Regulatory Issues for Orphan Medicines: a review

Publication date: Available online 27 November 2019Source: Health Policy and TechnologyAuthor(s): Maria Luísa Bouwman, João José Simões Sousa, Maria Eugénia Tavares PinaAbstractOrphan medicines are medicinal products intended for diagnosis, prevention or treatment of life-threatening or debilitating rare diseases. They are ‘orphans’ because the pharmaceutical industry has little interest under normal market conditions in developing and marketing drugs intended for only a small number of patients suffering from very rare conditions. Successful development of new treatments for rare diseases and their sustainable patient access require overcoming a series of challenges related to research and health technology assessment.The orphan drugs legislation provides a set of incentives and conditions to the pharmaceutical industry to develop medicines for the treatment of rare diseases. In fact, the number of orphan products approved (centralized level) is far exceeding anything seen before the orphan drug legislation. However, the final stage relates to whether a patient is able to receive treatment in a timely and reimbursed manner occurs at the payers’ level (decentralized process), which, leads to inconsistency in patient access between European countries. The challenge of regulatory authorities, patient groups, pharmaceutical companies, legislators and payers is to provide access to new therapies, without geographic or economic discrimination. Despite the progress in th...
Source: Health Policy and Technology - Category: Health Management Source Type: research