The concept of 'patient value' should be integrated in the pharmaceutical legislation
BRUSSELS - The European Commission is reviewing its pharmaceuticals strategy. We wrote a clear response to the public consultation on the revision of the EU general pharmaceuticals legislation: the benefit for the patient should come first.
The European Commission states its intention for its Pharmaceutical Strategy for Europe (1) to be ‘patient-centred’. However, it is crucial that the concept of ‘patient value’ is integrated in the pharmaceutical legislation. It is not sufficient that the EU pharmaceutical system simply ensures quality and safety of medicines, but has in place procedures to ensure that pharmaceuticals bring real added benefit to patients.
Assessment of the added clinical benefit (overall survival/quality of life) of a pharmaceutical is currently not within the remit of the European Medicines Agency (EMA), so market authorization applicants are therefore not obliged to deliver data (overall survival, quality of life or patient-reported outcomes) that are crucial for informed decision-making by Health Technology Assessors (HTA), payers, treating physicians and ultimately patients. It should therefore be mandatory for HTA to provide input to the design of clinical trials to ensure they address both the patient’s needs and the evidence needs of both regulatory authorities and downstream decision makers.
Individual clinical data
Currently, the European Medicines Agency (EMA) does not require manufacturers to submit individual participant level data from clinical trials to inform its approval decisions. This should become an evidentiary requirement to maximise learnings from this data beyond the specific trial research question. In addition to supporting development of new drugs, these data should also meet the FAIR (Findable, Accessible, Interoperable, Reusable) criteria and be available at no or reasonable cost for comparative effectiveness research by a pre-defined range of third parties (e.g. HTA-organisation networks) to inform health care systems.
In order to stimulate innovation and breakthrough therapies in areas of unmet need, third parties having no commercial intent (academia, research foundations, European Reference Networks, collaborative groups) should be incentivized to apply for regulatory approval of medicines not requiring industrial development (combinations of repurposed drugs, autologous cell therapies). In the case of repurposed drugs, public-private partnerships of third parties with drug producers should be supported by a legal system that allows the Market Authorisation Holder (MAH) to rely on data generated by an independent third party to apply for label extension. Scientific advice and regulatory filing should be at minimal cost but will ultimately lead to affordable medicines.
Conditional marketing authorisation
Innovative drugs, in particular Advanced Therapy Medicinal Products (ATMPs), often receive regular approval, albeit with incomplete data and uncertain therapeutic benefits. When only incomplete data are available, EMA should routinely use the ‘conditional marketing authorisation’ pathway. EMA, with input from European Health Technology Assessment bodies, should require post-marketing studies that directly address the uncertainties about the drug benefits and harms at the time of approval.
Infrastructures to facilitate the collection of real-world evidence and real-world data to confirm proof of efficacy must be prioritised, and investment should be made in data registries which use harmonised standards. It is imperative to allow the transfer of information across borders and as such the General Data Protection Regulation (GDPR) must not be an impediment to collection and sharing of patient data in such a way.
The COVID-19 pandemic emphasised the need for the EU’s competences in health to be expanded. Assuming that joint clinical assessment by a European HTA will become a reality soon, an important task could be dealing with false, unproven therapeutic claims. Especially in the domain of cell therapy and oncology, the offer of miracle treatments thrives in Europe, putting at risk patient safety. However, debunking false claims is not currently within the remit of the EU.
(1) Drawing lessons from the COVID-19 pandemic, the European Commission plans to evaluate and revise the EU’s general legislation on medicines for human use to ensure a future-proof and crisis-resistant medicines regulatory system. This is part of the EU pharmaceuticals strategy.
To find out more about the revision of the EU general pharmaceuticals legislation, to be adopted in the fourth quarter of 2022, please have a look here.