EU working on reforming pharmaceutical legislation to tackle rare diseases

Brussels – Too many rare diseases and too few answers. There is a lack of drugs and resources to promote their research and development, and the European Union is trying to strike a remedy. So announces Stella Kyriakides, Health Commissioner, on the eve of the International Day for Rare Diseases, . In Europe a disease is considered rare when it afflicts no more than 5 out of every 10,000 people. “For up to 36 million people across the EU today, living with a rare disease is a daily reality“, the commissioner stressed. She lamented that “most of them have no cure, and this causes fear and frustration”.

Here is the picture taken by the European Commission: only in the territory of the 27 member states exist more than six thousand rare diseases differing in their characteristics and prevalence. One disease may affect only a handful of patients; another may touch up to 250 thousand. Of these widespread diseases, about 80 per cent are genetic in origin, and of these, 70 per cent begin as early as childhood. There is a network of 1.619 specialized centres and 382 hospitals located among the member states of the European Union plus Norway (a country outside the EU but cooperating in health care), and to date, about 250 orphan drugs, specific medicines used for diagnosis, prevention, and treatment of rare diseases have been authorized, plus two thousand products under development.

Much has been done, but more is needed. From a financial point of view, “in the next few years, the EU will invest more than 77 million euros in consolidating and improving these networks” of specialized centres, Kyriakides stressed.”To better integrate them into national health systems, we are funding a new joint action with more than 18 million euros over the next three years.” And then, the commissioner continues, “We are providing financial support under the Horizon Europe program” for research, which includes “100 million euros for the development of diagnoses and treatments under a new European partnership on rare diseases.”

However, there is a more political part, which concerns the reorganization of the single drug market. “Looking to the future, as part of the pillars of the European Health Union, there is the reform of the EU pharmaceutical legislation,” the Health Commissioner continues. This reform “aims to target pharmaceutical investments in drugs for rare diseases and to focus on disadvantaged areas.” This is an addition to the critical drug alliance and intended to strengthen the health union that arose in the wake of the pandemic crisis.

EU action on rare diseases aims to improve the diagnosis, care, and treatment of patients with rare diseases through resource sharing and cooperation. The policy intends to facilitate medical and pharmaceutical sectors through “targeted incentives and regulatory support to reward exceptional therapeutic advances,” as well as foster the exchange of valuable data and information within the European health data space.