The Must-Have Collaborations for Successful Drug Development: Uniting Patients and Caregivers, Researchers, Industry, and Government Regulators at the Table A White Paper Expanding the Panel Discussion at the Global Genes Third Annual RARE Patient Advocac

Collaborations among academic and government researchers, pharmaceutical and biotechnology companies, regulatory agencies, and patients and their families are crucial to research and development of therapeutics for rare diseases. Industry, academic and government researchers, regulatory agencies, patients and their caregivers all confront challenges in today’s environment for therapeutic development. As a result, the stakeholders are realizing increasing value in collaborations, which can help tackle these challenges. Panelists at the 2014 RARE Patient Advocacy Summit on “The Must-Have Collaborations for Successful Drug Development: Uniting Patients and Caregivers, Researchers, Industry, and Government Regulators at the Table ” discussed the following lessons regarding collaborations on drug and other therapeutics development: • These collaborations serve to advance research and development, build understanding of each other’s perspectives, and develop trust • Each stakeholder has specific, unique strengths to bring to a collaboration, ensuring that a collaboration should be a true partnership among the participants • Recognizing this need for partnerships, patient organizations are being brought into collaborations earlier in the research and development process and also increasingly leading collaborations involving many institutions and stakeholders • However, the need for partnerships also has raised challenges in connecting with partners and managing the different perspectives within the collaboration • Successful collaborations require strong communication with integration of team members and full sharing of data These lessons described by the panelists are discussed in detail within this white paper