Building on a previous blog on the need to reduce medicine approval timelines, it is imperative that all Orphan Medicinal Products (OMPs) be eligible for consideration under the EMA’s Priority Medicines (PRIME) scheme as part of the Revised General Pharmaceutical Legislation. As the EU works towards a more efficient regulatory environment, ensuring faster access to treatments for rare diseases must remain a core objective. With Rare Disease Day on 28th February, now is the time to reinforce the importance of an expanded PRIME framework that prioritises OMPs, while also ensuring adequate resources for regulatory success.

Addressing the gaps in PRIME for rare disease therapies

Current proposals for PRIME as part of the Revision of the General Pharmaceutical Legislation maintain a conditional approach to OMP inclusion. While this recognises the importance of rare disease treatments, it does not go far enough. Given the inherent challenges in rare disease drug development—limited patient populations, high development costs, and scientific complexity—all OMPs should be eligible for enhanced scientific and regulatory support under PRIME. A fully inclusive approach would significantly reduce delays to access and ensure that promising therapies reach patients faster.

The link between PRIME and reduced approval timelines

The need to accelerate medicine approvals in the EU has already been well established. Reducing the EMA’s assessment timeframe from 210 to 180 days is a key step towards improving access. However, to achieve this ambition, PRIME must serve as a robust scheme to further streamline regulatory engagement and minimise bottlenecks that contribute to prolonged approval periods. Expanding PRIME to include all OMPs would enable more targeted early support, reducing the risk of late-stage setbacks and unnecessary regulatory delays.

Ensuring EMA has the resources to deliver

Expanding PRIME’s scope must be accompanied by a commitment to strengthening EMA’s operational capacity. The EU must ensure that EMA is equipped to handle an increased number of PRIME-designated products without compromising efficiency. This means investing in additional scientific expertise, digital infrastructure, and procedural innovations to support a growing pipeline of priority medicines.

The broader impact on innovation and investment

A more inclusive PRIME scheme for OMPs would send a strong signal to developers that Europe is committed to fostering innovation in rare disease treatments. Currently, the complexity and length of the EU regulatory process can act as a deterrent, pushing investment towards other markets with more predictable and supportive frameworks. By aligning PRIME with a broader and more consistent approach to rare disease drug development, the EU can position itself as a leader in this space, attracting further research and development investment.

Rare Disease Day: A timely reminder of what is at stake

Rare Disease Day highlights the struggles faced by millions of people across Europe who have limited or no treatment options. The expansion of PRIME to include all OMPs would represent a concrete step towards addressing these challenges, reinforcing the EU’s commitment to improving outcomes for people living with a rare disease.

The revision of the General Pharmaceutical Legislation offers a unique opportunity to align the PRIME scheme with the real-world needs of people living with a rare disease. Expanding PRIME to include all OMPs, coupled with adequate resource allocation for EMA, will enable faster, more predictable, and more efficient regulatory processes. If the EU is serious about reducing approval times and improving access to life-changing treatments, then a broader and stronger PRIME framework must be a priority.

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