Global pharmaceutical and services company Clinigen and news and information provider CheckOrphan have announced the 128th addition to a unique online access programme register for rare diseases, on the first anniversary of its launch.

The register is the first resource of its kind for the rare disease community, offering a searchable database of ethical and compliant access programmes for rare disease therapies not available through clinical trials or via the usual commercial route.

Since its launch in February 2015, the register now includes 128 active access programs from over 90 companies for rare disease and orphan drugs across a wide range of therapeutic areas and geographies. 

“Access to unlicensed medicines is an area of huge importance to the rare disease community and our hope is that the register will continue to expand,” noted Maxine Hamilton, managing director of Clinigen’s managed access arm Idis. “We want to ensure that physicians, patients and their families receive appropriate information about ongoing access programs for potentially life-saving treatments for their conditions”.

Over 6000 different rare diseases have been identified to date, affecting over 60 million people in Europe and the USA alone. But due to the low prevalence of each disease, medical expertise is rare, knowledge is scarce, care offering inadequate and research limited, says patient group alliance EURORDIS.

“People with rare diseases endure years of uncertainty waiting for their disease to be diagnosed and for an appropriate treatment to be found. We must pursue our efforts in helping to ensure that each person suffering from a rare disease gets the support and treatment she or he needs,” said Commissioner Vytenis Andriukaitis, EU Commissioner for Health and Food Safety, speaking at a symposium to mark Rare Disease Day 2016. 

“I am convinced that co-operation at EU level can make a difference. Indeed, the European Commission can help in pooling together expertise, for example through the European Reference Networks, in supporting research, in granting the authorisation to the best possible medicines and in fostering co-operation to make medicines more affordable,” he noted.

“Opportunities for the translation of scientific advances into new therapies are growing. Nevertheless, rare disease treatments are not being developed and accessed quickly enough,” said Yann Le Cam, EURORDIS’ chief executive. “Scientific innovation cannot be disconnected from access to medicines. If an innovative medicine is approved but does not reach all of the patients who need it, it fails in its primary objective.”