Rare diseases become a priority of the European health policy

1000 novel treatments by 2030

Mon, 2021 / 03 / 01
On occasion of yesterday's Rare Disease Day, people from all over the world came together colorfully painted to raise awareness for rare diseases and identify with affected patients. We at SKC were right among them! Additionally, we introduced six different rare diseases throughout the week. To round off our blog series, we report on the "Rare 2030 Foresight Study", which was initiated by the European Parliament with financial support from the "Pilot Projects and Preparatory Actions" program of the European Commission in 2019. Over the course of two years and together with affected patients, more than 200 experts from the fields of medicine, research, politics and pharmaceutical industry developed an answer to the question:

What future do we want for people living with a rare disease in Europe?

They presented their vision of a European policy which promotes the care of patients with rare diseases to the public at the "Final Policy Conference" on February 23rd, 2021.

The result was the idea of a Europe, which prioritizes societal responsibility, equity and collaborative policy frameworks and therefore together invests for social justice. To reach this goal, the participants of the “Rare 2030 Foresight Study” gave eight recommendations for a mutual European policy:

Among those are methodical recommendations such as the development of long-term, integrated European plans and strategies, the establishment of partnerships with patients and optimising data for patient and societal benefit.

They also call for a juster access to earlier, faster, more accurate diagnosis. Until 2030, all people living with a rare disease known in the literature shall receive their diagnosis within six months of seeking medical attention. To achieve this, all EU-citizens living with a rare disease shall gain access to the most effective diagnostic technologies, best practices and (screening) programs independent of their home location.

This is complemented by the goal of access to high-quality healthcare. Timely diagnoses, health care of high quality and safe, effective and affordable treatment shall be available to all patients with rare diseases as close to home a possible. Whenever this is impossible, access to cross-border health care shall be granted under an EU “whole system” approach without unnecessary delay.

In the context of integrated and person-centered care the psychological, social and economic burden of affected patients and their families shall be reduced by one third.

Additionally, innovative, need-led research and development shall enable patients to benefit from faster development and safer use of curative and symptomatic treatments, improved organization of care and holistic support.

Finally, treatments for rare diseases shall become available, accessible and affordable throughout the whole EU. Therefore, 1000 novel therapies for 500 rare diseases, which accrue 50 % of the overall rare disease patient population, shall be approved by 2030. Among those shall be curative treatments for 100 diseases, which affect less than one of 100,000 people. At the same time, costs for these therapies shall be three to five times more affordable than currently available treatments.

Even though these noble goals require great efforts from politics, pharmaceutic entrepreneurs and health care providers, the prospect of a pan European solution makes us optimistic for a timely improvement of the care and quality of life of patients living with a rare disease. It remains to be seen, how the proposed price reductions impact the endeavor of increasing the number of licensed treatments for rare diseases by six-fold.

We at SKC are looking forward to supporting the market access of pharmaceuticals for rare diseases and to tackle the mutual European challenge.

  • More information on “Rare 2030” can be found here.
  • Details on the recommendations of the “Rare2030 Foresight Study” can be looked up here.

About the author

Ihre Ansprechpartnerin  Svenja Sake, Ph.D.
Svenja Sake, Ph.D.
Fon: +49 511 64 68 14 0 – 0
Fax: +49 511 64 68 14 18
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