Rare Diseases: Pharma Industry Calls For Collaboration, Political Commitment For Research

Big Pharma wants to develop treatments for rare diseases, with government support. The International Federation of Pharmaceutical Manufacturers & Associations (IFPMA) this week launched a new report describing policy priorities to increase research and development into rare diseases.

Rare diseases are conditions which affect very small numbers of people. Currently between 5,000 and 8,000 rare diseases have been identified. The day 28 February was called Rare Diseases Day.

IFPMA issued a report entitled, “Rare diseases: shaping a future with no one left behind.” It is “a new resource to understand key challenges in areas such as R&D, clinical trials, diagnosis, and access to treatment,” IFPMA said in a press release.

According to the report, “Traditionally, research and development of treatments for rare diseases has been neglected in favour of more common diseases. However, in the last twenty years, concerted public policy efforts have led to a marked improvement in our understanding of many rare diseases and availability of effective treatment options.”

Rare diseases pose particular challenges for research and clinical development, the report said. This is due to the small numbers of patients, limited epidemiological data on the natural history of many rare diseases, and all stages of research and development being more challenging and lengthy than for more common diseases, it said.

The report provides a set of policy objectives, such as that continued R&D be promoted, “building political commitment to drive research, innovation and policies for rare diseases and innovation and policies for rare diseases, and increasing collaborative research efforts to enhance scientific understanding of all rare diseases.

The pharma industry group also called for sustainable access to diagnosis, treatment and care, improving the workforce and infrastructure to treat rare diseases, and developing and strengthening legislation that enhances access to orphan drugs, which are treatments for rare diseases.

Separately, the European Confederation of Pharmaceutical Entrepreneurs (EUCOPE) today called for increased collaboration to develop solutions for rare disease patients. Despite the success of the European Regulation on Orphan Medicinal Products in encouraging the development of new therapies for rare diseases, accessibility varies between European Union member states, EUCOPE said in a press release.