Wicker Celebrates New Innovation Hub Tackling Rare Diseases

Usually, bad news gets all the airtime, but this summer brought us a hopeful headline: A new innovation hub will accelerate the development of treatments and products for rare diseases.

I have been working with the Food and Drug Administration (FDA) to coordinate the development of treatments for illnesses that have been historically overlooked. This July, I welcomed the news that the FDA was creating a Rare Disease Innovation Hub, which will increase communication between the teams building various kinds of rare disease therapies. In Congress, I have helped empower these efforts through legislation, awareness initiatives, and my work as Co-chair of the Rare Disease Congressional Caucus.

Care and Cure for Duchenne Patients

The FDA's new innovation hub will expedite the development of treatments for many of the thousands of rare diseases. My work on this issue began when a Mississippian told me his son had been diagnosed with Duchenne Muscular Dystrophy (DMD). The tragic disease progressively weakens muscle strength and affects one in 3,600 boys. At the time, health care providers were focused on treating the symptoms of DMD patients, but few resources were being deployed to end the disease. I believed we could do both. Working with this passionate father, I helped pass a law, called the MD Care Act, that secured federal funding to kickstart the search for a cure.

Our efforts have been paying off. The increased research investment has led to new DMD treatments. That success has piqued wider interest in the condition, generating even more research. With these treatments, lifespans have been increasing for those with DMD. My congressional colleagues and I are overjoyed at that progress. However, we know that complications can arise as young patients enter adulthood, and, in 2014, we updated the MD Care Act to address that concern. That law removed the restrictions that had kept researchers focused on pediatric patients, and it paved the way for new treatments for adults.

Putting Patients at the Center

Congress certainly did not achieve this progress on its own. None of these advances would have seen the light of day without collaboration among government agencies, medical researchers, and the pharmaceutical industry - groups that will be collaborating at the new innovation hub. But most of all, patients and families deserve credit for raising the profile of rare diseases. They have overcome the initial pain of a diagnosis, and they are speaking out.

I have introduced legislation, called the BENEFIT Act, that would increase the role these patients and families play in the treatment-approval process. The FDA is responsible for approving the sale of breakthrough medicines, and it uses a set of metrics to test whether new drugs are ready to go to market. My bill would make sure that real-time patient experience data is one of those metrics. That information will help companies determine the practical benefits and risks of emerging medicines and therapies.

Streamlining Rare Disease Research

That legislation would support the goals of the FDA's rare disease hub. Many patients feel shut out by the FDA's treatment-approval system. It is often too complicated and time-consuming for advocates to stay involved. By streamlining efforts to find cures, the Rare Disease Innovation Hub will make the treatment development process more accessible to patients and their loved ones.

Of course, patients are the reason we have pursued all of these efforts. Since the beginning of the century, more rare disease patients than ever have had access to support, treatment, and hope. With this new initiative, we have even more reason for optimism that cures are on the way.

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