0-16,16v64h-64a16,16,0,0,0-16,16v64A16,16,0,0,0,159.88,175.82ZM568.07,336.13a39.91,39.91,0,0,0-55.93-8.47L392.47,415.84H271.86a16,16,0,0,1,0-32H350.1c16,0,30.75-10.87,33.37-26.61a32.06,32.06,0,0,0-31.62-37.38h-160a117.7,117.7,0,0,0-74.12,26.25l-46.5,37.74H15.87a16.11,16.11,0,0,0-16,16v96a16.11,16.11,0,0,0,16,16h347a104.8,104.8,0,0,0,61.7-20.27L559.6,392A40,40,0,0,0,568.07,336.13Z\"><\/path><\/svg>\t\t\t\t<\/span>\n\t\t\t<\/div>\n\t\t\t\n\t\t\t\t\t\t<div class=\"elementor-icon-box-content\">\n\n\t\t\t\t\t\t\t\t\t<h3 class=\"elementor-icon-box-title\">\n\t\t\t\t\t\t<span  >\n\t\t\t\t\t\t\tEndpoint che contano davvero (e come si scelgono)\t\t\t\t\t\t<\/span>\n\t\t\t\t\t<\/h3>\n\t\t\t\t\n\t\t\t\t\n\t\t\t<\/div>\n\t\t\t\n\t\t<\/div>\n\t\t\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t<div class=\"elementor-element elementor-element-7a6d86b elementor-widget-divider--view-line elementor-widget elementor-widget-divider\" data-id=\"7a6d86b\" data-element_type=\"widget\" data-e-type=\"widget\" data-widget_type=\"divider.default\">\n\t\t\t\t<div class=\"elementor-widget-container\">\n\t\t\t\t\t\t\t<div class=\"elementor-divider\">\n\t\t\t<span class=\"elementor-divider-separator\">\n\t\t\t\t\t\t<\/span>\n\t\t<\/div>\n\t\t\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t<div class=\"elementor-element elementor-element-054bfd3 elementor-widget elementor-widget-text-editor\" data-id=\"054bfd3\" data-element_type=\"widget\" data-e-type=\"widget\" data-widget_type=\"text-editor.default\">\n\t\t\t\t<div class=\"elementor-widget-container\">\n\t\t\t\t\t\t\t\t\t<p>Gli <strong>endpoint<\/strong> non sono una formalit\u00e0. Nelle rare devono essere <strong>sensibili<\/strong>, <strong>clinicamente significativi<\/strong> e misurabili nel tempo a disposizione. Talvolta un <strong>surrogato<\/strong> \u00e8 accettabile (enzimi, biomarcatori di accumulo, imaging quantitativo) se esiste una <strong>ragionevole plausibilit\u00e0<\/strong> che anticipi un beneficio clinico. Altre volte servono <strong>outcome funzionali<\/strong>: capacit\u00e0 motoria, respiratoria, cardiaca, neurocognitiva, <strong>PRO\/HRQoL<\/strong> con strumenti validati per quella popolazione. L\u2019errore tipico \u00e8 scegliere misure troppo \u201cpure\u201d da laboratorio o, all\u2019opposto, indicatori generici che non si muovono nei tempi di uno studio realisticamente fattibile. La via di mezzo \u00e8 un <strong>pannello di endpoint<\/strong> con una gerarchia chiara: il <strong>primario<\/strong> che decide, i <strong>secondari<\/strong> che spiegano, gli <strong>esploratori<\/strong> che preparano il futuro.<\/p>\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t<div class=\"elementor-element elementor-element-3fc2d84 elementor-position-inline-start elementor-tablet-position-inline-start elementor-view-default elementor-mobile-position-block-start elementor-widget elementor-widget-icon-box\" data-id=\"3fc2d84\" data-element_type=\"widget\" data-e-type=\"widget\" data-widget_type=\"icon-box.default\">\n\t\t\t\t<div class=\"elementor-widget-container\">\n\t\t\t\t\t\t\t<div class=\"elementor-icon-box-wrapper\">\n\n\t\t\t\t\t\t<div class=\"elementor-icon-box-icon\">\n\t\t\t\t<span  class=\"elementor-icon\">\n\t\t\t\t<svg aria-hidden=\"true\" class=\"e-font-icon-svg e-fas-flushed\" viewBox=\"0 0 496 512\" xmlns=\"http:\/\/www.w3.org\/2000\/svg\"><path d=\"M344 200c-13.3 0-24 10.7-24 24s10.7 24 24 24 24-10.7 24-24-10.7-24-24-24zm-192 0c-13.3 0-24 10.7-24 24s10.7 24 24 24 24-10.7 24-24-10.7-24-24-24zM248 8C111 8 0 119 0 256s111 248 248 248 248-111 248-248S385 8 248 8zM80 224c0-39.8 32.2-72 72-72s72 32.2 72 72-32.2 72-72 72-72-32.2-72-72zm232 176H184c-21.2 0-21.2-32 0-32h128c21.2 0 21.2 32 0 32zm32-104c-39.8 0-72-32.2-72-72s32.2-72 72-72 72 32.2 72 72-32.2 72-72 72z\"><\/path><\/svg>\t\t\t\t<\/span>\n\t\t\t<\/div>\n\t\t\t\n\t\t\t\t\t\t<div class=\"elementor-icon-box-content\">\n\n\t\t\t\t\t\t\t\t\t<h3 class=\"elementor-icon-box-title\">\n\t\t\t\t\t\t<span  >\n\t\t\t\t\t\t\tReal-world data e telemedicina: dal centro alla vita reale\t\t\t\t\t\t<\/span>\n\t\t\t\t\t<\/h3>\n\t\t\t\t\n\t\t\t\t\n\t\t\t<\/div>\n\t\t\t\n\t\t<\/div>\n\t\t\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t<div class=\"elementor-element elementor-element-f69b189 elementor-widget-divider--view-line elementor-widget elementor-widget-divider\" data-id=\"f69b189\" data-element_type=\"widget\" data-e-type=\"widget\" data-widget_type=\"divider.default\">\n\t\t\t\t<div class=\"elementor-widget-container\">\n\t\t\t\t\t\t\t<div class=\"elementor-divider\">\n\t\t\t<span class=\"elementor-divider-separator\">\n\t\t\t\t\t\t<\/span>\n\t\t<\/div>\n\t\t\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t<div class=\"elementor-element elementor-element-97d1dee elementor-widget elementor-widget-text-editor\" data-id=\"97d1dee\" data-element_type=\"widget\" data-e-type=\"widget\" data-widget_type=\"text-editor.default\">\n\t\t\t\t<div class=\"elementor-widget-container\">\n\t\t\t\t\t\t\t\t\t<p>Nei contesti rari, i <strong>real-world data<\/strong> non sono un ripiego: sono la prosecuzione naturale della prova. Telemedicina, sensori, cartelle cliniche e registri post-approval permettono di seguire coorti piccole nel tempo lungo che serve per capire se un beneficio regge. Le misure <strong>digitali<\/strong> continue (mobilit\u00e0, ritmo sonno-veglia, carichi aritmici, pattern respiratori) aggiungono granularit\u00e0 e trasformano la visita trimestrale in un <strong>racconto continuo<\/strong>. Ma valgono solo se c\u2019\u00e8 <strong>qualit\u00e0<\/strong>: definizioni stabili, piani di <strong>imputazione<\/strong> chiari, gestione del <strong>drift<\/strong> dei sensori, audit trail.<\/p>\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t<div class=\"elementor-element elementor-element-2ecb792 elementor-position-inline-start elementor-tablet-position-inline-start elementor-view-default elementor-mobile-position-block-start elementor-widget elementor-widget-icon-box\" data-id=\"2ecb792\" data-element_type=\"widget\" data-e-type=\"widget\" data-widget_type=\"icon-box.default\">\n\t\t\t\t<div class=\"elementor-widget-container\">\n\t\t\t\t\t\t\t<div class=\"elementor-icon-box-wrapper\">\n\n\t\t\t\t\t\t<div class=\"elementor-icon-box-icon\">\n\t\t\t\t<span  class=\"elementor-icon\">\n\t\t\t\t<svg aria-hidden=\"true\" class=\"e-font-icon-svg e-fas-file-medical\" viewBox=\"0 0 384 512\" xmlns=\"http:\/\/www.w3.org\/2000\/svg\"><path d=\"M377 105L279.1 7c-4.5-4.5-10.6-7-17-7H256v128h128v-6.1c0-6.3-2.5-12.4-7-16.9zm-153 31V0H24C10.7 0 0 10.7 0 24v464c0 13.3 10.7 24 24 24h336c13.3 0 24-10.7 24-24V160H248c-13.2 0-24-10.8-24-24zm64 160v48c0 4.4-3.6 8-8 8h-56v56c0 4.4-3.6 8-8 8h-48c-4.4 0-8-3.6-8-8v-56h-56c-4.4 0-8-3.6-8-8v-48c0-4.4 3.6-8 8-8h56v-56c0-4.4 3.6-8 8-8h48c4.4 0 8 3.6 8 8v56h56c4.4 0 8 3.6 8 8z\"><\/path><\/svg>\t\t\t\t<\/span>\n\t\t\t<\/div>\n\t\t\t\n\t\t\t\t\t\t<div class=\"elementor-icon-box-content\">\n\n\t\t\t\t\t\t\t\t\t<h3 class=\"elementor-icon-box-title\">\n\t\t\t\t\t\t<span  >\n\t\t\t\t\t\t\tPatient engagement: partecipazione che cambia la scienza\t\t\t\t\t\t<\/span>\n\t\t\t\t\t<\/h3>\n\t\t\t\t\n\t\t\t\t\n\t\t\t<\/div>\n\t\t\t\n\t\t<\/div>\n\t\t\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t<div class=\"elementor-element elementor-element-cb08623 elementor-widget-divider--view-line elementor-widget elementor-widget-divider\" data-id=\"cb08623\" data-element_type=\"widget\" data-e-type=\"widget\" data-widget_type=\"divider.default\">\n\t\t\t\t<div class=\"elementor-widget-container\">\n\t\t\t\t\t\t\t<div class=\"elementor-divider\">\n\t\t\t<span class=\"elementor-divider-separator\">\n\t\t\t\t\t\t<\/span>\n\t\t<\/div>\n\t\t\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t<div class=\"elementor-element elementor-element-4ae3f29 elementor-widget elementor-widget-text-editor\" data-id=\"4ae3f29\" data-element_type=\"widget\" data-e-type=\"widget\" data-widget_type=\"text-editor.default\">\n\t\t\t\t<div class=\"elementor-widget-container\">\n\t\t\t\t\t\t\t\t\t<p>Nelle rare, i pazienti e le associazioni sono <strong>co-autori<\/strong> del percorso. Dalla definizione degli endpoint (cosa \u00e8 \u201cbeneficio\u201d nella vita di tutti i giorni) alla fattibilit\u00e0 delle procedure (quante visite, quante ore, quali pesi), fino alla <strong>restituzione dei risultati<\/strong>, la loro voce modifica davvero il disegno e la <strong>retention<\/strong>. Il coinvolgimento non \u00e8 folklore: migliora l\u2019aderenza, riduce i missing, rende <strong>utili<\/strong> gli outcome di qualit\u00e0 di vita. \u00c8 anche una leva di <strong>etica pratica<\/strong>: chiediamo ci\u00f2 che serve, non ci\u00f2 che \u201csi chiede sempre\u201d.<\/p>\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t<div class=\"elementor-element elementor-element-37106fb elementor-position-inline-start elementor-tablet-position-inline-start elementor-view-default elementor-mobile-position-block-start elementor-widget elementor-widget-icon-box\" data-id=\"37106fb\" data-element_type=\"widget\" data-e-type=\"widget\" data-widget_type=\"icon-box.default\">\n\t\t\t\t<div class=\"elementor-widget-container\">\n\t\t\t\t\t\t\t<div class=\"elementor-icon-box-wrapper\">\n\n\t\t\t\t\t\t<div class=\"elementor-icon-box-icon\">\n\t\t\t\t<span  class=\"elementor-icon\">\n\t\t\t\t<svg aria-hidden=\"true\" class=\"e-font-icon-svg e-fas-flask\" viewBox=\"0 0 448 512\" xmlns=\"http:\/\/www.w3.org\/2000\/svg\"><path d=\"M437.2 403.5L320 215V64h8c13.3 0 24-10.7 24-24V24c0-13.3-10.7-24-24-24H120c-13.3 0-24 10.7-24 24v16c0 13.3 10.7 24 24 24h8v151L10.8 403.5C-18.5 450.6 15.3 512 70.9 512h306.2c55.7 0 89.4-61.5 60.1-108.5zM137.9 320l48.2-77.6c3.7-5.2 5.8-11.6 5.8-18.4V64h64v160c0 6.9 2.2 13.2 5.8 18.4l48.2 77.6h-172z\"><\/path><\/svg>\t\t\t\t<\/span>\n\t\t\t<\/div>\n\t\t\t\n\t\t\t\t\t\t<div class=\"elementor-icon-box-content\">\n\n\t\t\t\t\t\t\t\t\t<h3 class=\"elementor-icon-box-title\">\n\t\t\t\t\t\t<span  >\n\t\t\t\t\t\t\tSostenibilit\u00e0 clinica: oltre il p-value\t\t\t\t\t\t<\/span>\n\t\t\t\t\t<\/h3>\n\t\t\t\t\n\t\t\t\t\n\t\t\t<\/div>\n\t\t\t\n\t\t<\/div>\n\t\t\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t<div class=\"elementor-element elementor-element-cf91280 elementor-widget-divider--view-line elementor-widget elementor-widget-divider\" data-id=\"cf91280\" data-element_type=\"widget\" data-e-type=\"widget\" data-widget_type=\"divider.default\">\n\t\t\t\t<div class=\"elementor-widget-container\">\n\t\t\t\t\t\t\t<div class=\"elementor-divider\">\n\t\t\t<span class=\"elementor-divider-separator\">\n\t\t\t\t\t\t<\/span>\n\t\t<\/div>\n\t\t\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t<div class=\"elementor-element elementor-element-5de8d27 elementor-widget elementor-widget-text-editor\" data-id=\"5de8d27\" data-element_type=\"widget\" data-e-type=\"widget\" data-widget_type=\"text-editor.default\">\n\t\t\t\t<div class=\"elementor-widget-container\">\n\t\t\t\t\t\t\t\t\t<p>Una terapia rara \u00e8 sostenibile quando si <strong>integra<\/strong> senza spezzare i percorsi, quando i requisiti di <strong>monitoraggio<\/strong> sono realistici, quando il <strong>profilo di sicurezza<\/strong> \u00e8 presidiabile nella pratica e quando il <strong>valore clinico<\/strong> ha senso anche per payer e HTA. Per questo mettiamo a fuoco presto <strong>resource use<\/strong>, bisogno di centri esperti, training, dispositivi associati, e ipotizziamo <strong>scenari di adozione<\/strong>: non basta che funzioni, deve poter funzionare nel mondo reale. Gli impegni <strong>post-approval<\/strong> (studi di conferma, registri obbligatori, sorveglianza) non sono un peso accessorio: sono parte del patto.<\/p>\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t\t<\/div>\n\t\t<\/div>\n\t\t\t\t\t<\/div>\n\t\t<\/section>\n\t\t\t\t<section class=\"elementor-section elementor-top-section elementor-element elementor-element-d487a8e elementor-section-boxed elementor-section-height-default elementor-section-height-default\" data-id=\"d487a8e\" data-element_type=\"section\" data-e-type=\"section\" data-settings=\"{&quot;background_background&quot;:&quot;classic&quot;}\">\n\t\t\t\t\t\t<div class=\"elementor-container elementor-column-gap-extended\">\n\t\t\t\t\t<div class=\"elementor-column elementor-col-100 elementor-top-column elementor-element elementor-element-9c728c4\" data-id=\"9c728c4\" data-element_type=\"column\" data-e-type=\"column\">\n\t\t\t<div class=\"elementor-widget-wrap elementor-element-populated\">\n\t\t\t\t\t\t<div class=\"elementor-element elementor-element-d539d93 elementor-position-inline-start elementor-tablet-position-inline-start elementor-view-default elementor-mobile-position-block-start elementor-widget elementor-widget-icon-box\" data-id=\"d539d93\" data-element_type=\"widget\" data-e-type=\"widget\" data-widget_type=\"icon-box.default\">\n\t\t\t\t<div class=\"elementor-widget-container\">\n\t\t\t\t\t\t\t<div class=\"elementor-icon-box-wrapper\">\n\n\t\t\t\t\t\t<div class=\"elementor-icon-box-icon\">\n\t\t\t\t<span  class=\"elementor-icon\">\n\t\t\t\t<svg aria-hidden=\"true\" class=\"e-font-icon-svg e-fas-file-signature\" viewBox=\"0 0 576 512\" xmlns=\"http:\/\/www.w3.org\/2000\/svg\"><path d=\"M218.17 424.14c-2.95-5.92-8.09-6.52-10.17-6.52s-7.22.59-10.02 6.19l-7.67 15.34c-6.37 12.78-25.03 11.37-29.48-2.09L144 386.59l-10.61 31.88c-5.89 17.66-22.38 29.53-41 29.53H80c-8.84 0-16-7.16-16-16s7.16-16 16-16h12.39c4.83 0 9.11-3.08 10.64-7.66l18.19-54.64c3.3-9.81 12.44-16.41 22.78-16.41s19.48 6.59 22.77 16.41l13.88 41.64c19.75-16.19 54.06-9.7 66 14.16 1.89 3.78 5.49 5.95 9.36 6.26v-82.12l128-127.09V160H248c-13.2 0-24-10.8-24-24V0H24C10.7 0 0 10.7 0 24v464c0 13.3 10.7 24 24 24h336c13.3 0 24-10.7 24-24v-40l-128-.11c-16.12-.31-30.58-9.28-37.83-23.75zM384 121.9c0-6.3-2.5-12.4-7-16.9L279.1 7c-4.5-4.5-10.6-7-17-7H256v128h128v-6.1zm-96 225.06V416h68.99l161.68-162.78-67.88-67.88L288 346.96zm280.54-179.63l-31.87-31.87c-9.94-9.94-26.07-9.94-36.01 0l-27.25 27.25 67.88 67.88 27.25-27.25c9.95-9.94 9.95-26.07 0-36.01z\"><\/path><\/svg>\t\t\t\t<\/span>\n\t\t\t<\/div>\n\t\t\t\n\t\t\t\t\t\t<div class=\"elementor-icon-box-content\">\n\n\t\t\t\t\t\t\t\t\t<h3 class=\"elementor-icon-box-title\">\n\t\t\t\t\t\t<span  >\n\t\t\t\t\t\t\tCome lavoriamo quando la patologia \u00e8 rara\t\t\t\t\t\t<\/span>\n\t\t\t\t\t<\/h3>\n\t\t\t\t\n\t\t\t\t\n\t\t\t<\/div>\n\t\t\t\n\t\t<\/div>\n\t\t\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t<div class=\"elementor-element elementor-element-7fd0026 elementor-widget-divider--view-line elementor-widget elementor-widget-divider\" data-id=\"7fd0026\" data-element_type=\"widget\" data-e-type=\"widget\" data-widget_type=\"divider.default\">\n\t\t\t\t<div class=\"elementor-widget-container\">\n\t\t\t\t\t\t\t<div class=\"elementor-divider\">\n\t\t\t<span class=\"elementor-divider-separator\">\n\t\t\t\t\t\t<\/span>\n\t\t<\/div>\n\t\t\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t<div class=\"elementor-element elementor-element-0151eb5 elementor-widget elementor-widget-text-editor\" data-id=\"0151eb5\" data-element_type=\"widget\" data-e-type=\"widget\" data-widget_type=\"text-editor.default\">\n\t\t\t\t<div class=\"elementor-widget-container\">\n\t\t\t\t\t\t\t\t\t<p>All\u2019avvio chiediamo tre certezze. Una <strong>diagnosi<\/strong> operativa chiara (criteri e test), una <strong>storia naturale<\/strong> minima su cui ancorare scelte e dimensionamenti, e un\u2019idea precisa di <strong>endpoint<\/strong> misurabili nel tempo realistico dello studio. Aiutiamo a costruire o potenziare i <strong>registri<\/strong>, a scegliere <strong>disegni<\/strong> compatibili con la biologia e con l\u2019etica, a mappare fin da subito la <strong>via regolatoria<\/strong> pi\u00f9 sensata (Orphan, percorsi condizionati) per non rimandare questioni che torneranno comunque. Quando serve, progettiamo <strong>piani di raccolta RWD<\/strong> e telemedicina per estendere lo sguardo oltre il perimetro del trial.<\/p>\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t\t<\/div>\n\t\t<\/div>\n\t\t\t\t\t<\/div>\n\t\t<\/section>\n\t\t\t\t<section class=\"elementor-section elementor-top-section elementor-element elementor-element-c6a7408 elementor-section-content-middle elementor-reverse-mobile elementor-section-boxed elementor-section-height-default elementor-section-height-default\" data-id=\"c6a7408\" data-element_type=\"section\" data-e-type=\"section\" data-settings=\"{&quot;background_background&quot;:&quot;classic&quot;}\">\n\t\t\t\t\t\t<div class=\"elementor-container elementor-column-gap-extended\">\n\t\t\t\t\t<div class=\"elementor-column elementor-col-33 elementor-top-column elementor-element elementor-element-ebc0b4e\" data-id=\"ebc0b4e\" data-element_type=\"column\" data-e-type=\"column\" data-settings=\"{&quot;background_background&quot;:&quot;classic&quot;}\">\n\t\t\t<div class=\"elementor-widget-wrap elementor-element-populated\">\n\t\t\t\t\t\t<div class=\"elementor-element elementor-element-aeaed70 e-grid-align-center e-grid-align-mobile-center e-grid-align-tablet-center elementor-shape-circle elementor-grid-0 elementor-widget elementor-widget-social-icons\" data-id=\"aeaed70\" data-element_type=\"widget\" data-e-type=\"widget\" data-widget_type=\"social-icons.default\">\n\t\t\t\t<div class=\"elementor-widget-container\">\n\t\t\t\t\t\t\t<div class=\"elementor-social-icons-wrapper elementor-grid\" role=\"list\">\n\t\t\t\t\t\t\t<span class=\"elementor-grid-item\" role=\"listitem\">\n\t\t\t\t\t<a class=\"elementor-icon elementor-social-icon elementor-social-icon-linkedin elementor-repeater-item-9083030\" href=\"https:\/\/www.linkedin.com\/company\/technoscience-parco-scientifo-e-tecnologico-pontino\/\">\n\t\t\t\t\t\t<span class=\"elementor-screen-only\">Linkedin<\/span>\n\t\t\t\t\t\t<svg aria-hidden=\"true\" class=\"e-font-icon-svg e-fab-linkedin\" viewBox=\"0 0 448 512\" xmlns=\"http:\/\/www.w3.org\/2000\/svg\"><path d=\"M416 32H31.9C14.3 32 0 46.5 0 64.3v383.4C0 465.5 14.3 480 31.9 480H416c17.6 0 32-14.5 32-32.3V64.3c0-17.8-14.4-32.3-32-32.3zM135.4 416H69V202.2h66.5V416zm-33.2-243c-21.3 0-38.5-17.3-38.5-38.5S80.9 96 102.2 96c21.2 0 38.5 17.3 38.5 38.5 0 21.3-17.2 38.5-38.5 38.5zm282.1 243h-66.4V312c0-24.8-.5-56.7-34.5-56.7-34.6 0-39.9 27-39.9 54.9V416h-66.4V202.2h63.7v29.2h.9c8.9-16.8 30.6-34.5 62.9-34.5 67.2 0 79.7 44.3 79.7 101.9V416z\"><\/path><\/svg>\t\t\t\t\t<\/a>\n\t\t\t\t<\/span>\n\t\t\t\t\t\t\t<span class=\"elementor-grid-item\" role=\"listitem\">\n\t\t\t\t\t<a class=\"elementor-icon elementor-social-icon elementor-social-icon-facebook elementor-repeater-item-2f55736\" href=\"https:\/\/www.facebook.com\/PSTechnoscience\">\n\t\t\t\t\t\t<span class=\"elementor-screen-only\">Facebook<\/span>\n\t\t\t\t\t\t<svg aria-hidden=\"true\" class=\"e-font-icon-svg e-fab-facebook\" viewBox=\"0 0 512 512\" xmlns=\"http:\/\/www.w3.org\/2000\/svg\"><path d=\"M504 256C504 119 393 8 256 8S8 119 8 256c0 123.78 90.69 226.38 209.25 245V327.69h-63V256h63v-54.64c0-62.15 37-96.48 93.67-96.48 27.14 0 55.52 4.84 55.52 4.84v61h-31.28c-30.8 0-40.41 19.12-40.41 38.73V256h68.78l-11 71.69h-57.78V501C413.31 482.38 504 379.78 504 256z\"><\/path><\/svg>\t\t\t\t\t<\/a>\n\t\t\t\t<\/span>\n\t\t\t\t\t\t\t<span class=\"elementor-grid-item\" role=\"listitem\">\n\t\t\t\t\t<a class=\"elementor-icon elementor-social-icon elementor-social-icon-instagram elementor-repeater-item-aecdcd0\" href=\"https:\/\/www.instagram.com\/technoscience_pstp\/\">\n\t\t\t\t\t\t<span class=\"elementor-screen-only\">Instagram<\/span>\n\t\t\t\t\t\t<svg aria-hidden=\"true\" class=\"e-font-icon-svg e-fab-instagram\" viewBox=\"0 0 448 512\" xmlns=\"http:\/\/www.w3.org\/2000\/svg\"><path d=\"M224.1 141c-63.6 0-114.9 51.3-114.9 114.9s51.3 114.9 114.9 114.9S339 319.5 339 255.9 287.7 141 224.1 141zm0 189.6c-41.1 0-74.7-33.5-74.7-74.7s33.5-74.7 74.7-74.7 74.7 33.5 74.7 74.7-33.6 74.7-74.7 74.7zm146.4-194.3c0 14.9-12 26.8-26.8 26.8-14.9 0-26.8-12-26.8-26.8s12-26.8 26.8-26.8 26.8 12 26.8 26.8zm76.1 27.2c-1.7-35.9-9.9-67.7-36.2-93.9-26.2-26.2-58-34.4-93.9-36.2-37-2.1-147.9-2.1-184.9 0-35.8 1.7-67.6 9.9-93.9 36.1s-34.4 58-36.2 93.9c-2.1 37-2.1 147.9 0 184.9 1.7 35.9 9.9 67.7 36.2 93.9s58 34.4 93.9 36.2c37 2.1 147.9 2.1 184.9 0 35.9-1.7 67.7-9.9 93.9-36.2 26.2-26.2 34.4-58 36.2-93.9 2.1-37 2.1-147.8 0-184.8zM398.8 388c-7.8 19.6-22.9 34.7-42.6 42.6-29.5 11.7-99.5 9-132.1 9s-102.7 2.6-132.1-9c-19.6-7.8-34.7-22.9-42.6-42.6-11.7-29.5-9-99.5-9-132.1s-2.6-102.7 9-132.1c7.8-19.6 22.9-34.7 42.6-42.6 29.5-11.7 99.5-9 132.1-9s102.7-2.6 132.1 9c19.6 7.8 34.7 22.9 42.6 42.6 11.7 29.5 9 99.5 9 132.1s2.7 102.7-9 132.1z\"><\/path><\/svg>\t\t\t\t\t<\/a>\n\t\t\t\t<\/span>\n\t\t\t\t\t\t\t<span class=\"elementor-grid-item\" role=\"listitem\">\n\t\t\t\t\t<a class=\"elementor-icon elementor-social-icon elementor-social-icon-youtube elementor-repeater-item-664baeb\" href=\"https:\/\/www.youtube.com\/@technoscience3169\">\n\t\t\t\t\t\t<span class=\"elementor-screen-only\">Youtube<\/span>\n\t\t\t\t\t\t<svg aria-hidden=\"true\" class=\"e-font-icon-svg e-fab-youtube\" viewBox=\"0 0 576 512\" xmlns=\"http:\/\/www.w3.org\/2000\/svg\"><path d=\"M549.655 124.083c-6.281-23.65-24.787-42.276-48.284-48.597C458.781 64 288 64 288 64S117.22 64 74.629 75.486c-23.497 6.322-42.003 24.947-48.284 48.597-11.412 42.867-11.412 132.305-11.412 132.305s0 89.438 11.412 132.305c6.281 23.65 24.787 41.5 48.284 47.821C117.22 448 288 448 288 448s170.78 0 213.371-11.486c23.497-6.321 42.003-24.171 48.284-47.821 11.412-42.867 11.412-132.305 11.412-132.305s0-89.438-11.412-132.305zm-317.51 213.508V175.185l142.739 81.205-142.739 81.201z\"><\/path><\/svg>\t\t\t\t\t<\/a>\n\t\t\t\t<\/span>\n\t\t\t\t\t<\/div>\n\t\t\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t\t<\/div>\n\t\t<\/div>\n\t\t\t\t<div class=\"elementor-column elementor-col-33 elementor-top-column elementor-element elementor-element-47061e0\" data-id=\"47061e0\" data-element_type=\"column\" data-e-type=\"column\" data-settings=\"{&quot;background_background&quot;:&quot;classic&quot;}\">\n\t\t\t<div class=\"elementor-widget-wrap elementor-element-populated\">\n\t\t\t\t\t\t<div class=\"elementor-element elementor-element-7bc1e52 elementor-widget elementor-widget-heading\" data-id=\"7bc1e52\" data-element_type=\"widget\" data-e-type=\"widget\" data-widget_type=\"heading.default\">\n\t\t\t\t<div class=\"elementor-widget-container\">\n\t\t\t\t\t<h2 class=\"elementor-heading-title elementor-size-default\">Scegli Technoscience!<\/h2>\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t<div class=\"elementor-element elementor-element-a854892 elementor-widget elementor-widget-divider\" data-id=\"a854892\" data-element_type=\"widget\" data-e-type=\"widget\" data-widget_type=\"divider.default\">\n\t\t\t\t<div class=\"elementor-widget-container\">\n\t\t\t\t\t\t\t<div class=\"elementor-divider\">\n\t\t\t<span class=\"elementor-divider-separator\">\n\t\t\t\t\t\t<\/span>\n\t\t<\/div>\n\t\t\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t<div class=\"elementor-element elementor-element-50d59b0 elementor-widget elementor-widget-heading\" data-id=\"50d59b0\" data-element_type=\"widget\" data-e-type=\"widget\" data-widget_type=\"heading.default\">\n\t\t\t\t<div class=\"elementor-widget-container\">\n\t\t\t\t\t<p class=\"elementor-heading-title elementor-size-default\">Se stai progettando uno studio che richiede campioni e dati di qualit\u00e0 verificata, chiedi un fit check: in una call breve allineiamo domanda clinica o industriale, disponibilit\u00e0 di campioni, requisiti etici e standard di qualit\u00e0; se il perimetro \u00e8 solido, pianifichiamo un percorso che porti dall\u2019accesso responsabile alla validazione, senza perdere tempo..\n<\/p>\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t<div class=\"elementor-element elementor-element-5e2e314 elementor-button-align-start elementor-mobile-button-align-stretch elementor-widget elementor-widget-form\" data-id=\"5e2e314\" data-element_type=\"widget\" data-e-type=\"widget\" data-settings=\"{&quot;step_next_label&quot;:&quot;Next&quot;,&quot;step_previous_label&quot;:&quot;Previous&quot;,&quot;button_width&quot;:&quot;40&quot;,&quot;button_width_tablet&quot;:&quot;50&quot;,&quot;button_width_mobile&quot;:&quot;100&quot;,&quot;step_type&quot;:&quot;number_text&quot;,&quot;step_icon_shape&quot;:&quot;circle&quot;}\" data-widget_type=\"form.default\">\n\t\t\t\t<div class=\"elementor-widget-container\">\n\t\t\t\t\t\t\t<form class=\"elementor-form\" method=\"post\" name=\"New Form\" aria-label=\"New Form\">\n\t\t\t<input type=\"hidden\" name=\"post_id\" value=\"3766\"\/>\n\t\t\t<input type=\"hidden\" name=\"form_id\" value=\"5e2e314\"\/>\n\t\t\t<input type=\"hidden\" name=\"referer_title\" value=\"Rare Disease Studies: Endpoints and Models - Technoscience\" \/>\n\n\t\t\t\t\t\t\t<input type=\"hidden\" name=\"queried_id\" value=\"3766\"\/>\n\t\t\t\n\t\t\t<div class=\"elementor-form-fields-wrapper elementor-labels-\">\n\t\t\t\t\t\t\t\t<div class=\"elementor-field-type-text elementor-field-group elementor-column elementor-field-group-name elementor-col-50 elementor-field-required\">\n\t\t\t\t\t\t\t\t\t\t\t\t<label for=\"form-field-name\" class=\"elementor-field-label elementor-screen-only\">\n\t\t\t\t\t\t\t\tNome\t\t\t\t\t\t\t<\/label>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<input size=\"1\" type=\"text\" name=\"form_fields[name]\" id=\"form-field-name\" class=\"elementor-field elementor-size-md  elementor-field-textual\" placeholder=\"Nome\" required=\"required\">\n\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t<div class=\"elementor-field-type-text elementor-field-group elementor-column elementor-field-group-field_24422e2 elementor-col-50 elementor-field-required\">\n\t\t\t\t\t\t\t\t\t\t\t\t<label for=\"form-field-field_24422e2\" class=\"elementor-field-label elementor-screen-only\">\n\t\t\t\t\t\t\t\tCognome\t\t\t\t\t\t\t<\/label>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<input size=\"1\" type=\"text\" name=\"form_fields[field_24422e2]\" id=\"form-field-field_24422e2\" class=\"elementor-field elementor-size-md  elementor-field-textual\" placeholder=\"Cognome\" required=\"required\">\n\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t<div class=\"elementor-field-type-email elementor-field-group elementor-column elementor-field-group-email elementor-col-50 elementor-field-required\">\n\t\t\t\t\t\t\t\t\t\t\t\t<label for=\"form-field-email\" class=\"elementor-field-label elementor-screen-only\">\n\t\t\t\t\t\t\t\tEmail\t\t\t\t\t\t\t<\/label>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<input size=\"1\" type=\"email\" name=\"form_fields[email]\" id=\"form-field-email\" class=\"elementor-field elementor-size-md  elementor-field-textual\" placeholder=\"Email\" required=\"required\">\n\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t<div class=\"elementor-field-type-tel elementor-field-group elementor-column elementor-field-group-field_189ff95 elementor-col-50\">\n\t\t\t\t\t\t\t\t\t\t\t\t<label for=\"form-field-field_189ff95\" class=\"elementor-field-label elementor-screen-only\">\n\t\t\t\t\t\t\t\tTelefono\t\t\t\t\t\t\t<\/label>\n\t\t\t\t\t\t\t\t<input size=\"1\" type=\"tel\" name=\"form_fields[field_189ff95]\" id=\"form-field-field_189ff95\" class=\"elementor-field elementor-size-md  elementor-field-textual\" placeholder=\"Telefono\" pattern=\"[0-9()#&amp;+*-=.]+\" title=\"Only numbers and phone characters (#, -, *, etc) are accepted.\">\n\n\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t<div class=\"elementor-field-type-textarea elementor-field-group elementor-column elementor-field-group-message elementor-col-100 elementor-field-required\">\n\t\t\t\t\t\t\t\t\t\t\t\t<label for=\"form-field-message\" class=\"elementor-field-label elementor-screen-only\">\n\t\t\t\t\t\t\t\tMessaggio\t\t\t\t\t\t\t<\/label>\n\t\t\t\t\t\t<textarea class=\"elementor-field-textual elementor-field  elementor-size-md\" name=\"form_fields[message]\" id=\"form-field-message\" rows=\"4\" placeholder=\"Messaggio\" required=\"required\"><\/textarea>\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t<div class=\"elementor-field-type-acceptance elementor-field-group elementor-column elementor-field-group-field_5f71bcb elementor-col-100 elementor-field-required\">\n\t\t\t\t\t\t\t\t\t\t\t\t<label for=\"form-field-field_5f71bcb\" class=\"elementor-field-label elementor-screen-only\">\n\t\t\t\t\t\t\t\tAcceptance\t\t\t\t\t\t\t<\/label>\n\t\t\t\t\t\t\t\t<div class=\"elementor-field-subgroup\">\n\t\t\t<span class=\"elementor-field-option\">\n\t\t\t\t<input type=\"checkbox\" name=\"form_fields[field_5f71bcb]\" id=\"form-field-field_5f71bcb\" class=\"elementor-field elementor-size-md  elementor-acceptance-field\" required=\"required\">\n\t\t\t\t<label for=\"form-field-field_5f71bcb\">Acconsento a Technoscience di raccogliere i miei dati tramite questo modulo. Dichiaro di aver letto e di accettare l'informativa sulla protezione dei dati disponibile al seguente link \n<a href=\"https:\/\/www.technoscience.it\/politicaprivacy\/\" target=\"_blank\">https:\/\/www.technoscience.it\/politicaprivacy\/<\/a><\/label>\t\t\t<\/span>\n\t\t<\/div>\n\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t<div class=\"elementor-field-group elementor-column elementor-field-type-submit elementor-col-40 e-form__buttons elementor-md-50 elementor-sm-100\">\n\t\t\t\t\t<button class=\"elementor-button elementor-size-md\" type=\"submit\">\n\t\t\t\t\t\t<span class=\"elementor-button-content-wrapper\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<span class=\"elementor-button-text\">invia<\/span>\n\t\t\t\t\t\t\t\t\t\t\t\t\t<\/span>\n\t\t\t\t\t<\/button>\n\t\t\t\t<\/div>\n\t\t\t<\/div>\n\t\t<\/form>\n\t\t\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t\t<\/div>\n\t\t<\/div>\n\t\t\t\t<div class=\"elementor-column elementor-col-33 elementor-top-column elementor-element elementor-element-547022b\" data-id=\"547022b\" data-element_type=\"column\" data-e-type=\"column\">\n\t\t\t<div class=\"elementor-widget-wrap elementor-element-populated\">\n\t\t\t\t\t\t<div class=\"elementor-element elementor-element-5a8e72a elementor-widget elementor-widget-image\" data-id=\"5a8e72a\" data-element_type=\"widget\" data-e-type=\"widget\" data-widget_type=\"image.default\">\n\t\t\t\t<div class=\"elementor-widget-container\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<img decoding=\"async\" width=\"1920\" height=\"1920\" src=\"https:\/\/www.technoscience.it\/wp-content\/uploads\/2025\/11\/scegli-tech.jpg\" class=\"attachment-full size-full wp-image-3264\" alt=\"Biotech contacts: professionals ready to collaborate and innovate\" srcset=\"https:\/\/www.technoscience.it\/wp-content\/uploads\/2025\/11\/scegli-tech.jpg 1920w, https:\/\/www.technoscience.it\/wp-content\/uploads\/2025\/11\/scegli-tech-300x300.jpg 300w, https:\/\/www.technoscience.it\/wp-content\/uploads\/2025\/11\/scegli-tech-1024x1024.jpg 1024w, https:\/\/www.technoscience.it\/wp-content\/uploads\/2025\/11\/scegli-tech-150x150.jpg 150w, https:\/\/www.technoscience.it\/wp-content\/uploads\/2025\/11\/scegli-tech-768x768.jpg 768w, https:\/\/www.technoscience.it\/wp-content\/uploads\/2025\/11\/scegli-tech-1536x1536.jpg 1536w, https:\/\/www.technoscience.it\/wp-content\/uploads\/2025\/11\/scegli-tech-12x12.jpg 12w\" sizes=\"(max-width: 1920px) 100vw, 1920px\" title=\"Biotech Contacts \u2013 Collaboration and Network\" \/>\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t\t<\/div>\n\t\t<\/div>\n\t\t\t\t\t<\/div>\n\t\t<\/section>\n\t\t\t\t<section class=\"elementor-section elementor-top-section elementor-element elementor-element-38ce07d elementor-section-boxed elementor-section-height-default elementor-section-height-default\" data-id=\"38ce07d\" data-element_type=\"section\" data-e-type=\"section\" data-settings=\"{&quot;background_background&quot;:&quot;classic&quot;}\">\n\t\t\t\t\t\t<div class=\"elementor-container elementor-column-gap-extended\">\n\t\t\t\t\t<div class=\"elementor-column elementor-col-100 elementor-top-column elementor-element elementor-element-8c99e09\" data-id=\"8c99e09\" data-element_type=\"column\" data-e-type=\"column\">\n\t\t\t<div class=\"elementor-widget-wrap elementor-element-populated\">\n\t\t\t\t\t\t<div class=\"elementor-element elementor-element-eec6deb elementor-widget elementor-widget-heading\" data-id=\"eec6deb\" data-element_type=\"widget\" data-e-type=\"widget\" data-widget_type=\"heading.default\">\n\t\t\t\t<div class=\"elementor-widget-container\">\n\t\t\t\t\t<h2 class=\"elementor-heading-title elementor-size-default\">FAQ<\/h2>\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t<div class=\"elementor-element elementor-element-cf24555 elementor-widget elementor-widget-heading\" data-id=\"cf24555\" data-element_type=\"widget\" data-e-type=\"widget\" data-widget_type=\"heading.default\">\n\t\t\t\t<div class=\"elementor-widget-container\">\n\t\t\t\t\t<h3 class=\"elementor-heading-title elementor-size-default\">Le cose che ci chiedete pi\u00f9 spesso<\/h3>\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t<section class=\"elementor-section elementor-inner-section elementor-element elementor-element-847c234 elementor-section-boxed elementor-section-height-default elementor-section-height-default\" data-id=\"847c234\" data-element_type=\"section\" data-e-type=\"section\">\n\t\t\t\t\t\t<div class=\"elementor-container elementor-column-gap-wide\">\n\t\t\t\t\t<div class=\"elementor-column elementor-col-50 elementor-inner-column elementor-element elementor-element-292b52c\" data-id=\"292b52c\" data-element_type=\"column\" data-e-type=\"column\">\n\t\t\t<div class=\"elementor-widget-wrap elementor-element-populated\">\n\t\t\t\t\t\t<div class=\"elementor-element elementor-element-49653fc elementor-widget elementor-widget-toggle\" data-id=\"49653fc\" data-element_type=\"widget\" data-e-type=\"widget\" data-widget_type=\"toggle.default\">\n\t\t\t\t<div class=\"elementor-widget-container\">\n\t\t\t\t\t\t\t<div class=\"elementor-toggle\">\n\t\t\t\t\t\t\t<div class=\"elementor-toggle-item\">\n\t\t\t\t\t<div id=\"elementor-tab-title-7691\" class=\"elementor-tab-title\" data-tab=\"1\" role=\"button\" aria-controls=\"elementor-tab-content-7691\" aria-expanded=\"false\">\n\t\t\t\t\t\t\t\t\t\t\t\t<span class=\"elementor-toggle-icon elementor-toggle-icon-right\" aria-hidden=\"true\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<span class=\"elementor-toggle-icon-closed\"><svg class=\"e-font-icon-svg e-fas-plus\" viewBox=\"0 0 448 512\" xmlns=\"http:\/\/www.w3.org\/2000\/svg\"><path d=\"M416 208H272V64c0-17.67-14.33-32-32-32h-32c-17.67 0-32 14.33-32 32v144H32c-17.67 0-32 14.33-32 32v32c0 17.67 14.33 32 32 32h144v144c0 17.67 14.33 32 32 32h32c17.67 0 32-14.33 32-32V304h144c17.67 0 32-14.33 32-32v-32c0-17.67-14.33-32-32-32z\"><\/path><\/svg><\/span>\n\t\t\t\t\t\t\t\t<span class=\"elementor-toggle-icon-opened\"><svg class=\"elementor-toggle-icon-opened e-font-icon-svg e-fas-plus\" viewBox=\"0 0 448 512\" xmlns=\"http:\/\/www.w3.org\/2000\/svg\"><path d=\"M416 208H272V64c0-17.67-14.33-32-32-32h-32c-17.67 0-32 14.33-32 32v144H32c-17.67 0-32 14.33-32 32v32c0 17.67 14.33 32 32 32h144v144c0 17.67 14.33 32 32 32h32c17.67 0 32-14.33 32-32V304h144c17.67 0 32-14.33 32-32v-32c0-17.67-14.33-32-32-32z\"><\/path><\/svg><\/span>\n\t\t\t\t\t\t\t\t\t\t\t\t\t<\/span>\n\t\t\t\t\t\t\t\t\t\t\t\t<a class=\"elementor-toggle-title\" tabindex=\"0\">Come si definiscono gli endpoint nelle malattie rare?<\/a>\n\t\t\t\t\t<\/div>\n\n\t\t\t\t\t<div id=\"elementor-tab-content-7691\" class=\"elementor-tab-content elementor-clearfix\" data-tab=\"1\" role=\"region\" aria-labelledby=\"elementor-tab-title-7691\"><p>Si parte dalla <strong>storia naturale<\/strong> e dal bisogno clinico. Se un surrogato \u00e8 ben legato al meccanismo, pu\u00f2 anticipare il beneficio; altrimenti servono <strong>outcome funzionali<\/strong> e <strong>PRO<\/strong> validati. La gerarchia degli endpoint va fissata prima, con tempi compatibili alla progressione della malattia.<\/p><\/div>\n\t\t\t\t<\/div>\n\t\t\t\t\t\t\t<div class=\"elementor-toggle-item\">\n\t\t\t\t\t<div id=\"elementor-tab-title-7692\" class=\"elementor-tab-title\" data-tab=\"2\" role=\"button\" aria-controls=\"elementor-tab-content-7692\" aria-expanded=\"false\">\n\t\t\t\t\t\t\t\t\t\t\t\t<span class=\"elementor-toggle-icon elementor-toggle-icon-right\" aria-hidden=\"true\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<span class=\"elementor-toggle-icon-closed\"><svg class=\"e-font-icon-svg e-fas-plus\" viewBox=\"0 0 448 512\" xmlns=\"http:\/\/www.w3.org\/2000\/svg\"><path d=\"M416 208H272V64c0-17.67-14.33-32-32-32h-32c-17.67 0-32 14.33-32 32v144H32c-17.67 0-32 14.33-32 32v32c0 17.67 14.33 32 32 32h144v144c0 17.67 14.33 32 32 32h32c17.67 0 32-14.33 32-32V304h144c17.67 0 32-14.33 32-32v-32c0-17.67-14.33-32-32-32z\"><\/path><\/svg><\/span>\n\t\t\t\t\t\t\t\t<span class=\"elementor-toggle-icon-opened\"><svg class=\"elementor-toggle-icon-opened e-font-icon-svg e-fas-plus\" viewBox=\"0 0 448 512\" xmlns=\"http:\/\/www.w3.org\/2000\/svg\"><path d=\"M416 208H272V64c0-17.67-14.33-32-32-32h-32c-17.67 0-32 14.33-32 32v144H32c-17.67 0-32 14.33-32 32v32c0 17.67 14.33 32 32 32h144v144c0 17.67 14.33 32 32 32h32c17.67 0 32-14.33 32-32V304h144c17.67 0 32-14.33 32-32v-32c0-17.67-14.33-32-32-32z\"><\/path><\/svg><\/span>\n\t\t\t\t\t\t\t\t\t\t\t\t\t<\/span>\n\t\t\t\t\t\t\t\t\t\t\t\t<a class=\"elementor-toggle-title\" tabindex=\"0\">I controlli esterni sono accettabili?<\/a>\n\t\t\t\t\t<\/div>\n\n\t\t\t\t\t<div id=\"elementor-tab-content-7692\" class=\"elementor-tab-content elementor-clearfix\" data-tab=\"2\" role=\"region\" aria-labelledby=\"elementor-tab-title-7692\"><p>Possono esserlo quando randomizzare \u00e8 impraticabile o eticamente dubbio. Ma il registro di controllo deve avere <strong>qualit\u00e0<\/strong>: criteri comparabili, raccolta prospettica o ben documentata, <strong>matching<\/strong> trasparente. \u00c8 comunque una scelta da discutere presto con il regolatore.<\/p><\/div>\n\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t\t<\/div>\n\t\t<\/div>\n\t\t\t\t<div class=\"elementor-column elementor-col-50 elementor-inner-column elementor-element elementor-element-e1b06d0\" data-id=\"e1b06d0\" data-element_type=\"column\" data-e-type=\"column\">\n\t\t\t<div class=\"elementor-widget-wrap elementor-element-populated\">\n\t\t\t\t\t\t<div class=\"elementor-element elementor-element-748645b elementor-widget elementor-widget-toggle\" data-id=\"748645b\" data-element_type=\"widget\" data-e-type=\"widget\" data-widget_type=\"toggle.default\">\n\t\t\t\t<div class=\"elementor-widget-container\">\n\t\t\t\t\t\t\t<div class=\"elementor-toggle\">\n\t\t\t\t\t\t\t<div class=\"elementor-toggle-item\">\n\t\t\t\t\t<div id=\"elementor-tab-title-1221\" class=\"elementor-tab-title\" data-tab=\"1\" role=\"button\" aria-controls=\"elementor-tab-content-1221\" aria-expanded=\"false\">\n\t\t\t\t\t\t\t\t\t\t\t\t<span class=\"elementor-toggle-icon elementor-toggle-icon-right\" aria-hidden=\"true\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<span class=\"elementor-toggle-icon-closed\"><svg class=\"e-font-icon-svg e-fas-plus\" viewBox=\"0 0 448 512\" xmlns=\"http:\/\/www.w3.org\/2000\/svg\"><path d=\"M416 208H272V64c0-17.67-14.33-32-32-32h-32c-17.67 0-32 14.33-32 32v144H32c-17.67 0-32 14.33-32 32v32c0 17.67 14.33 32 32 32h144v144c0 17.67 14.33 32 32 32h32c17.67 0 32-14.33 32-32V304h144c17.67 0 32-14.33 32-32v-32c0-17.67-14.33-32-32-32z\"><\/path><\/svg><\/span>\n\t\t\t\t\t\t\t\t<span class=\"elementor-toggle-icon-opened\"><svg class=\"elementor-toggle-icon-opened e-font-icon-svg e-fas-plus\" viewBox=\"0 0 448 512\" xmlns=\"http:\/\/www.w3.org\/2000\/svg\"><path d=\"M416 208H272V64c0-17.67-14.33-32-32-32h-32c-17.67 0-32 14.33-32 32v144H32c-17.67 0-32 14.33-32 32v32c0 17.67 14.33 32 32 32h144v144c0 17.67 14.33 32 32 32h32c17.67 0 32-14.33 32-32V304h144c17.67 0 32-14.33 32-32v-32c0-17.67-14.33-32-32-32z\"><\/path><\/svg><\/span>\n\t\t\t\t\t\t\t\t\t\t\t\t\t<\/span>\n\t\t\t\t\t\t\t\t\t\t\t\t<a class=\"elementor-toggle-title\" tabindex=\"0\">Che ruolo hanno i registri post-approval?<\/a>\n\t\t\t\t\t<\/div>\n\n\t\t\t\t\t<div id=\"elementor-tab-content-1221\" class=\"elementor-tab-content elementor-clearfix\" data-tab=\"1\" role=\"region\" aria-labelledby=\"elementor-tab-title-1221\"><p>Sono spesso parte dell\u2019<strong>impegno regolatorio<\/strong> nelle rare: consolidano evidenza su efficacia e sicurezza nel lungo periodo, misurano uso di risorse e qualit\u00e0 di vita, e possono informare <strong>HTA<\/strong> e payer nelle revisioni di rimborsabilit\u00e0.<\/p><\/div>\n\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t\t<\/div>\n\t\t<\/div>\n\t\t\t\t\t<\/div>\n\t\t<\/section>\n\t\t\t\t<div class=\"elementor-element elementor-element-f4b816c elementor-align-center elementor-button-info elementor-mobile-align-justify elementor-widget elementor-widget-button\" data-id=\"f4b816c\" data-element_type=\"widget\" data-e-type=\"widget\" data-widget_type=\"button.default\">\n\t\t\t\t<div class=\"elementor-widget-container\">\n\t\t\t\t\t\t\t\t\t<div class=\"elementor-button-wrapper\">\n\t\t\t\t\t<a class=\"elementor-button elementor-button-link elementor-size-md\" href=\"https:\/\/www.technoscience.it\/servizi\/biotecnologie-e-innovazione\/faq\/\">\n\t\t\t\t\t\t<span class=\"elementor-button-content-wrapper\">\n\t\t\t\t\t\t\t\t\t<span class=\"elementor-button-text\">Vedi tutte le domande<\/span>\n\t\t\t\t\t<\/span>\n\t\t\t\t\t<\/a>\n\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t\t<\/div>\n\t\t<\/div>\n\t\t\t\t\t<\/div>\n\t\t<\/section>\n\t\t\t\t<\/div>\n\t\t","protected":false},"excerpt":{"rendered":"<p>Le malattie rare obbligano a ripensare il modo in cui si fa evidenza. I numeri sono piccoli, l\u2019eterogeneit\u00e0 \u00e8 alta, la storia naturale \u00e8 spesso incompleta. Eppure proprio qui si [&hellip;]<\/p>\n","protected":false},"author":1,"featured_media":3780,"parent":3685,"menu_order":0,"comment_status":"closed","ping_status":"closed","template":"","meta":{"_price":"","_stock":"","_tribe_ticket_header":"","_tribe_default_ticket_provider":"","_tribe_ticket_capacity":"0","_ticket_start_date":"","_ticket_end_date":"","_tribe_ticket_show_description":"","_tribe_ticket_show_not_going":false,"_tribe_ticket_use_global_stock":"","_tribe_ticket_global_stock_level":"","_global_stock_mode":"","_global_stock_cap":"","_tribe_rsvp_for_event":"","_tribe_ticket_going_count":"","_tribe_ticket_not_going_count":"","_tribe_tickets_list":"[]","_tribe_ticket_has_attendee_info_fields":false,"footnotes":"","_tec_slr_enabled":"","_tec_slr_layout":""},"class_list":["post-3766","page","type-page","status-publish","has-post-thumbnail","hentry"],"yoast_head":"<!-- This site is optimized with the Yoast SEO plugin v28.5 - https:\/\/yoast.com\/product\/yoast-seo-wordpress\/ -->\n<title>Rare-Disease Studies: Models, Endpoints and | Technoscience<\/title>\n<meta name=\"description\" content=\"From natural history studies to registries, from adaptive designs to PROs: how we design rare disease studies with meaningful endpoints and genuine clinical.\" \/>\n<meta name=\"robots\" content=\"index, follow, max-snippet:-1, max-image-preview:large, max-video-preview:-1\" \/>\n<link rel=\"canonical\" href=\"https:\/\/www.technoscience.it\/en\/servizi\/biotecnologie-e-innovazione\/biopharma-terapie\/studi-su-malattie-rare-modelli-endpoint-e-sostenibilita-clinica\/\" \/>\n<meta property=\"og:locale\" content=\"en_GB\" \/>\n<meta property=\"og:type\" content=\"article\" \/>\n<meta property=\"og:title\" content=\"Rare-Disease Studies: Models, Endpoints and | Technoscience\" \/>\n<meta property=\"og:description\" content=\"From natural history studies to registries, from adaptive designs to PROs: how we design rare disease studies with meaningful endpoints and genuine clinical.\" \/>\n<meta property=\"og:url\" content=\"https:\/\/www.technoscience.it\/en\/servizi\/biotecnologie-e-innovazione\/biopharma-terapie\/studi-su-malattie-rare-modelli-endpoint-e-sostenibilita-clinica\/\" \/>\n<meta property=\"og:site_name\" content=\"Technoscience\" \/>\n<meta property=\"article:modified_time\" content=\"2025-11-24T12:07:40+00:00\" \/>\n<meta property=\"og:image\" content=\"https:\/\/www.technoscience.it\/wp-content\/uploads\/2025\/11\/studi-clinici-su-malattie-rare.jpg\" \/>\n\t<meta property=\"og:image:width\" content=\"1920\" \/>\n\t<meta property=\"og:image:height\" content=\"1920\" \/>\n\t<meta property=\"og:image:type\" content=\"image\/jpeg\" \/>\n<meta name=\"twitter:card\" content=\"summary_large_image\" \/>\n<meta name=\"twitter:title\" content=\"Rare-Disease Studies: Models, Endpoints and | Technoscience\" \/>\n<meta name=\"twitter:description\" content=\"Explore Technoscience programmes, services and partnerships in biotechnology, applied research, innovation and technology transfer.\" \/>\n<meta name=\"twitter:label1\" content=\"Estimated reading time\" \/>\n\t<meta name=\"twitter:data1\" content=\"6 minutes\" \/>\n<script type=\"application\/ld+json\" class=\"yoast-schema-graph\">{\"@context\":\"https:\\\/\\\/schema.org\",\"@graph\":[{\"@type\":\"WebPage\",\"@id\":\"https:\\\/\\\/www.technoscience.it\\\/servizi\\\/biotecnologie-e-innovazione\\\/biopharma-terapie\\\/studi-su-malattie-rare-modelli-endpoint-e-sostenibilita-clinica\\\/\",\"url\":\"https:\\\/\\\/www.technoscience.it\\\/servizi\\\/biotecnologie-e-innovazione\\\/biopharma-terapie\\\/studi-su-malattie-rare-modelli-endpoint-e-sostenibilita-clinica\\\/\",\"name\":\"Malattie rare: studi, endpoint e sostenibilit\u00e0 - Technoscience\",\"isPartOf\":{\"@id\":\"https:\\\/\\\/www.technoscience.it\\\/#website\"},\"primaryImageOfPage\":{\"@id\":\"https:\\\/\\\/www.technoscience.it\\\/servizi\\\/biotecnologie-e-innovazione\\\/biopharma-terapie\\\/studi-su-malattie-rare-modelli-endpoint-e-sostenibilita-clinica\\\/#primaryimage\"},\"image\":{\"@id\":\"https:\\\/\\\/www.technoscience.it\\\/servizi\\\/biotecnologie-e-innovazione\\\/biopharma-terapie\\\/studi-su-malattie-rare-modelli-endpoint-e-sostenibilita-clinica\\\/#primaryimage\"},\"thumbnailUrl\":\"https:\\\/\\\/www.technoscience.it\\\/wp-content\\\/uploads\\\/2025\\\/11\\\/studi-clinici-su-malattie-rare.jpg\",\"datePublished\":\"2025-11-24T11:54:19+00:00\",\"dateModified\":\"2025-11-24T12:07:40+00:00\",\"description\":\"Dalla storia naturale ai registri, dai disegni adattativi ai PRO: come impostiamo studi su malattie rare con endpoint significativi e sostenibilit\u00e0 clinica reale.\",\"breadcrumb\":{\"@id\":\"https:\\\/\\\/www.technoscience.it\\\/servizi\\\/biotecnologie-e-innovazione\\\/biopharma-terapie\\\/studi-su-malattie-rare-modelli-endpoint-e-sostenibilita-clinica\\\/#breadcrumb\"},\"inLanguage\":\"en-GB\",\"potentialAction\":[{\"@type\":\"ReadAction\",\"target\":[\"https:\\\/\\\/www.technoscience.it\\\/servizi\\\/biotecnologie-e-innovazione\\\/biopharma-terapie\\\/studi-su-malattie-rare-modelli-endpoint-e-sostenibilita-clinica\\\/\"]}]},{\"@type\":\"ImageObject\",\"inLanguage\":\"en-GB\",\"@id\":\"https:\\\/\\\/www.technoscience.it\\\/servizi\\\/biotecnologie-e-innovazione\\\/biopharma-terapie\\\/studi-su-malattie-rare-modelli-endpoint-e-sostenibilita-clinica\\\/#primaryimage\",\"url\":\"https:\\\/\\\/www.technoscience.it\\\/wp-content\\\/uploads\\\/2025\\\/11\\\/studi-clinici-su-malattie-rare.jpg\",\"contentUrl\":\"https:\\\/\\\/www.technoscience.it\\\/wp-content\\\/uploads\\\/2025\\\/11\\\/studi-clinici-su-malattie-rare.jpg\",\"width\":1920,\"height\":1920,\"caption\":\"Multidisciplinary team designing rare-disease clinical studies, reviewing study models, endpoints, patient registries and real-world data.\"},{\"@type\":\"BreadcrumbList\",\"@id\":\"https:\\\/\\\/www.technoscience.it\\\/servizi\\\/biotecnologie-e-innovazione\\\/biopharma-terapie\\\/studi-su-malattie-rare-modelli-endpoint-e-sostenibilita-clinica\\\/#breadcrumb\",\"itemListElement\":[{\"@type\":\"ListItem\",\"position\":1,\"name\":\"Home\",\"item\":\"https:\\\/\\\/www.technoscience.it\\\/\"},{\"@type\":\"ListItem\",\"position\":2,\"name\":\"Servizi\",\"item\":\"https:\\\/\\\/www.technoscience.it\\\/servizi\\\/\"},{\"@type\":\"ListItem\",\"position\":3,\"name\":\"Biotecnologie e Innovazione\",\"item\":\"https:\\\/\\\/www.technoscience.it\\\/servizi\\\/biotecnologie-e-innovazione\\\/\"},{\"@type\":\"ListItem\",\"position\":4,\"name\":\"BioPharma &#038; Terapie\",\"item\":\"https:\\\/\\\/www.technoscience.it\\\/servizi\\\/biotecnologie-e-innovazione\\\/biopharma-terapie\\\/\"},{\"@type\":\"ListItem\",\"position\":5,\"name\":\"Studi su malattie rare: modelli, endpoint e sostenibilit\u00e0 clinica\"}]},{\"@type\":\"WebSite\",\"@id\":\"https:\\\/\\\/www.technoscience.it\\\/#website\",\"url\":\"https:\\\/\\\/www.technoscience.it\\\/\",\"name\":\"Technoscience\",\"description\":\"Pontine Science and Technology Park for biotechnology, applied research, innovation and technology transfer.\",\"publisher\":{\"@id\":\"https:\\\/\\\/www.technoscience.it\\\/#organization\"},\"alternateName\":\"Parco Scientifico e Tecnologico Pontino\",\"potentialAction\":[{\"@type\":\"SearchAction\",\"target\":{\"@type\":\"EntryPoint\",\"urlTemplate\":\"https:\\\/\\\/www.technoscience.it\\\/?s={search_term_string}\"},\"query-input\":{\"@type\":\"PropertyValueSpecification\",\"valueRequired\":true,\"valueName\":\"search_term_string\"}}],\"inLanguage\":\"en-GB\"},{\"@type\":\"Organization\",\"@id\":\"https:\\\/\\\/www.technoscience.it\\\/#organization\",\"name\":\"Technoscience | Consorzio Parco Scientifico e Tecnologico Pontino\",\"alternateName\":\"Technoscience\",\"url\":\"https:\\\/\\\/www.technoscience.it\\\/\",\"logo\":{\"@type\":\"ImageObject\",\"inLanguage\":\"en-GB\",\"@id\":\"https:\\\/\\\/www.technoscience.it\\\/#\\\/schema\\\/logo\\\/image\\\/\",\"url\":\"https:\\\/\\\/www.technoscience.it\\\/wp-content\\\/uploads\\\/2024\\\/02\\\/Technoscience-logooriz-w.png\",\"contentUrl\":\"https:\\\/\\\/www.technoscience.it\\\/wp-content\\\/uploads\\\/2024\\\/02\\\/Technoscience-logooriz-w.png\",\"width\":2042,\"height\":336,\"caption\":\"Technoscience | Consorzio Parco Scientifico e Tecnologico Pontino\"},\"image\":{\"@id\":\"https:\\\/\\\/www.technoscience.it\\\/#\\\/schema\\\/logo\\\/image\\\/\"},\"legalName\":\"Consorzio Parco Scientifico e Tecnologico Pontino\",\"description\":\"Science and technology park specialising in biotechnology, applied research, technology transfer and innovation for industry, universities and public institutions.\",\"email\":\"segreteria@technoscience.it\",\"taxID\":\"02850910593\",\"address\":{\"@type\":\"PostalAddress\",\"streetAddress\":\"Via Enrico Toti, 15\",\"postalCode\":\"04100\",\"addressLocality\":\"Latina\",\"addressRegion\":\"LT\",\"addressCountry\":\"IT\"}}]}<\/script>\n<!-- \/ Yoast SEO plugin. -->","yoast_head_json":{"title":"Rare-Disease Studies: Models, Endpoints and | Technoscience","description":"From natural history studies to registries, from adaptive designs to PROs: how we design rare disease studies with meaningful endpoints and genuine clinical.","robots":{"index":"index","follow":"follow","max-snippet":"max-snippet:-1","max-image-preview":"max-image-preview:large","max-video-preview":"max-video-preview:-1"},"canonical":"https:\/\/www.technoscience.it\/en\/servizi\/biotecnologie-e-innovazione\/biopharma-terapie\/studi-su-malattie-rare-modelli-endpoint-e-sostenibilita-clinica\/","og_locale":"en_GB","og_type":"article","og_title":"Rare-Disease Studies: Models, Endpoints and | Technoscience","og_description":"From natural history studies to registries, from adaptive designs to PROs: how we design rare disease studies with meaningful endpoints and genuine clinical.","og_url":"https:\/\/www.technoscience.it\/en\/servizi\/biotecnologie-e-innovazione\/biopharma-terapie\/studi-su-malattie-rare-modelli-endpoint-e-sostenibilita-clinica\/","og_site_name":"Technoscience","article_modified_time":"2025-11-24T12:07:40+00:00","og_image":[{"width":1920,"height":1920,"url":"https:\/\/www.technoscience.it\/wp-content\/uploads\/2025\/11\/studi-clinici-su-malattie-rare.jpg","type":"image\/jpeg"}],"twitter_card":"summary_large_image","twitter_title":"Rare-Disease Studies: Models, Endpoints and | Technoscience","twitter_description":"Explore Technoscience programmes, services and partnerships in biotechnology, applied research, innovation and technology transfer.","twitter_misc":{"Estimated reading time":"6 minutes"},"schema":{"@context":"https:\/\/schema.org","@graph":[{"@type":"WebPage","@id":"https:\/\/www.technoscience.it\/servizi\/biotecnologie-e-innovazione\/biopharma-terapie\/studi-su-malattie-rare-modelli-endpoint-e-sostenibilita-clinica\/","url":"https:\/\/www.technoscience.it\/servizi\/biotecnologie-e-innovazione\/biopharma-terapie\/studi-su-malattie-rare-modelli-endpoint-e-sostenibilita-clinica\/","name":"Rare diseases: studies, endpoints and sustainability - Technoscience","isPartOf":{"@id":"https:\/\/www.technoscience.it\/#website"},"primaryImageOfPage":{"@id":"https:\/\/www.technoscience.it\/servizi\/biotecnologie-e-innovazione\/biopharma-terapie\/studi-su-malattie-rare-modelli-endpoint-e-sostenibilita-clinica\/#primaryimage"},"image":{"@id":"https:\/\/www.technoscience.it\/servizi\/biotecnologie-e-innovazione\/biopharma-terapie\/studi-su-malattie-rare-modelli-endpoint-e-sostenibilita-clinica\/#primaryimage"},"thumbnailUrl":"https:\/\/www.technoscience.it\/wp-content\/uploads\/2025\/11\/studi-clinici-su-malattie-rare.jpg","datePublished":"2025-11-24T11:54:19+00:00","dateModified":"2025-11-24T12:07:40+00:00","description":"From natural history studies to registries, from adaptive designs to PROs: how we design rare disease studies with meaningful endpoints and genuine clinical sustainability.","breadcrumb":{"@id":"https:\/\/www.technoscience.it\/servizi\/biotecnologie-e-innovazione\/biopharma-terapie\/studi-su-malattie-rare-modelli-endpoint-e-sostenibilita-clinica\/#breadcrumb"},"inLanguage":"en-GB","potentialAction":[{"@type":"ReadAction","target":["https:\/\/www.technoscience.it\/servizi\/biotecnologie-e-innovazione\/biopharma-terapie\/studi-su-malattie-rare-modelli-endpoint-e-sostenibilita-clinica\/"]}]},{"@type":"ImageObject","inLanguage":"en-GB","@id":"https:\/\/www.technoscience.it\/servizi\/biotecnologie-e-innovazione\/biopharma-terapie\/studi-su-malattie-rare-modelli-endpoint-e-sostenibilita-clinica\/#primaryimage","url":"https:\/\/www.technoscience.it\/wp-content\/uploads\/2025\/11\/studi-clinici-su-malattie-rare.jpg","contentUrl":"https:\/\/www.technoscience.it\/wp-content\/uploads\/2025\/11\/studi-clinici-su-malattie-rare.jpg","width":1920,"height":1920,"caption":"Multidisciplinary team designing rare-disease clinical studies, reviewing study models, endpoints, patient registries and real-world data."},{"@type":"BreadcrumbList","@id":"https:\/\/www.technoscience.it\/servizi\/biotecnologie-e-innovazione\/biopharma-terapie\/studi-su-malattie-rare-modelli-endpoint-e-sostenibilita-clinica\/#breadcrumb","itemListElement":[{"@type":"ListItem","position":1,"name":"Home","item":"https:\/\/www.technoscience.it\/"},{"@type":"ListItem","position":2,"name":"Servizi","item":"https:\/\/www.technoscience.it\/servizi\/"},{"@type":"ListItem","position":3,"name":"Biotecnologie e Innovazione","item":"https:\/\/www.technoscience.it\/servizi\/biotecnologie-e-innovazione\/"},{"@type":"ListItem","position":4,"name":"BioPharma &#038; Terapie","item":"https:\/\/www.technoscience.it\/servizi\/biotecnologie-e-innovazione\/biopharma-terapie\/"},{"@type":"ListItem","position":5,"name":"Studi su malattie rare: modelli, endpoint e sostenibilit\u00e0 clinica"}]},{"@type":"WebSite","@id":"https:\/\/www.technoscience.it\/#website","url":"https:\/\/www.technoscience.it\/","name":"Technoscience","description":"Pontine Science and Technology Park for biotechnology, applied research, innovation and technology transfer.","publisher":{"@id":"https:\/\/www.technoscience.it\/#organization"},"alternateName":"Parco Scientifico e Tecnologico Pontino","potentialAction":[{"@type":"SearchAction","target":{"@type":"EntryPoint","urlTemplate":"https:\/\/www.technoscience.it\/?s={search_term_string}"},"query-input":{"@type":"PropertyValueSpecification","valueRequired":true,"valueName":"search_term_string"}}],"inLanguage":"en-GB"},{"@type":"Organization","@id":"https:\/\/www.technoscience.it\/#organization","name":"Technoscience | Pontine Science and Technology Park Consortium","alternateName":"Technoscience","url":"https:\/\/www.technoscience.it\/","logo":{"@type":"ImageObject","inLanguage":"en-GB","@id":"https:\/\/www.technoscience.it\/#\/schema\/logo\/image\/","url":"https:\/\/www.technoscience.it\/wp-content\/uploads\/2024\/02\/Technoscience-logooriz-w.png","contentUrl":"https:\/\/www.technoscience.it\/wp-content\/uploads\/2024\/02\/Technoscience-logooriz-w.png","width":2042,"height":336,"caption":"Technoscience | Consorzio Parco Scientifico e Tecnologico Pontino"},"image":{"@id":"https:\/\/www.technoscience.it\/#\/schema\/logo\/image\/"},"legalName":"Consorzio Parco Scientifico e Tecnologico Pontino","description":"Science and technology park specialising in biotechnology, applied research, technology transfer and innovation for industry, universities and public institutions.","email":"segreteria@technoscience.it","taxID":"02850910593","address":{"@type":"PostalAddress","streetAddress":"Via Enrico Toti, 15","postalCode":"04100","addressLocality":"Latina","addressRegion":"LT","addressCountry":"IT"}}]}},"ticketed":false,"_links":{"self":[{"href":"https:\/\/www.technoscience.it\/en\/wp-json\/wp\/v2\/pages\/3766","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/www.technoscience.it\/en\/wp-json\/wp\/v2\/pages"}],"about":[{"href":"https:\/\/www.technoscience.it\/en\/wp-json\/wp\/v2\/types\/page"}],"author":[{"embeddable":true,"href":"https:\/\/www.technoscience.it\/en\/wp-json\/wp\/v2\/users\/1"}],"replies":[{"embeddable":true,"href":"https:\/\/www.technoscience.it\/en\/wp-json\/wp\/v2\/comments?post=3766"}],"version-history":[{"count":0,"href":"https:\/\/www.technoscience.it\/en\/wp-json\/wp\/v2\/pages\/3766\/revisions"}],"up":[{"embeddable":true,"href":"https:\/\/www.technoscience.it\/en\/wp-json\/wp\/v2\/pages\/3685"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/www.technoscience.it\/en\/wp-json\/wp\/v2\/media\/3780"}],"wp:attachment":[{"href":"https:\/\/www.technoscience.it\/en\/wp-json\/wp\/v2\/media?parent=3766"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}