Essential list of medicinal products for rare diseases: recommendations from the IRDiRC Rare Disease Treatment Access Working Group

Abstract Background Treatments are often unavailable for rare disease patients, especially in low-and-middle-income countries. Reasons this include lack of financial support therapies and onerous regulatory requirements approval drugs. Other barriers reimbursement, administrative infrastructure, knowledge about diagnosis drug treatment options. The International Rare Diseases Research Consortium set up the Disease Treatment Access Working Group with first objective to develop an essential list medicinal products diseases. Results extracted 204 drugs diseases FDA, EMA databases and/or China’s NMPA marketing authorization. were organized seven categories: metabolic, neurologic, hematologic, anti-inflammatory, endocrine, pulmonary, immunologic, plus a miscellaneous category. Conclusions proposed is intended initiate discussion collaboration among patient advocacy groups, health care providers, industry government agencies enhance access appropriate medicines all patients throughout world.