Last month, the Energy & Commerce Committee of the U.S. House of Representatives launched a bipartisan effort called the 21st Century Cures initiative to accelerate the process by which drugs are discovered and delivered to patients. Congress has sought input from the patient community and advocacy organizations like MDA.
MDA-associated medical, research and advocacy professionals responded to questions from Congress and included input from MDA community members including families and clinical and research professionals, based on an online survey sent out June 2.
Here are some responses from the MDA survey that are included in the full comment document:
In answering a question about barriers to achieving goals, one researcher said, “The funding environment in the U.S. makes it very difficult to take on a project that might not pan out in the next few years. I have several promising projects that are currently on the shelf; they are too risky to put resources into because other projects are more likely to provide the data I need for the next grant.”
Responding to a question about how regulators should evaluate benefit versus risk in drug development, one MDA-associated clinician said, “Drug screening and safety are important, but it must be recognized that patients with uniformly fatal neuromuscular disease may be willing to endure side effects of an effective drug that may be unacceptable for a healthier patient population. I think that combining the safety and efficacy trials may help move promising drugs through the pipeline.”
In answer to a question about how better treatments or cures might help save money, many of the surveyed families served by MDA said that Congress should “consider other cost reductions that are recouped through decreased dependency on programs such as Medicaid, Social Security and Medicare that would result from increased funding of therapy development.”