A delicate dance for rare diseases

Europe’s complex rare disease ecosystem relies primarily on a fragile complementary relationship between academia, start-ups, and well-established companies to bring scientific and technological progress. The recent publication of draft impact assessment by Politico Europe renewed concerns that the Commission's revision of the orphan medicinal products (OMP) Regulation may upset the delicate balance required to bring new therapies and solutions to rare disease patients.

This revision will shape the regulatory environment for orphan medicine development for the next 25 to 30 years. Today’s political priorities of affordability and accessibility are shared by all stakeholders including industry. These must be balanced with the need for innovation and competitiveness in Europe. Achieving our shared interest, to deliver innovation that reaches patients, requires creating a future proof regulatory framework and supporting industry’s ability to conduct disruptive R&D in the rare disease space.

Building on a legislative success story

The current framework for orphan medicines has delivered significant societal benefits, with more than 207 orphan medicines approved by EMA since 2000 and patient access across the EU accelerated by 9 months on average. Ultimately this has been achieved by a stable regulatory framework which incentivises increased investments into R&D for treatments for rare diseases, especially for the rarest diseases. This has been driven by a flourishing SME ecosystem that has been sustained by OMP legislation.

Preserving and building upon the stability and predictability of the orphan designation, approvals, marketing exclusivity, and regulatory data protection are essential to bring further innovation to market and support the rare disease ecosystem. Incentives are essential to enable the growth and success of smaller companies who use them to raise the necessary funds to continue their pharmaceutical development. Small and medium sized biotechnology companies are on average much more likely to develop highly specialized medicines. In most cases, those companies evolve from research and development stage start-ups, and build their commercial organisations based on successful product development. The reduction in incentives risks harming the ability of Europe’s small and mid-size biotechnology to raise capital, decrease their value, and ultimately drive innovation out of Europe.

Take heed of the warning signs

The proposals under consideration by the Commission would lower orphan marketing exclusivity, by half from 10 to 5 years but also condition additional rewards on elements beyond the control of the developers of therapies for rare diseases, such as sufficient progress in disease understanding to develop therapies for highest unmet medical needs (HUMN) or access by launching in all 27 Member States regardless of the patient population. Orphan medicines would also be impacted by lower and conditional regulatory data protection under consideration in the revision of the general pharmaceutical legislation.
Should a developer manage to clear all these new hurdles, launching a new orphan medicine addressing highest unmet medical needs in all 27 EU Member States, they would be rewarded with the same 10-year marketing exclusivity available under the current framework. Lower and restrictive incentives risk hindering developers’ ability to attract investments, continue their product development, and bring new innovative therapies to a European population where 95% of rare diseases remain without therapeutic solutions.

Conditioning incentives on launch in all Member States assumes developers are solely responsible for access delay without due consideration for factors at national level. It would also increase the burden on developers to manage 27 different market access strategies and on public authorities to assess pricing and reimbursement applications for medicines they may not have a patient population for.

A disconnect for Europe

At its core, the approach being considered is at odds with the reality of R&D for rare diseases. Incentives should reflect the lengthy development timeframe for orphan medicines and consider the significant risk of failure in areas of high-risk science and low number of patients. Industry invests and develops where the science and their expertise are as companies have specific expertise in certain technology.

Ensuring innovation happens in Europe is critical to avoid further dependencies and deliver for patients. The Commission should strive to maintain a balance between access and competitiveness that will send the right signals to scientists, industry, investors, and patients that Europe is willing to invest in innovation.

Getting the balance right

With the right policies and incentives in place, Europe has the possibility to capitalise on the resources of twenty-seven Member States and break the barriers to growth for established companies, SMEs, and start-ups developing orphan medicinal products. The revision of the OMP Regulation is an opportunity to build on past successes and enable Europe to become a leading region for rare disease innovation in the decades to come. But Europe must seize that opportunity.