Since its adoption, the Orphan Medicinal Products (OMP) Regulation greatly contributed to the development of orphan therapies, with over 200 drugs approved in the EU, improving the lives of the 6.3 million people living with rare diseases in Europe, their families, and carers. The legislation is an example of how a regulatory framework can foster and drive innovation and investments to address patients’ needs. However, there are still important steps to be taken to address the 95% of rare diseases without an authorised treatment. We must work in partnership and build on the success of the past two decades.
The OMP Regulation has contributed to attracting investment in life-changing therapies for rare disease patients, and can continue with the right incentives for pharmaceutical companies, especially smaller ones. An investment in rare diseases is an investment in everyone’s health: breakthrough scientific discovery often starts in rare diseases and then finds broader applications.
By fostering collaboration between different stakeholders such as industry, patients, and ERNs, among others, we can develop novel therapies and address the unmet needs. Multi-stakeholder partnerships such as the OD Expert Group and Together4RareDiseases embody these principles echoed by the recent launch of the Rare Disease Moonshot initiative.
"At the current pace of R&D, it could take more than 100 years to find treatments for all current rare conditions due to limited knowledge, regulatory hurdles, and safety and financial risks”
To benefit from these partnerships, we should avoid raising new barriers, such as introducing a definition of unmet medical need (UMN) in the review of the OMP Regulation. UMN is a concept that is not fixed, evolves over time, and is variable between patients. The inclusion of this concept fails to reflect the realities of medical innovation and can impact the development of therapies that generate significant value and benefit for patients and healthcare systems. Instead of a restrictive definition, the EU should maintain a broad, common understanding of the highest unmet medical need.
By 2028, all OMPs will be subject to the new EU Health Technology Assessment (HTA) procedure, whose methodology is under development. In order to avoid delays or a lack of access, it is crucial to ensure a suitable methodology that recognises the specificities of rare disease therapies. Similarly, the introduction of a launch conditionality in the OMP Regulation does not help improve access, and could lead to unintended consequences. Due to infrastructure limitation, some therapies may not be launched in all Member States plus many smaller rare disease companies do not have the capacity to launch in all Member States within a limited timeframe. Developers are just one piece of a larger puzzle, thus attaching incentives to launching is not the right tool to improve access and limits the viability of a therapy.
This article was commissioned as part of a series focused on Rare Disease Day 2023, in partnership with Takeda, Eurordis, Efpia and Eucope. Click here to read the full report
At the current pace of R&D, it could take more than 100 years to find treatments for all current rare conditions due to limited knowledge, regulatory hurdles, and safety and financial risks in developing drugs for small populations. At EUCOPE, our small and mid-sized biopharmaceutical companies are dedicated to shaping an agile European rare disease regulatory ecosystem. While the journey to reaching Europe’s shared goal of addressing the 95% of rare diseases without an authorised treatment is long, we are confident that a thoughtfully calibrated framework that fosters innovation and investments can take us one step closer.