More available, accessible, and affordable treatments

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More available, accessible, and affordable treatments

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Treatments are essential for people living with a rare disease. They can provide relief from symptoms, slow the progression of the disease, and, in some cases, even cure the condition. For people with rare diseases, treatments can be life-changing, improving their health and quality of life, and enabling them to fully participate in society.

However, despite the importance of treatments, many people with rare diseases do not have access to them. This is due to a range of factors, including the high cost of developing and producing treatments for rare diseases and the lack of research into these conditions.

Ensuring the accessibility, affordability, and availability of treatments for rare diseases is critical. Accessible treatments mean that people, no matter where they live, can easily get the help they need. Affordable treatments ensure that individuals can access these crucial interventions without facing financial hardships. Moreover, the availability of treatments must be consistent to ensure ongoing care. Lastly, ensuring the effectiveness of these treatments is equally important, to make sure that interventions not only exist but also deliver meaningful improvements in health outcomes.

The EU Regulation on Orphan Medicinal Products has been successful in attracting investment to the development of therapies for life-threatening or debilitating diseases for millions of people who previously lacked satisfactory treatment options. Although over 2,000 orphan designations and roughly 200 orphan medicines have been authorised since 1999, there remain issues with national-level pricing and reimbursement decisions that prevent patients from accessing treatments. Many patients have also received treatments that are intended to treat or cure a different disease through off-label use.

Pink icon of pills, a bottle, and a syringe.

22% of people with rare diseases could not get, in 2019, the treatments they needed because it was not available where they live.

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12% of people with rare diseases could not get, in 2019, the treatments they needed because they could not pay for it.

1/3 of people with rare diseases have never received a treatment directly linked to their rare disease, because it does not exist.

Dark blue icon of an outstretched hand, presenting a shape of a First Aid Cross.

94% of rare diseases don’t have a specific treatment

“EURORDIS was a huge source of support in our advocacy throughout the process which led to the European Medicines Agency giving a positive opinion for a treatment for AKU. The EURORDIS Summer School in June 2011 was a particular highlight as it opened my mind to the whole regulatory process and helped define our strategy ”

Nick Sireau, Chair & CEO, AKU Society

EURORDIS Objectives in Treatments

EURORDIS aims to create a research, development, and delivery ecosystem for rare disease therapies in Europe that is co-designed by both the public and private sectors. This ecosystem must remain concerted for success, and tailored approaches to care, research, and treatment may be required for diseases that have historically been neglected or for whom curative treatments have not yet materialised. We are committed to working towards the faster uptake of new technologies and the adaptation of decision-making processes to make treatments available, accessible, and affordable for all rare disease patients, no matter where they live.

When it comes to treatments for rare diseases, our key objectives are:

Ensure accessible treatments: people with rare diseases, no matter where they live, should easily get the help they need.

Promote affordable treatments: those living with a rare disease should be able to access crucial interventions without facing financial hardships.

Maintain consistent availability of treatments: ongoing care requires the reliable presence of necessary interventions.

Prioritise treatment effectiveness: interventions should not only exist but also deliver meaningful improvements in health outcomes.

EURORDIS initiatives, projects and networks

EURORDIS is dedicated to enhancing access to treatments and therapies for individuals living with rare diseases through a range of initiatives, projects, and collaborations at European and International levels.

Call to Action and Policy Recommendations

1000 new therapies should be available by 2030, in line with the IRDiRC vision

Therapies should be 3 to 5 times more affordable than currently available treatments

Europe is a world leader in the development of rare disease therapies with a competitive regulatory ecosystem and a more robust pharmaceutical and biotech manufacturing presence

Our advocacy and the positions we take on important topics around rare disease treatments are bolstered by the views and experience of our member patient organisations, who collectively represent the 30 million people living with a rare disease in Europe. We collect their input through consultations (often held through webinars and events) or through Rare Barometer surveys, as can be seen in the results of the 2019 treatments survey.

The European Union should:

Evolve the incentives framework in the Pharmaceutical Package to focus on diseases lacking treatments, encourage early dialogue for evidence generation, enhance European competitiveness, and ensure consistent rewards for sponsors;
Develop an R&D model centred on the unmet needs of rare disease patients, mandating patient advocate involvement in all stages, including research, develop-ment, regulatory processes, and assessment;
Create a comprehensive European pathway from development to access, balancing innovation and affordability, aiming for strategic autonomy in R&D, and reducing delays in patient access to treatments. Strategies could include expanding early access programmes, clarifying legal aspects of cross-border treatment access, and revising the directive on pricing transparency for medicinal products under national health systems;
Strengthen European collaboration in pricing and negotiations, building on the momentum of multi-country negotiation platforms and voluntary cooperation mechanisms. These should be further unified and may include common purchasing initiatives, particularly for orphan medicinal products;
Define and implement specific policy and legislative measures to establish a European pathway to access complex therapies such as Advanced Therapy Medicinal Products, that can only be delivered in a few specialised centres throughout the EU.

How can you and your patient organisation make a difference in improving access to rare disease treatments?

As a patient organisation, you can make a significant difference in improving access to rare disease treatments. Here are some ways you and your organisation can contribute:

Participate in research:
Encourage your members to participate in research studies and clinical trials. This can help to generate the evidence needed to support regulatory approval of new treatments and improve access to these treatments for people with rare diseases. Visit our Open Academy website to take part in the EURORDIS School on Medicines Research & Development.

Advocate for policy change:
Work with policymakers to create policies that improve access to rare disease treatments. Use our collective voice to advocate for changes in pricing, reimbursement, and regulatory policies. Join our Brussels Rare Disease Week to become a strong advocate at the EU level.

If you are a policymaker, we invite you to become a member of our Network of Parliamentary Advocates for Rare Diseases, which brings together members of parliament to ensure strong international and local action, shape political input for current and future legislation, and integrate rare diseases into all relevant policies at all levels.

Collaborate with members of EURORDIS and other patient organisations:
Collaborate with other patient organisations, both locally and internationally, to share resources and knowledge, and to amplify your advocacy efforts. Additionally, consider engaging with Patient Community Advisory Boards (CABs), collaborative groups established by patient advocates to discuss and address the latest developments, challenges, and policy-related issues in medical research within specific disease areas.

Raise awareness:
Raising awareness can help to increase funding for research and development of rare disease treatments. Educate the public and healthcare providers about the challenges faced by people with rare diseases and the need for more effective treatments. You can do so by contributing to the EURORDIS Rare Barometer Programme or through the international Rare Disease Day campaign, held annually on the last day of February.