Grant: Emerging Team Grant: Rare Diseases - Health Services/GELS

The number of promising technologies for rare diseases has grown recently, bringing new hope to patients. Simultaneously, they have challenged public policy makers who have to decide which technologies to fund. As the number of patients is small, most of these technologies are too expensive for any individual patient to afford and evidence on how well they work is often limited. Thus, the 'risk' of making a wrong funding decision is often higher than for technologies that treat common conditions. The consequences of a wrong decision can be significant - poorer patient outcomes and waste of scarce resources. Therefore, policies enabling access to technologies that optimize both the health of patients and the use of healthcare resources are needed. We describe plans for an Emerging Team on Effective Policies for Managing Rare Diseases. It will bring together members from all stakeholder communities (including patients & families, physicians, policy makers, industry, and the public) in order to generate the knowledge base needed to develop innovative policies. Its activities encompass 5 inter-related themes: coverage decision-making; values & preferences; clinical effectiveness measures; social, legal & ethical issues; innovation policy. These themes represent areas that must be incorporated in such options. Guided by an integrated inter-disciplinary group of researchers, policy-makers, physicians, patients, and industry representatives called the Knowledge Integration Hub, activities will build upon lessons learned from national and international experiences with technologies for rare diseases and those for common conditions. They will examine the methods proposed for addressing issues related to each theme. The anticipated outputs are policy options that span research & development, regulatory approval, and coverage decision-making for new health technologies for rare diseases, and approaches to implementing them that reflect emerging best practice.