Gus Bilirakis Helps Shape Food and Drug Amendments

Last week, the U.S. House passed the Food and Drug Amendments which included several proposals from U.S. Rep. Gus Bilirakis, R-Fla.

Back in May, Bilirakis showcased his support for a proposal “to help further development of pediatric medical devices by reauthorizing the Pediatric Device Consortia (PDC) grant program and allowing profits for certain Humanitarian Device Exemption (HDE) devices.” U.S. Rep. Lizzie Fletcher, D-Texas, introduced the proposal with Bilirakis as a co-sponsor.

“Our children deserve the same commitment to the development of life-saving and life-changing devices that we make to adults,” said Fletcher. “I am glad to partner with Congressman Bilirakis in this bipartisan effort to bring beneficial treatments and technologies to children across our country.”

The congressman’s office offered some of the reasons behind why he was backing the proposal.

“Medical devices for children often lag five to ten years behind that of adults because of the unique challenges that device manufacturers face in creating safe and effective medical devices for children, such as differences in size, weight, and metabolism rate. The market for pediatric devices is also smaller than the market for adult devices, leaving little economic incentive for manufacturers to develop these devices. To encourage manufacturers to create devices for children, Congress lifted the profit restrictions placed on pediatric devices that goes through the HDE pathway. The HDE pathway is for devices that treat or diagnose a rare disease or condition that affects fewer than 8,000 people in the United States. The PDC program brings together individuals and institutions to support pediatric medical device development, and assists scientists and innovators with technical and financial resources to improve the number of medical devices available to children. The PDC program funds five consortia throughout the United States,” the congressman’s office noted.

Bilirakis weighed in after his proposal was included in the package that passed the House.

“Manufacturing devices specifically made for pediatric patients have presented unique challenges and innovation has not kept pace with those developed for adults, leaving many children with rare diseases without devices that appropriately meet their individual needs,” Bilirakis said “Our bill will help address this shortage by continuing to incentivize the development of child-specific medical devices that account for variables like size and body chemistry. By reauthorizing these successful pediatric and humanitarian device programs, we are taking an important step forward in ensuring that our youngest Americans have access to high quality health care.”

The package that passed the House also included a proposal from Bilirakis and U.S. Rep. G. K. Butterfield, D-NC, to reauthorize the FDA’s Orphan Drug Grant Program for five more years. The program “funds studies that address knowledge gaps, support clinical trials and advance rare disease medical product development.”

A year after he introduced the proposal, Bilirakis also saw part of his bill to have the U.S. Food and Drug Administration (FDA) speed up its processes to approve therapies for rare diseases gaining momentum included in the package that passed the House last week.

In March 2021, Bilirakis brought out the “Speeding Therapy Access Today (STAT) Act” with Butterfield as a co-sponsor.

“This bipartisan, bicameral legislation was developed with the input of the rare disease community, and is aimed at improving the development of and access to therapies for the rare disease community,” the Florida congressman’s office noted. “Rare diseases are not a rare problem; more than 30 million Americans are living with one or more rare disease. idly, between 93 percent and 95 percent of the more than 7,000 known rare diseases have no U.S. Food and Drug Administration (FDA)-approved therapy. Traditional regulatory processes have become more complex involving combinations of therapies, genomics, novel diagnostic tests, multi-systemic diseases, small patient populations, and precision medicine. As a result, numerous parts of the regulatory system need to cohesively work together. The development process for a rare disease drug currently takes an average of 15 years, which leaves rare disease patients and their families waiting without access to life-changing or even life-saving treatments. Further complicating the situation is that when new therapies for rare diseases are finally approved under the current structure, patients often face unnecessary delays and barriers to access, resulting in avoidable health deterioration.

“The STAT Act will tackle these issues by enacting targeted, impactful, and attainable policy reforms at the FDA to accelerate development of therapies across the spectrum of rare diseases and disorders and facilitate patient access to such therapies. Specifically, the STAT Act will: improve rare disease coordination, stakeholder engagement, and policy development within FDA by expanding existing authority to create a Rare Disease Center of Excellence, inform rare disease policies and actions by creating a Rare Disease and Condition Drug Advisory Committee, fund regulatory science and related activities to support development of therapies to treat very small rare disease populations, and strengthen rare disease patient access to FDA-approved therapies in both public and commercial plans through enhanced FDA and Centers for Medicare and Medicaid Services coordination, proactive engagement of payers, and specific actions intended to strengthen Medicare and Medicaid beneficiary access to novel therapies,” the congressman’s office added.

Bilirakis weighed in on the bill when he introduced it.

“For me, this work is very personal because I have close family members and friends who suffer with rare diseases. The lack of available treatments and therapies for rare disease patients is a common problem,” said Bilirakis. “The targeted FDA reforms contained in this legislation will have a meaningful impact on the acceleration of treatment development and will provide hope to the millions of patients and families who are coping with a rare disease.”

“There are over 7,000 known rare diseases and no more than 5 percent have FDA-approved therapy. Millions of Americans are suffering avoidable delays and barriers to new therapies due to overly complex regulatory processes that can take over a decade to develop. Patients in the rare disease community are in desperate need of options and cannot wait,” said Butterfield. “I’m proud to join my co-chairs of the Congressional Rare Disease Caucus in introducing the Speeding Therapy Access Today (or STAT) Act to accelerate development and access to potentially life-saving rare disease therapies.”
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Bilirakis reeled in more than 35 co-sponsors including U.S. Rep. Darren Soto, D-Fla. U.S. Rep. Alcee Hastings, D-Fla., also backed the proposal before his death in April 2021.

Over in the U.S. Senate, U.S. Sen. Amy Klobuchar, D-Minn., introduced the companion bill l in March 2021 with U.S. Sen. Roger Wicker, R-Miss., as the co-sponsor.

“Thirty million Americans are impacted by one of 7,000 known rare diseases, but nearly 93 percent of these known rare diseases have no FDA-approved treatment,” Klobuchar said. “It is critical that we pass this legislation to increase the number of safe, effective, and affordable treatments available for people suffering from diseases. As the co-chair of the Rare Disease Congressional Caucus, I’ll keep working to prioritize the needs of rare disease patients and their loved ones.”

“Millions of Americans are living with one or more rare disease, the majority of which do not have FDA approved treatments,” Wicker said. “Creating a Rare Disease Center of Excellence would accelerate the development of therapies for rare diseases and disorders at the FDA while putting patient input first. This would be an important step forward as we work to find cures for all diseases.”

Bilirakis was able to bring in his call to expand the Orphan Drug Grant program to “include new methods in developing regulatory science of individualized therapies for those with rare diseases and conditions” even as the rest of the STAT Act was not included in the package that passed the House.

“With these important measures to reauthorize and expand the Orphan Drug Grant Program, we will facilitate the continued research and development of treatments and acceleration of cures for the more than 30 million people in the U.S. with rare diseases or conditions. This health package, with the addition of our bipartisan language, will help patients across the nation have better access to care,” Bilirakis noted last week, adding his “disappointment that the entirety of the STAT Act was not included” while praising the House “for advancing key measures to accelerate development of therapies across the spectrum of rare diseases and disorders, and better facilitate patient access to such therapies, and he remains hopeful to continue to advance policies championed in the STAT Act through an alternative legislative package.”

Kevin Derby
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