Lessons learned from the EUnetHTA Forum — Europe’s effort to make medicines available to all

by Sidharth Tripathi

At RPP, we seek opportunities to better inform our knowledge on health policy so that we may best improve the effectiveness of our services.

So when EUnetHTA, the EU’s joint action promoting Member State collaboration in the field of developing and assessing health technologies, hosted a Forum this past April with various industry, patient, provider, payer and regulatory stakeholder groups to discuss European Union (EU) cooperation on Health Technology Assessments (HTA), our colleagues were steadfast in attending and reporting the outcomes of the event. Here are their main take-aways from the discussions.

What is EUnetHTA?

EUnetHTA is a European Commission-lead effort to create an effective and sustainable network for HTA across Europe in order to develop and implement practical tools to provide timely, reliable, transparent, and transferable information to contribute to HTAs in Member States.

Why does EUnetHTA’s work matter?

EUnetHTA’s ambition is to provide a unique input for policy makers to create meaningful change for patients. Patients may face challenges voicing their concerns in regard to their health conditions, and an EU effort may be a unique opportunity for their voices to be heard.

Ironically enough, the theme of this year’s Forum focused on how EU efforts in the field of HTA will answer to the issue of unmet medical needs (UMNs) in European patient populations. UMNs are conditions for which there exist no satisfactory method of diagnosis, prevention or treatment in the EU, or, in the case that a therapy does exist, this medicinal product would be of great therapeutic advantage to the affected patient groups.

…So what are the implications of unmet medical need for Europe?

Providing timely and affordable access to medicines for patients with UMNs is vastly impacted by the quality of evidence available to government health agencies. Given that medicines for UMNs health conditions must undergo market authorization procedures, the lack of quality data for UMNs impact pricing and reimbursement decisions made during the market authorization process for such medicines. The implications of a limited data market authorization process mean that medicines for such conditions become expensive and inaccessible to patients.

The European Medicines Agency (EMA) currently basis market authorization for UMNs on clinical effectiveness of the medicine and certainty of the risk of potential side effects. EMA offers a route for unauthorized medicines (that is, the medicines have completed the early stages of clinical trials and entered the market authorization process) to reach patients with life-threatening, long-lasting, or seriously debilitating illnesses, yet this case-by-case basis does not effectively counter the ambiguity in a concrete framework to approach UMNs.

What is the way forward?

In regard to UMNs, the European Union is considering pathways for stakeholder involvement and understandings of UMNs yet this may not necessarily be translated into legislation. The heterogeneity of UMNs definitions and lack of discussion amongst various stakeholders in coming to a clear consensus on UMNs have proven to provide barriers on national and European levels for creating transparent processes in market authorization of UMN-related medicines.

The fact of the matter remains that in agreeing with a set definition of UMNs through the integration and validation of various stakeholder viewpoints from across the EU, a mechanism for sustainable cooperation may be developed to push forth the voices of Europe’s most vulnerable – although there is no consensus on the need for increased cooperation either…