Frequent communications with the FDA is highly recommended throughout a project to create as smooth a path forward as possible.
On May 17, I attended the National Organization for Rare Disorders (NORD) Corporate Council meeting in Washington DC. This was “A Day of Dialogue” with FDA senior staff and other key opinion leaders including Christopher-Paul Milne, DVM, MPH, JD, Associate Director of the Tufts Center for the Study of Drug Development and Yann Le Cam, CEO of EURORDIS, the European Rare Disease Organization.
The NORD Corporate Council meets twice a year to discuss issues and opportunities related to the development of orphan products. There was much discussion around making the drug approval process more flexible on a case-by-case basis, and developing ways to reduce failure as early as possible. Frequent communications with the FDA is highly recommended throughout a project to create as smooth a path forward as possible.
As this was a members-only event, specifics can’t be shared, but there were many valuable programs and resources mentioned that are public knowledge and can be shared.
Involving Patients in the Drug Approval Process
According to Timothy Coté, MD, MPH, the Food & Drug Administration (FDA) is committed to having patients involved in the decision making for drug approvals. To do so, patients can apply to become a patient representative (you have the disease) or a patient consultant (you have close ties to a specific patient community). Once accepted, these patient advocates are invited to participate in the Advisory Committee meetings.
Ideally, there would be a patient representative or consultant in all approval programs. But the FDA needs help from the industry side. Companies should alert the FDA when a program is starting so that the FDA can then work with NORD to recruit representatives or consultants. Read more about the program.
To further this type of collaboration, attendees were encouraged to become part of the Alliance for a Stronger FDA. This is an independent, not-for-profit organization whose mission is to “assure that the FDA has sufficient resources to protect patients and consumers and to maintain public confidence and trust in the FDA”.
It should be noted that Dr. Coté recently left the FDA’s Office of Orphan Products Development. In June, he took roles with two different organizations: Professor of Regulatory Practice at Keck Graduate Institute and Chief Medical Officer at NORD.
FDA Staff Not Just Pushing Paper
Another piece of information I found fascinating was that once a month Dr. Coté invited a different patient to sit in during his staff meetings. He felt that this helped his staff connect to the human side of their work – and realize that they’re not just pushing paper. Hopefully this practice will continue now that he is no longer in that office.
More Collaboration in Rare and Neglected Diseases
In May 2009, the National Institutes of Health (NIH) announced a new program: Therapeutics for Rare and Neglected Diseases (TRND). The program was designed to bridge the gap that often exists between a basic research discovery and the testing of new drugs in humans. Christopher Austin, MD, Director of NIH Center for Translational Therapeutics and Director of TRND, discussed the agency’s application process and success stories. The overall goal of the program is to be a resource to help advance rare and neglected disease drug discovery projects from academia or industry. Instead of funding, applicants receive significant in-kind research and scientific expertise from the TRND staff.
Mark Your Calendars
On October 11-13, 2011 the DIA U.S Conference on Rare Diseases and Orphan Products will take place in DC. The event is being described as: “The first annual program representing a collaboration among academic researchers, clinical researchers, pharmaceutical and biotechnology companies, regulatory and health agencies, patient organizations, payors and venture capital/private equity concerns to address the need for effective therapies for rare diseases. The Summit will provide a forum for all participants to share of state-of-the-art knowledge and to explore issues and needs from multiple perspectives. Through these dialogs, we hope to facilitate accelerated development of new therapies and improved access to these and existing therapies for rare diseases.”
In addition to the general plenary sessions, there will be three tracks for specific interests:
- Patient Group/Advocacy, as well as general plenary sessions.
Siren is proud to be a sponsor of the event, so I hope to see you there!