Clinical trials offer patients access to potentially ground-breaking treatments or therapies that are not yet available to the general public. They play a vital role in advancing medical knowledge, improving patient care, and ultimately, saving lives.

Europe has rich scientific traditions and a strong academic infrastructure, earning it a reputation as a pharmaceutical innovation powerhouse. In the past, many breakthrough treatments for cancer, cardiovascular diseases, infectious diseases, and neurological conditions among others were researched, developed and introduced in Europe. However, this proud legacy is fading. Just 25 years ago, every second new treatment originated in Europe. Today, that number has dwindled to less than one in every five.[1]

While the number of European clinical trials might not be declining, they are not experiencing the same level of growth as seen in some other regions[2], where there is increased investment in clinical research ecosystems.

The relative decline in European clinical trials has led to:

• Fewer opportunities for European patients to access cutting-edge treatments.
• The characteristics of European patient populations being less likely to be prioritised in trials.
• Reduced experience for European healthcare systems of novel treatments prior to approval.
• Steady erosion of the skills base and the competitiveness of the clinical trials investigator community in Europe.

EFPIA, working closely with other stakeholders, aims to reverse this trend. Whilst it is encouraging to see all regions of the world contributing to better healthcare, our vision is clear. We are committed to advancing faster, smarter and more patient-centric trials, and increasing the global share of clinical trials in Europe.

Together, we can realise this vision and ensure that Europe regains its position at the forefront of medical progress.

Focusing on what really matters

We have witnessed several attempts in recent years to simplify and harmonise the regulatory environment for clinical trials in Europe. These include the Clinical Trials Regulation (CTR) and the introduction of the single submission portal, the Clinical Trial Information System (CTIS).

Despite these positive efforts, there are still opportunities for further improvements to address remaining complexities, rigidity, and a lack of pragmatism in the submission and approval of clinical trial applications. For example, in some countries, the timelines for regulatory and ethical approvals are unacceptably long. Although the main objective of the CTR is to harmonise the framework for conducting clinical trials across Europe, conflicting application requirements persist among countries. The regulation aims to streamline and simplify the process of conducting clinical trials while ensuring the safety and rights of participants. It is crucial that we fully utilise its potential.[3]

We need a regulatory system which ensures that safe and effective treatments reach patients as quickly as possible. Mature, well-functioning and interoperable systems must support an iterative, integrated approach that involves seamless engagement with multiple stakeholders. Furthermore, interactions between regulators and sponsors in the latter stages of the review process can help clarify any outstanding issues. However, this is often not possible, depriving both parties of a valuable opportunity to engage views and expertise. Additionally, safeguards need to be proportionate to the risks that they are intended to mitigate, and procedures should be designed with sufficient flexibility to ensure rules can be applied pragmatically. To achieve this, having sufficiently skilled resources is imperative.

However, the assessment by regulatory and ethics committees is only one part of the process. If we truly want to move the needle, we need to address the entire process. Simple changes, like standard contractual clauses for agreements between clinical trial sponsors and investigational sites, or harmonised rules governing advanced therapies, can lead to shorter timelines. Achieving reforms like these requires political will and the joint effort of all key stakeholders.[4]

Prepared for the opportunities of today – and tomorrow 

We believe that now is the time to ensure that clinical research respects, supports and values patients’ participation. This means embracing diverse evidence sources as well as novel technologies and approaches. It is time to modernise clinical trials by adopting decentralised trial elements and fully harnessing the potential of digital health technologies and advanced data analytics tools.

As we look toward 2030 and beyond, it’s essential that we equip ourselves with the tools and knowledge needed to thrive in an ever evolving and globally competitive clinical environment. An open-minded, bold, and collaborative approach is critical. Consequently, there's a pressing need for a platform that facilitates open, timely and continuous multi-stakeholder dialogue. It is essential to build on existing initiatives like the ACT EU Multistakeholder platform.

Connectivity between future methods/technologies and fit-for-purpose legislative environment is crucial for seamless integration. This includes ensuring the compatibility of regulations such as the Clinical Trials Regulation (CTR), In Vitro Diagnostic Regulation (IVDR), and Medical Device Regulation (MDR) and the European Health Data Space (EHDS) legislation. Additionally, maximising the use of data, including the secondary use of data, will be essential for driving insights, innovation, and improved patient outcomes in the years to come.

By embracing these principles of readiness and adaptability, we can confidently prepare ourselves for the challenges and opportunities that lie ahead in healthcare.

Patients at the heart of research

In our quest for more patient-centric clinical trials, we are committed to collaborating closely with patients and prioritising their needs. We want to gain a deeper understanding of their challenges and address these accordingly.

The first, most important step is to ensure everyone can easily access information about available clinical trials. Despite technological progress, patients, carers, and healthcare professionals still struggle to find current, trustworthy, and easily accessible clinical trial information in one place. And we should not forget that raising awareness on the importance of clinical trials is a shared responsibility.

We need to cut through unnecessary red tape and enable cross-border access to clinical trials. Particularly in smaller European countries and for rare diseases, patients are often unable to participate in trials which are available in neighbouring countries. By removing administrative hurdles we can facilitate access for patients, broaden the pool of participants, and ultimately have more representative results more quickly. This is especially important for patients with rare diseases who need to access trials through specialist centres.

Lastly, we need to ensure that patient experience data (PED) captured in clinical trials is taken into consideration when evaluating the benefit-risk of a product and included in the regulatory assessment. It is important that PED evaluation is made transparent in the product assessment report and included in the labelling when relevant.

Better for patients, better for Europe

The care and needs of patients are what drives our industry. Medical innovations can help patients and their families to reclaim their lives. This can positively impact individuals, communities, and Europe as a whole.

It is crucial to break down existing barriers between systems, countries, legislative frameworks, and stakeholders, move away from unnecessary complexities, and fully exploit novel technologies and approaches.

Therefore, we propose the following five steps towards faster, smarter, and patient centric trials in Europe:

• Adequate resources at both the European and national levels across Europe to enable more interactions between regulators and innovators.
• Platforms for continued scientific multistakeholder, multidisciplinary and cross-sector dialogue.
• More opportunities for flexible interactions, especially towards the end of the clinical trials review process to avoid unnecessary rejections or unfeasible conditional approvals.
• Streamlined approval processes across Europe supported by interoperable systems and procedures.
• Mechanisms to facilitate patients’ and physicians’ awareness and access to trials within and across borders. 

If we want to secure competitive access to clinical trials and future medical innovations for European patients, we must act decisively and immediately.

[1] https://www.efpia.eu/media/676753/cra-efpia-investment-location-final-report.pdf  

[2] https://pharmaboardroom.com/interviews/peter-arlett-head-of-data-analytics-and-methods-european-medicines-agency-ema/

[3] https://research-and-innovation.ec.europa.eu/document/download/47554adc-dffc-411b-8cd6-b52417514cb3_en

[4] https://www.consilium.europa.eu/media/ny3j24sm/much-more-than-a-market-report-by-enrico-letta.pdf