Rare Disease Consortium Presents Information on Plans for Meeting 2020 Goal of Developing Diagnostics for Mesothelioma and All Rare Diseases
In a session at the Biotechnology Industry Organization (BIO) meeting in Washington, DC this month, the International Rare Disease Research Consortium (IRDiRC) leaders agreed that encouraging more countries and pharmaceutical companies to join the consortium is critical for meeting their lofty goals laid out earlier this year. Their intention is to provide diagnostic tools for each of the close to 7,000 rare diseases, such as mesothelioma, AIDS and celiac disease, and to develop 200 new treatments by 2020.
A rare or “orphan” disease status is assigned to a disease or disorder if it affects fewer than 200,000 Americans at any given time. Although the number of people with mesothelioma and some other diseases may be low, the loss of life and quality of life places an undue burden on the patients and their families. Between 85 and 90 percent of orphan diseases are serious or life-threatening, yet only about 200 of them currently have any effective treatments.
The initial funding for the consortium is managed through the founding agencies, U.S. National Institutes of Health (NIH) and the European Commission. As additional countries commit to the effort, they will each pay $10 million. “We hope that when it starts rolling other countries come on board to create diagnostics for rare diseases and speed up the development and approval of treatments for rare diseases,” Indridi Benediktsson of the European Commission’s Health Directorate, told delegates at BIO.
Stephen Groft, Director of the Office of Rare Diseases at NIH, added that rare diseases are a “global issue and the needs are the same regardless of country.” He said to meet the goals it calls for a “tremendous, coordinated effort.”
The consortium leaders acknowledge that there will be many barriers to overcome, such as intellectual property rights, but they all agree that rare diseases need to be an international priority. It can take 8-12 years to develop a new drug, and they note that there needs to be methods and agreement for testing and re-purposing existing drug compounds to meet their goals.
Industry watchdogs have their eyes on the consortium’s meeting scheduled for October, in Montreal, Canada, where the consortium will finalize their research agenda. The agenda will be used to build interest within the pharmaceutical industry and attract investigators, clinicians and countries into the rare disease effort.
