Biopharmaceutical Pipelines: From Proof of Concept to TRL 7
A biopharmaceutical is not born on the day it is formulated: it begins to exist when the biological hypothesis proves able to withstand its first encounters with reality. The proof-of-concept is the point at which an idea stops being a promise and becomes a measurable result; from there begins a trajectory that, if pursued methodically, leads to the TRL7, meaning demonstration in an operational environment with processes, quality and safety under control. In between lies patient work: CMC that does not fail at scale, study designs that answer real clinical questions, statistical controls that prevent illusions, and a mature relationship with EMA/FDA. This page explains how we set that trajectory and which milestone cannot be missing and which KPI we use to stay on track.
From bench to the first “yes”: the proof of concept that matters
A PoC credible clarifies three things. First: mechanism of action and biological rationale are not a story, but a coherent chain of evidence (in vitro, ex vivo, appropriate preclinical models). Second: there is a endpoint measurable that moves in the expected direction when the intervention is present. Third: the safety profile initial is compatible with continuation. This “yes” is not definitive, but it is enough to turn a project into programme: defines hypotheses, resources, timelines and risks in a language that investors, clinicians and quality teams can share.
CMC: quality that does not fall apart as you scale
The Chemistry, Manufacturing and Controls is the quiet heart of biotech. A process that works at bench scale can fail at the first scale-up step if purity, yield and stability have not been designed to scale. This is where the traceability of batches, consistency between process and product, the strategy for analytical comparability when plants or raw materials change. The question is simple and unforgiving: will what you promise today still be repeat in the same way tomorrow, in six months, at another site? If the answer is uncertain, the pipeline will stall later, when it costs ten times as much.
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The study design that answers the question, not the one that pleases
A good design starts with the right question. What is the plausible clinical effect? In which population is it most clearly observed? What is theendpoint that captures that effect without lending itself to arbitrary interpretation? At an early stage, prioritise endpoints of biology (target-engagement markers, robust surrogates), at a later stage you measure clinical outcomes that matter to patients and regulators. Every statistical choice—randomisation, stratification, power—must be declared a priori; changes of direction are possible, but only if justified and traceable. The difference between a signal and a mirage often lies here.
The milestones you cannot skip
After PoC and regulatory preclinical development then come the human steps. Phase 1: safety, pharmacokinetics, first characterisation of the dose-response profile. Phase 2: signal confirmation in more clearly defined populations, dose and regimen selection. Phase 3: definitive verification of clinical endpoints agreed with regulators. Between one phase and the next there is no vacuum: there are Go/No-Go linked to specific KPIs (safety, consistency of outcomes, operational feasibility, data integrity). A healthy pipeline does not simply “keep going”: decides.
KPIs to avoid fooling ourselves
We use indicators that describe the substance, not just progress against the Gantt chart. In CMC: yield, purity, stability real, rate of deviation and batch-release times. In clinical development: recruitment and retention vs plan, protocol adherence, data quality (queries, missingness), consistency of effect across pre-specified subgroups, safety signals and comparison with the standard of care. In the regulatory pathway: timeliness of interactions with EMA/FDA, clarity of the scientific advice, status of the CMC packages and of dossier.
Towards TRL7: demonstrating that it can genuinely be done
Reaching TRL7 means demonstrating that the product does not exist only within the confines of the study. Production is repeatable at pilot/industrial scale, the supply chain is reliable, the quality holds up, the safety is properly managed and the clinical value is documented with sufficient evidence to engage with payer and HTA. This is the point at which the biopharmaceutical can be assessed in operational environments realistic, including the complications of real life: logistics, administration timing, interactions with other therapies, adherence.
How we work together
When a project is proposed to us in the BioPharma, we ask for a briefing note essential: biological hypothesis, CMC status, study design and status, main risk, available data. Within a few days, we provide a fit check with a map of the gap, milestones and a pathway TRL credible. If there is substance, we coordinate the right stakeholders (clinical, quality, regulatory, finance) and define precisely what to measure and when. If the conditions are not there, we say so immediately: an honest no is better than a yes that wastes resources.
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If you are designing a study that requires samples and data of verified quality, request a fit check: in a short call, we align the clinical or industrial question, sample availability, ethical requirements and quality standards; if the scope is sound, we plan a pathway from responsible access to validation without wasting time.
Frequently Asked Questions
The questions you ask us most often
The Phase 1 assesses safety and the dose-response profile (often in healthy volunteers or small groups of patients). The Phase 2 look for confirmation of effectiveness in selected populations, defines dose and regimen and prepares endpoints for the next phase. The Phase 3 verify definitively efficacy and safety on larger samples and with clinical endpoints shared with regulators.
It is thecompassionate use of an investigational medicinal product outside clinical trials, in patients with no valid therapeutic alternatives. It is governed by specific rules and does not replace trials: it addresses exceptional situations, with risk-benefit assessment and complete traceability.
The Orphan Drug status applies to therapies for rare diseases with an unmet clinical need. It may include incentives through market exclusivity, reduced fees and dedicated regulatory support: it shortens timelines and makes development sustainable where the numbers would otherwise be discouraging.