Clinical trials & start-ups: entering the clinical trial phase is a decisive milestone for any innovative health company. For a start-up developing a drug, a targeted immunotherapy, or a connected medical device, this transition to human evaluation is both an opportunity and a challenge. A partnership with a Contract Research Organization (CRO) quickly becomes essential to secure and accelerate the process. Understanding this role is critical for young companies aiming to transform a discovery into a therapeutic solution available to patients.
Clinical trials & start-ups: CROs represent a key support
CROs support health companies throughout the clinical development process. For start-ups, often limited in human and financial resources, this external expertise allows them to:
- design a protocol in compliance with regulations,
- obtain the required ethical and legal approvals,
- recruit investigator sites and patients,
- ensure monitoring and data quality,
- draft the final reports needed for Marketing Authorization (MA) or CE marking.
In other words, the CRO acts as the operational arm of the start-up. It provides access to its investigator network, regulatory expertise, and data management tools. This strategic partnership is at the core of the success of clinical trials for start-ups.
The key stages of drug development
Before a clinical trial can even be considered, a drug candidate must go through the preclinical phase. This phase includes in vitro and in vivo tests to evaluate pharmacology, toxicity, and pharmacokinetics. These data are essential to estimate the starting dose in humans. Despite advances in cellular models and computer simulations, animal experimentation remains an indispensable step to ensure future patient safety.
The clinical phase then takes place in several stages:
- Phase I: evaluate tolerance and safety in healthy volunteers.
- Phase II: test efficacy and adjust dosing in patients.
- Phase III: confirm benefit/risk on larger cohorts.
- Phase IV: post-marketing follow-up in real life.
Each stage requires rigor, funding, and expertise, highlighting the importance of a close collaboration between start-ups and CROs.
The specific case of medical devices
For medical devices, the regulatory path is slightly different. Obtaining CE marking relies on demonstrating safety, performance, and compliance with European regulations 2017/745 (MDR) and 2017/746 (IVDR). Clinical studies are often required but remain less standardized than those for drugs. In France, the ANSM supervises compliance and monitoring, while notified bodies such as GMED or AFNOR issue CE certification.
For a medtech start-up, collaboration with a specialized CRO facilitates the design of the necessary clinical studies and the preparation of the technical dossier. Here again, clinical trials for start-ups benefit from the know-how of teams experienced in regulatory requirements.
European regulation: a rapidly evolving framework
Since January 2022, European regulation EU 536/2014 has governed clinical trials of medicinal products. Its aim: simplify and harmonize procedures across Member States while increasing transparency thanks to the Clinical Trial Information System (CTIS). For start-ups, this regulation represents an opportunity: a single submission can now cover multiple European countries, facilitating multinational trial launches.
However, simplification does not erase complexity. This is why the involvement of a CRO, used to working with both national and European authorities, remains a decisive advantage.
Challenges and perspectives for start-ups
Despite the opportunities created by therapeutic innovation, start-ups face several obstacles:
- high costs of clinical phases,
- difficulties in recruiting patients,
- strict regulatory requirements,
- the need for medico-economic evidence to secure reimbursement later from bodies such as HAS and CEPS in France.
In this context, the start-up/CRO collaboration is not just a technical partnership. It becomes a true strategic lever for turning innovation into a marketable health product.
Conclusion: a winning duo for innovation
Clinical trials for start-ups are a crucial but challenging step. By relying on CROs, young companies can concentrate their resources on research and innovation, while entrusting operational and regulatory management to experts. This collaboration model not only accelerates market access but also ensures patient safety and the quality of generated data.
At a time when Europe is reinforcing its attractiveness in clinical research, the success of start-ups will depend on their ability to integrate this strategic alliance with CROs. A synergy that, beyond constraints, paves the way for new therapies and medical devices serving patients.
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