Fast Tracking Advanced Therapies – How to Make Europe a World Leader in Medical Research
In the fourth blog in our series looking at the story behind the EFPIA manifesto, Sini Eskola, Director of Regulatory, Drug Development and Manufacturing at EFPIA looks at the case for fast-tracking advanced therapies for patients across Europe.
Our vision is to improve Europe’s capability to fast-track breakthrough therapies which meet the unmet medical needs of patients. Key to realising that ambition is ensuring that the European Medicines Agency (EMA) has the resources and flexibility to be at the forefront of regulatory excellence globally. This is even more critical in the context of the challenges presented by the Agency’s relocation as a result of Brexit.
A good example of these advanced therapies has been the exciting development of cell/gene/immunotherapies such as CAR T-cell therapy. The treatment activates the patient’s own T-cells to attack cancer cells. The first two CAR T-cell therapies were approved in Europe in 2018, but translation from preclinical research to clinical trials for treatments that are classified as advanced therapies poses many challenges that can slow down the development and assessment process while patients desperately await new treatment options.
EFPIA and the Innovative Medicines Initiative (IMI) held a “Think Big: Advanced Therapies” workshop in June 2018 with the goal of transforming outcomes for patients by speeding up the development of, and patient access to, advanced therapies. The key challenges and opportunities were examined leading to the conclusion that more needs to be done in Europe to signal the urgency of accelerating patient access to the most beneficial new medicines.
In Europe, bringing innovative medicines to patients is a multi-faceted challenge, it involves not only the regulatory authorities, but navigating an increasingly complex array of health technology assessments and other market access processes.
Collaboration and partnership will be key, with ongoing involvement of the EMA and the European Commission in public private partnerships to help drive new ideas and the development of robust and impactful proposals on the following areas:
- Regulatory standardisation of gene therapy specific requirements
- Achieving broad and sustained efficacy by collaborating on immunogenicity and vector biology
- Addressing bottlenecks and challenges in manufacturing
- CAR-T specific clinical centres of excellence and standardisation of toxicity management
- Real world data, patient relevant outcomes registries and access
- Developing optimal designs for clinical trials that utilise gene therapy
- Regulatory framework for ultra-rare diseases
In serious conditions with high unmet medical need, the regulatory assessment should be flexible enough to provide patients with early access to new treatments. This includes setting up a European clinical assessment system that accelerates the process of assessing modern clinical trial designs and that harmonises clinical data requirements while removing the duplicate assessments.
Regulatory authorities in other regions are evolving processes to get advanced therapies to patients safely and faster. The US FDA’s breakthrough designation and Japan’s SAKIGAKE procedures are global examples of what can be done to accelerate patient access to novel treatments. In Europe compassionate use programmes already today allow medicines that do not yet have full marketing authorisation, but whose compelling clinical evidence suggests substantial improvement over the existing therapies, to be made available to patients who have no other satisfactory treatment available to them. The programmes, such as the French ATU (The temporary authorisation for use), are co-ordinated and implemented by the EU Member States which decide independently how and when to open such a programme. This is subject to national rules and legislation. EFPIA would encourage more systematic implementation of compassionate use programmes in Europe to realise the full potential of this type of scheme.
The PRIority Medicines scheme (PRIME) which was launched in Europe in March 2016, introduces the possibility to identify (at an earlier stage of development) those products that have the potential to address an unmet medical need and to enhance the regulatory and scientific support for these products through scientific advice at key milestones in their development. Through the PRIME scheme, medicine developers are offered early and proactive support to optimise the generation of robust data on a medicine's benefits and risks and enable accelerated assessment of medicines applications. This will help patients to benefit as early as possible from therapies that may significantly improve their quality of life.
At EFPIA we are ambitious about making Europe a world leader in medical research and development and dramatically improving the lives of patients living with unmet medical needs. #WeWontRest until we have worked with our partners in research, in medicines regulation and in healthcare to get breakthrough therapies to the patients that need them.