EUCOPE Resources / News - July 12, 2023

Sounds of Science Podcast #16 on OMP Revision’s effects on rare disease companies

EUCOPE’S Sounds of Science Podcast – Episode 16

Rare Reflections: Impact of the OMP revision on rare disease companies in Europe

On 26 April 2023, the European Commission published its long awaited proposal revising the foundational pharmaceutical legislation, including the Orphan Medicinal Products (OMP) Regulation. The OMP Regulation has proven to be a successful legislative initiative, paving the way for over 230 therapies to be developed and spurring a flurry of novel therapies for people living with rare diseases, and beyond. This revision is a key opportunity to build on the successes of the OMP Regulation.

In this episode, we explore the revision of the OMP Regulation as part of the Pharmaceutical Package, and its impact on small and mid-sized pharmaceutical companies, the key drivers of innovation in Europe. This new Regulation will set the tone for research and development for at least the next 20 – 30 years, [3] so we should build a system that reinforces itself, supports partnerships and most importantly, benefits patients. We deep-dive into the implications of specific Commission proposals, and how innovative pharmaceutical companies view the introduction of concepts such as high unmet medical need (HUMN) as the basis for a modulated incentive framework.

EUCOPE is no stranger to proposals to modulate the orphan incentive, having worked with the multi-stakeholder European Expert Group on Orphan Drug Incentives to establish an alternative model, and today’s guest played an important role in developing that approach.

Building on the success of the past 20 years, and establishing a system that continues to drive research, and crucially address the 95% of rare diseases that have no established treatments, is no small task. This requires creative and alternative solutions, and the small and mid-sized innovative pharmaceutical industry is keen to play its role.

To help us understand the impact of this review on rare diseases companies and patients, our special guests include:

This episode is hosted by EUCOPE’s Policy Officer Victor Maertens.

You can listen to Episode 16 and all other Sounds of Science podcast episodes using the links below:

Click on the button to load the content from

Load content

Episode Highlights

“So what we need at the end of the day is not a modulation of the incentives alone but we need much stronger support in terms of public and private basic and medical research through various support, large grants, de-taxation of industrial investment, flexible and tailored regulatory and HTA approaches, massive data collection and reuse for research and development from the ERNs and the healthcare system in general”

“Demonstrating something that is beyond significant benefit and potentially even beyond the therapeutic relevance, which is already tested today within the regulatory environment, is a ridiculously high bar that has very little chance to be passed by the vast majority of drugs in Europe. To me, this HUMN definition has all these limitations.”

“So we really need to make sure that we keep Europe competitive and that we are not losing ground by way of introducing a high unmet medical need (HUMN) stamp to a medicine at a very early point in the life cycle of a medicine which can have drastic consequences for companies deciding to launch in Europe or not launching in Europe.

“We really need to make sure that we’re learning from those experiences with regulatory data protection and that we’re not making the same mistakes in orphan exclusivity. When it comes to addressing the 95% of rare diseases without a treatment option, it doesn’t really matter at the end of the day, if it’s a new compound or old compound, because the patient just cares if its a solution to their condition.” 


For more information on EUCOPE’s efforts on rare diseases and orphan drugs or how your organisation can contribute to it, please contact Victor Maertens

If your company would like to put forward a topic for us to discuss or to participate in a future podcast episode, please reach out to our producer, Dante Di Iulio.