There was an air of both celebration and trepidation at World Orphan Drug Congress Europe (WODC) as the Rare Disease (RD) community looked back on the remarkable achievements of the last 20 years of the General Pharmaceutical Legislation (GPL) in Europe. Much has been achieved to turn the tide on Europe's lagging leadership in Research & Development (R&D) and translation of life science research into commercialized medicines; perhaps nowhere more so than in RD.
But as the EU prepares to finalize changes to both the GPL and the joint revision of its Orphan Medicines and Pediatric legislation, the question on industry and patient advocacy minds alike is: what will the next 20 years look like for Europe? This EU revision comes on top of new policies in the United States via the Inflation Reduction Act (IRA) and enactment of drastic changes to free pricing for new medicines in Germany. Anxiety is, not surprisingly, running high and 'uncertainty' was a dominating theme of the meeting. This Point of View revolves around three key questions:
- The Question of Incentives: Similar to the situation in the US with the IRA, there is concern that the European Commission will weaken Europe's competitiveness for both R&D and commercialization by reducing market exclusivity and changing both the designation of orphan status and definition of unmet need. A leaked draft in September showed market exclusivity could be cut to eight years. Companies would only be able to claw back two years of exclusivity, and return to the current 10 years, if they launch in all 27 EU markets within two years of EMA approval. This sets a high, and potentially off-putting, bar given the varied reimbursement processes and willingness to pay amongst all 27 member states, especially in rare and ultra-rare diseases where some smaller countries may not have patients to treat.
- The Question of Simplification: Starting in 2025 for medicinal products and 2028 for orphan products, Joint Clinical Assessments (JCAs) will be introduced as part of recently passed EU legislation on centralized Health Technology Assessment (HTA). One joint assessment of clinical benefit for the product will, supposedly, be applicable across all EU countries. Theoretically, this should reduce the burden of HTA processes for both manufacturer and Member State and speed up the time from market authorisation to reimbursement in EU countries, especially those with limited HTA capacity. But given pricing and reimbursement will continue to be a Member State competency, the consensus (even from representatives from regulators and HTA bodies present) appears to be that that JCAs will end up adding an additional layer of bureaucracy in the first instance and do little to accelerate time to reimbursement.
- The Question of Collaboration and Trust: Speakers and panel discussions alike repeatedly returned to the importance (and opportunity) of meaningful collaboration. While OPM and European Network for Health Technology Assessment took center stage, there was discussion about the need for improved connectivity and early dialogue across all elements of the RD ecosystem (companies, patients, regulators, payors, healthcare providers). The infrastructure already exists in the form of Centers of Excellence (CoEs) and European Reference Networks (ERNs) but they only work when all stakeholders participate and collaborate. As one senior executive highlighted, "everyone needs to take one step forward for us to make a significant impact for RD patients." What goes unsaid in this situation is trusting that in stepping forward, everyone else will do so at the same time.
The bottom-line in the policy changes is that: where good policy exists, innovation flourishes. Thus, with the US turning up the heat in the global marketplace, what happens in the EU on both
the OPM legislation revision and implementation of HTA regulations is going to have a profound impact.
