#WeWontRest until we make treatments for rare disease less rare

When policy drives progress: Europe’s OMP legislation
Prior to the introduction of the legislation, only 8 orphan-like medicines were available for patients[8] while in just 18 years that number has risen to more than 190. European policy makers should be extremely proud of the fundamental role they have played in tackling rare diseases by developing and approving the Orphan Medicinal Products Regulation. After years of lagging behind other regions of the world that had implemented specific legislation earlier than Europe, the regulation created an environment that has fostered investment and innovation specifically in Europe, leading to a greater understanding of rare diseases and importantly to new treatments. The legislation has raised the profile of rare disease in Europe making it easier for patients to form networks thereby accessing crucial information and communicate their experience and expertise across the research and healthcare communities.
For every case where a new treatment has been developed there are however many more rare diseases where currently no treatment option exists. So much more needs to be done for the 1 in 17[1] Europeans living with a rare disease. We need to ensure a stable and predictable regulatory environment that continues to support and inspire further research into new treatments for rare disease. We need to work collaboratively across the life sciences sector, including critical SMEs on its implementation and create an environment in Europe that realises the potential of rapidly advancing science like gene and cell therapies.
Access today, hope for the future
There are two parts to the OMP access equation:
- Access for the people living with 95% of rare disease where there are no treatment options
- Access for patients living with the 5% of rare conditions where a treatment exists
Of course the potential of these new treatments can only be realised if patients have access to them and in our experience, everyone in the healthcare eco-system; the patients, their families and carers, clinicians, payers, health systems and the companies that develop the new treatment all share the goal of access.

In Europe, our industry, regulators and health systems need to work together to make new treatments available to patients faster. From the regulatory perspective, that means maintaining Europe’s world class regulatory system, making the most of real world evidence and advances in data analytics to evolve our systems and processes to keep pace with the rapidly advancing science and changing treatment paradigms.
- by 2014, 20 European countries had developed national rare disease plans[9].
- A number of countries across Europe have adapted HTA[10] and reimbursement practices specially designed to address the questions posed by the introduction of orphan medicines into the system.
- Companies too, are bringing forward solutions to support patients’ access to new medicines. Flexible approaches such as managed entry agreements were implemented between 2006 and 2012 across a number of European Countries for 26 orphan medicines, featuring innovative approaches such as performance-based arrangements or financial-based agreements.
Right policy, right priority
For patients living with rare disease, it is critical that we find the right policy solution for each priority.
Addressing the needs of the 95% of patients living with rare disease where no treatment option exists means standing by the OMP regulation to drive further research in to the next generation of treatments and cures.
To ensure patients get access to these new treatments is a different discussion which has to include regulators, health system partners, industry, and governments in finding collaborative approaches to support access to and availability of treatments for rare disease. We believe that a High-Level Forum on Access and Healthcare Transformation would be the ideal vehicle to address these issues in partnership.

[1] EURORDIS. Available at: http://www.eurordis.org/about-rare-diseases
[2] Evaluating the Orphan Regulation and its impact on patients and rare disease R&D in the European Union. Available at: http://www.pugatch-consilium.com/reports/Benchmarking_success.pdf
[3] Groft SC. Chest. 2013;144(1):16–23
[4] Kesselheim A and Avorn J. Nature Reviews Drug Discovery. 2013;12;425–31
[5] EMA. Available at: https://www.ema.europa.eu/documents/other/orphan-medicines-figures-2000-2018_en.pdf
[6] Evaluating the Orphan Regulation and its impact on patients and rare disease R&D in the European Union. Available at: http://www.pugatch-consilium.com/reports/Benchmarking_success.pdf
[7] European Commission. 2015. Inventory of Union and Member State incentives to support research into, and the development and availability of, orphan medicinal products — state of play 2015
[8] Office of Health Economics, 2010. Available at: https://www.ohe.org/publications/assessment-impact-orphan-medicinal-products-european-economy-and-society. Accessed: Feb 2017
[9] Rodwell C and Ayme S. Biochimica et Biophysica Acta 1852. 2015;2329–35
[10] Health Technology Assessment
[11] A shared vision for health, Paving the Way in 2019, recommendations in Full. EU Health Coalition 2019