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Senator Braun introduces Conditional Approval Act for patients with fatal diseases, asks for comments

WASHINGTON, D.C. — To make good on the federal government’s promise to allow those with life threatening illnesses better access to potentially meaningful treatment options, Senator Braun has introduced a bill to amend the Federal Food, Drug, and Cosmetic Act to establish a time-limited conditional approval pathway for those with terminal illnesses.

Senator Braun is soliciting comments from all interested parties on how to improve the legislation and provide a more promising pathway for meaningful treatments to reach Americans struggling with deadly diseases.

“Patients with fatal diseases are fighting for their lives every day while real, meaningful, life-extending treatments sit on the shelf just beyond their reach,” said Senator Braun. “For those who are so courageously battling these terrible diseases, the least we can do is not stand in their way.”

Please submit comments or questions to ConditionalApproval@Braun.Senate.gov for consideration.

Comments must be submitted by Monday, February 3, 2020

View the full bill text here:

Bill Text

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“I firmly believe that patients with limited time should have the liberty to seek meaningful treatments in their own country—in a timely manner—where they can be close to their family and loved ones.  It is my hope that the harsh realities associated with Amyotrophic Lateral Sclerosis, Duchenne syndrome, and other devastating diseases—and the limitations associated with clinical research—will encourage policymakers to do everything in their power to provide faster access to meaningful treatments.

“While the U.S. Food & Drug Administration (FDA)—working with disease communities across the country—has been steadfast in its quest to provide more access to meaningful treatments to patients suffering from life-threatening, serious, and chronic diseases—most patients with these diseases, and their families, are fed up with the lack of available treatment options.  Indeed, there are real concerns with respect to the FDA’s progress for expediting approval for new medications shown to meet currently unmet needs for the many Americans who continue to look for meaningful treatment options.  

“For those diagnosed with a life-threatening, serious, and chronic disease—timely access to already existing, safe treatments, that have demonstrated effectiveness should be a given.  The current “all or nothing” drug approval system does not reflect the realities of clinical research or the economics of the prescription drug market.  For example, FDA typically requires three phases of clinical trials before manufacturers can market and patients can benefit from a new treatment.  These trials can easily take 12 years or longer and usually cost the sponsoring company upwards of $2 billion.  

“The Conditional Approval Act, if implemented, would offer patients with life-threatening, serious, and chronic diseases—who are not enrolled in clinical trials—the ability to access meaningful treatments sooner.  As part of this new conditional approval pathway, FDA would engage in enhanced safety monitoring and continued processes towards full approval.  Indeed, under this approach, the FDA could allow patients to access safe and promising treatments, while still maintaining the option to revoke the conditional approval should unexpected data come to light.  But in circumstances where promising treatments have gone through early phase clinical testing—having established safety and a reasonable expectation of effectiveness—patients and their medical providers would be offered the opportunity to gain access to potentially meaningful treatments in a manner regulated by the FDA.  This would also allow patients with life-threatening, serious, and chronic diseases a route to access potentially meaningful treatments without facing the obstacles associated with the Compassionate Use, Expanded Access, or Investigational New Drug processes at FDA.

“The legislation seeks to remove the existing barriers for patients with devastating diseases—including those previously enrolled in a promising clinical trial and whose role in the trial has ended—and provide them the ability to access promising treatment before it is too late.” – Senator Mike Braun

BACKGROUND:

Press Release from the Office of Congressman Bruce Westerman, sponsor of Conditional Approval Act in the House of Representatives:

The FDA refers to the clinical trial process for prescription drugs in phases, i.e. Phase I, II and III. Early clinical trials (Phases I and II) establish and confirm safety while providing a considerable amount of data, often enough to statistically predict successful confirmatory trials. Confirmatory trials (Phase III) further test effectiveness and are the last step to a drug entering the market for patients’ use under a New Drug Application or Biologics License Application approval.

However, Phase III trials are extremely time-consuming and expensive, in some cases costing billions of dollars. If the FDA determines a drug doesn’t meet its standards of safety or effectiveness, small pharmaceuticals attempting to bring innovative drugs to market and compete with large drug companies can go bankrupt during Phase III trials.

The Conditional Approval Act would allow pharmaceutical companies to petition the FDA for conditional approval if the drug has cleared early stage clinical trials, already having proven safety and showing significant evidence of effectiveness. The company could then sell their drug at a market acceptable rate, give patients access to innovative treatments and compete with large, monopolistic pharmaceuticals to lower consumer cost.

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