Innovation in medicine is happening at lightning speed. But because of government regulatory roadblocks, patients are being left behind. The U.S. Food and Drug Administration (FDA) is looking at how to change all that with a Rare Disease Innovation Hub. But when it does, it should put those suffering from rare diseases—and those who are seeking cures—at the forefront.
In a new letter, Goldwater Institute Director of Healthcare Policy Dr. Patrick Bailey urges the FDA to use its Rare Disease Innovation Hub to modernize the processes for making advancements in medical innovation more readily available to rare disease patients.
Goldwater’s leadership in federal passage of the original Right to Try, a landmark law that allows terminally ill patients to access innovative treatments that could save their life, and the Right to Try for Individualized Treatments (now law in six states), a reform that expands the right to try to personalized treatments, make the Institute uniquely qualified to weigh in on the new Innovation Hub.
“Both initiatives are rooted in Goldwater’s firm belief that patient autonomy is fundamental to individual liberty, as well as a bedrock principle of medical ethics,” Dr. Bailey writes. “Respectfully, the Institute also shares the opinion that the agency’s current drug approval process is outdated and in disrepair, especially as it applies to the next generation of individualized medical treatments, because Congress has not significantly modified that process in over six decades. Thus, the current means for drug and device approval has not evolved contemporaneously with the rapid advancements in medical technology and innovation occurring over the past six decades. GI believes this has resulted in a system that is excessively paternalistic.”
The FDA’s plan for the Rare Disease Innovation Hub could well achieve its stated goal of enhanced collaboration and cooperation between the agency’s Center for Biologics Evaluation and Research and the Center for Drug Evaluation and Research (CDER) to advance therapy of rare diseases. “However, we are also concerned that it could just as easily serve as a mechanism to further thicken the bureaucratic morass that encumbers patients with rare diseases from benefitting from novel treatments and advancements in care,” Dr. Bailey says.
“To enhance the prospect for achieving its stated goals of aligning review efforts, identifying innovative approaches to endpoints, and enhancing communications with the rare disease community, the Institute advocates that the Rare Disease Innovation Hub Steering Committee include representatives from the rare disease patient community and appropriate key stakeholders from industry who are attempting to revolutionize care for those with rare diseases. Incorporating patient and industry representatives will keep priority focus on patients and on the efforts of those who seek to provide treatments with the potential for valuable impact,” he adds.
Rare disease patients, many of whom are racing against the clock, eagerly seek therapeutic interventions that could help. Including patients will provide them the opportunity to exercise their right to advocate for possible means of saving their own life. This right is “deeply rooted in our nation’s history and tradition,” Dr. Bailey says. “That right is also among those crucial rights that are protected by due process principles, and thereby implicit to our concept of ordered liberty.”
Dr. Bailey’s letter also suggests that the Innovation Hub’s Steering Committee should include stakeholder leaders at the forefront of developing unique products to bring to market benefitting rare disease patients. “The inclusion of these stakeholders will, at the very least, facilitate more effective interaction between the FDA and those innovators by providing a means for more direct communication. It is hoped that such will improve the efficiency of the processes and procedures mandated by FDA to make their novel products available,” he writes.
Lastly, Dr. Bailey advocates that the FDA utilize the Rare Disease Innovation Hub as a means to “explore, initiate, and implement rational processes that simply get out of the way of the trailblazing innovators.” For far too long, federal regulations and superfluous layers of bureaucracy have slowed medical innovations. “For rare- and ultra-rare disease patients to benefit from the next generation of medicine, the federal government must develop a new and more responsive ‘lighter touch’ regulatory approach toward groundbreaking individualized treatments,” Dr. Bailey concludes.
A paradigm shift in medicine warrants a paradigm shift in regulation.
Read the full letter here.
