Make the regulatory system fit for the future of medical innovation
The devastating human cost of the COVID-19 crisis drove everyone involved in bringing new diagnostics, vaccines and treatments to patients to adopt a mind-set of simplifying and accelerating procedures. The “new normal” in regulatory terms should embrace this attitude, aiming at decreasing complexity while ensuring the quality, safety and efficacy of the medicines for patients who need them.
Today’s regulatory system plays a vital role in ensuring that new treatments for patients are safe and effective. And in an innovative field like ours, things move fast. We must ensure that our regulatory system keeps pace with advances in science and technology and, at the same time, ensure that Europe has an agile regulatory infrastructure in place to face unexpected public health threats.
It’s important to highlight that the European Medicines Agency (EMA) and national regulators have transformed the lives of millions of people in Europe living with diseases like cancer and diabetes, HIV and hepatitis C. Since its foundation in 1995, the EMA alone has recommended authorisation of over 1,400 medicines. It is within this environment that Europe’s innovative pharmaceutical sector has developed – investing heavily in R&D and contributing to economic growth.
There are 60,000 clinical trials ongoing in Europe and the pipeline of next-generation biotherapeutics – like cell and gene therapies – more than doubled in the years 2015-2018. There is much to celebrate.
However, Europe is facing increasing competition from other regions in the race for medical excellence. This disparity in innovation becomes very apparent when looking at the state of global competitiveness in next generation cell and gene therapies, such as CAR-T. According to a 2018 article in The Scientist magazine, China is now engaged in more clinical trials than the rest of the world combined. Europe accounts for only 30% of the US total of ongoing clinical CAR-T trials and for 16% of China’s current research activity.
That’s why we worked with our members to look at how the current European regulatory system can evolve and the Regulatory Road to Innovation is the result of this endeavour. Again, it’s an evolution rather than a revolution. It looks into proposals to make the European regulatory system more agile and competitive without necessarily having to make changes to existing legislation. We narrowed it down to four key areas: encouraging the use of new types of clinical trials; accelerating the use and acceptance of real-world evidence in regulatory decision making; strengthening dialogue between industry and regulators throughout the development process; and simplifying how medicines and medical devices, so-called combination products, are regulated in a more coherent and streamlined way.
The potential of innovative clinical trials designs
In normal times, innovative clinical trial designs promise to make research more efficient. These trials are conceived to allow changes or updates to the study protocol after the trial has begun, without compromising its integrity. Adaptive trials, and more complex trial designs such as master protocol trials, are among the most significant innovations in the clinical research in recent years.
Innovative trial designs revealed their added value during the exceptional circumstances around the COVID-19 outbreak when, for instance, novel approaches for data collection were needed to face participants’ travel bans. Innovative designs allowed patients to continue to participate in trials and allowed the researchers to evaluate treatments, even in the setting of a public health emergency.
Just before the COVID-19 outbreak, EFPIA contracted a third party (Technopolis) to conduct a survey amongst its membership to explore the acceptance of these novel designs. Around 80% of EFPIA members responding to this survey (26 out of 32 respondents) say they have used more innovative clinical trial designs. More than half of these trials are in oncology and rare diseases and many are found in other areas too.
Despite this, innovative trials have been used in regulatory decisions for only a dozen products. Given the lessons learnt from the COVID-19 experience, this is likely to increase; the pace of growth depends on how open regulators in the system are to assess these innovative studies and what kind of guidance is given to support their use for regulatory decision making, should the investigated medicine in the trials one day be granted an authorisation to enter the market.
The regulators and the European Commission can support the uptake of innovative clinical trial designs
Our Regulatory Road to Innovation spells out actions that stakeholders can take within the current EU legislative framework. For instance, it is extremely important that the EMA and the Clinical Trials Facilitation Group (CTFG) find ways to minimise variation across Europe when it comes to acceptance of innovative trials. The adoption of a common Information System to collect and exploit this new kind of information is an essential success factor.
For its part, the European Commission could provide clarity on how innovative trials fit into the EU Clinical Trial Regulation, expected to come into the application later in 2021 or early 2022.
We will play our part too. EFPIA is ready to work with the EMA, the European Commission and national competent authorities and ethics committees to support pilot projects that would demonstrate the validity of novel designs to bring new treatments to patients earlier and more efficiently.
Europe’s medicines regulations have served patients well for decades – now is the time to adapt our approach to make it fit for the future.