Wizemann, Theresa, Robinson, Sally, Giffin, Robert. "7 Strategies for Facilitating Clinical Trials." Breakthrough Business Models: Drug Development for Rare and Neglected Diseases and Individualized Therapies: Workshop Summary. Washington, DC: The National Academies Press, 2008.
The following HTML text is provided to enhance online
readability. Many aspects of typography translate only awkwardly to HTML.
Please use the page image
as the authoritative form to ensure accuracy.
Breakthrough Business Models: Drug Development for Rare and Neglected Diseases and Individualized Therapies - Workshop Summary
points for particular diseases, whether the natural history of the diseases is known, and steps that need to be taken to move forward. Based on the results of those meetings, MDA has issued requests for applications and is funding projects to fill the identified gaps. For example, MDA funded a project at the University of Rochester to develop a survey instrument on the natural history of myotonic dystrophy.
The open discussion in this session expanded on the concept of collaboration among disease foundations that compete directly for funding, and on the importance of considering issues of affordability and access when foundations fund the development of new drugs.
Collaboration Among Foundations
Hesterlee noted that collaborating on the same disease can be difficult because of fundraising issues. It is easier, however, to collaborate across different diseases—for example, looking at common mechanisms. Young pointed out that MMRC recently announced a collaboration with the Leukemia and Lymphoma Society, an organization with which MMRC competes directly for funding. The collaboration is focused on stem cell research, identifying and targeting the myeloma stem cell. Young suggested that collaboration works best in a high-risk area, an area that is unlikely to lead to any therapies in the near term, and one that is somewhat outside of both organizations’ direct missions. With regard to exploring common mechanisms, Pariser stressed the need for caution in designing trials. Diseases such as the muscular dystrophies may look the same, and some, such as progressive muscular weakness, may have a great deal in common, but if their etiologies differ, responses may differ as well, and the desired end points may not be achieved.
Affordability and Access
So inquired how MMRC and MDA are handling the issue of affordability at the end of the pipeline. Hesterlee responded that MDA is considering this issue, and internal discussions are focused on whether the association is going to advocate for Medicare and Medicaid coverage. Part of the plan is to address affordability from the outset by absorbing some of the infrastructure costs. Young noted that there is a limited number of new and expensive drugs for myeloma. Until 4 years ago, patients were treated with chemotherapy and steroids that were all generic. The mission of MMRC is research, and while access has not yet been an issue, it will have to be considered as more drugs become available.