Many biopharmaceutical companies are actively engaged in the research and development of therapies to treat rare disease, despite challenges such as much smaller patient populations than more common diseases. Companies seek opportunities to partner with patient advocacy organizations, academia and the government in order to make even greater progress against rare disease, which collectively affect 1 in 10 Americans.
Last month, we featured a guest opinion by Dr. Steven Groft, who has since retired from the Office of Rare Diseases Research at the National Institutes of Health (NIH), as the first in a series on the importance of collaboration between industry, patient advocacy organizations, academia, and government to continue to make progress against rare disease.
Our next contribution is from Peter Saltonstall, the President and CEO of the National Organization for Rare Disorders (NORD). Founded in 1983, NORD provides information, advocacy, research, and patient services to help patients and families affected by rare diseases. NORD is the U.S. sponsor of Rare Disease Day, an international advocacy day to bring recognition of rare diseases as a global health challenge, on February 28th.
Orphan drugs are starting to lead the way in medical progress and drug development. Approximately one-third of the new molecular entities (NME) approved by the FDA in recent years have been for rare diseases. In 2013, for example, nine of 27 approved NMEs were for rare diseases. The previous year, it was 13 out of 39.
This is remarkably good news for patients with rare diseases. We, and the FDA, expect that orphan drug approvals will continue to trend upward.
One reason for this exciting progress is that FDA has put in place processes that pay special attention to orphan drugs. Anne Pariser, MD, Associate Director for Rare Diseases at FDA’s Center for Drug Evaluation and Research (CDER), at a recent NORD Corporate Council meeting discussed three new programs made possible by the 2012 FDA Safety and Innovation Act: Breakthrough Therapies, Patient-Focused Drug Development, and Pediatric Rare Disease Priority Review Vouchers.
We who represent patients with rare diseases are grateful that the pharmaceutical industry has decided to invest in orphan drugs. Orphan drug development is now regarded in many financial circles as the new focal point for successful investment.
To help expedite still further the development of new orphan products, we at NORD have worked with the FDA to identify some glaring gaps in knowledge that, with relatively modest investment and in a short time frame, could advance orphan drug development even more rapidly.
One example: Since rare diseases can often pose research challenges, we believe that patient registries are needed to expedite patient recruitment. We are seeking funding for a project to develop more systematic ways to develop patient registries. We have learned that patients with rare diseases will participate in registries. We need better models to systematize the registry process.
A related area of need is to develop more natural histories for diseases, an effort avidly supported by the FDA and NIH as a potentially beneficial aid in testing and evaluating new drugs. Again, we believe that drug companies can contribute substantially to this effort and could also benefit significantly from it.
Another project that merits support is our new outreach to state governments to educate legislators and state reimbursement programs about the special needs of patients with rare diseases. We hope this education will help assure that orphan drugs are reimbursed under state programs. We are looking for opportunities to collaborate with companies, most of whom already have well-functioning state outreach programs.
Still another area of collaboration and support is a program to facilitate patient involvement in the FDA approval process. Two years ago, NORD worked with FDA on FDA's first-ever Patient Advocacy Workshop for Rare Disease Day 2012. It was significant in that it was the first time most of the patient advocates who attended had ever set foot on the FDA campus. The event marked the start of a new era in FDA-patient interaction, an effort that merits industry support.
These are just a few of the projects that we at NORD believe merit industry support, and that would create an environment that is even more conducive to orphan drug development and expedited approvals.
FDA is not the only federal agency supporting orphan drug development. At NIH, the National Center for Advancing Translational Sciences (NCATS) is finding innovative ways to bridge gaps in the research process to translate promising early findings into tangible results.
And in the devices area, FDA and NORD are partners in the first-ever public-private partnership to spur progress in the development of medical devices. We see this initiative – the Medical Device Innovation Consortium – as potentially a model for collaboration in other areas of medical diagnostics and therapies.
These programs all represent tremendous opportunities for progress. What is needed is even greater collaboration among research-based drug companies, the patient community, government and academia to take full advantage of these promising opportunities.
At NORD we are excited about the interest demonstrated by so many companies in our Corporate Council, which is contributing to our understanding of the challenges faced by drug and device companies, and which is helping to inform our policy development. We encourage all companies with an interest in orphan drugs or devices to actively participate in the Council.
As we approach Rare Disease Day 2014, which will be the last day in February, NORD encourages the drug industry to build upon this pattern of progress through continued and new support of valuable, innovative programs. Only by working together can we produce results that will have a very real impact on the lives of patients and families.
Peter L. Saltonstall is the President and CEO of NORD
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Stephanie Fischer Stephanie is former Senior Director of Communications at PhRMA, focusing on science advocacy and regulatory issues. As a rare disease patient and stroke survivor, she is very passionate about the need for public policy that encourages innovation and the development of safe and effective new therapies.