ECRD 2020: OMPs Development in a post-COVID-19 World

The 10th European Conference on Rare Diseases (ECRD), the largest patient-led event on rare diseases and orphan products, presented an excellent opportunity for exchange among the patient community and experts from various backgrounds.  Bringing the perspective of OMP focused small to medium companies, EUCOPE held fruitful dialogues with the key stakeholders in the rare space.

Marking the 20th Anniversary of the EU Regulation on Orphan Medicinal Products (OMPs), EUCOPE commissioned a study to the Office of Health Economics aimed at assessing how far the legislation has incentivised investment in the R&D of OMPs and what would be the impact of changing some of its core legislative elements.

The key findings of the study were shared in an online poster presentation at the ECRD. One of the key takeaways is how the EU OMP Regulation has successfully incentivised companies to invest in the development of OMPs. Orphan Designations granted by the European Commission have grown at a rate of 15% since 2000 and 35% of the marketing authorisations granted between 2008 and 2018 were to OMP companies focused solely on developing products for rare conditions.

Maintaining a strong incentives framework for OMPs is vital

EUCOPE also had the opportunity contribute to the broader policy debate with a presentation delivered by Secretary-General Alexander Natz on the evaluation of incentives for orphan drugs and the future outlooks in the development of treatments for rare diseases.

Commenting on the topic, Alexander Natz said: ‘’Maintaining a strong incentives framework for OMPs is vital for Europe. However 95% of rare diseases patients still don’t have any treatment options. Thus, policymakers would need to take into account the boarder challenges related to development and access in the OMPs field, including challenges in the generation of real word evidence and the higher financial vulnerability of small and medium OMP-focused companies.’’

Given its impact on the health and pharmaceutical sector and economies at large, we looked at OMP development future outlooks in light of the current COVID-19 pandemic. On that note Alexander added.

We experienced a 10% erosion in biotech commercialization between 2018-2019 and this was before the impact of the economic shutdown of COVID-19. Funding shortages will be much larger post-COVID-19, however, if Europe does not invest in next generation of science with its pharmaceutical strategy, to be closely linked to COVID-19 recovery plan, we risk to lose OMP infrastructure and further harm competitiveness and innovation

 A lively debate followed the presentation with exchanges on the importance of the use of real-world evidence in the HTA process for OMP and further inclusion of patients in these mechanisms. The discussion further highlighted how only a joint effort of decision-makers, patients, healthcare professionals, HTA authorities, OMP developers and payers can successfully address these challenges.

May 15, 2020

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