Hoosier State Today

The bipartisan bill will help patients with ALS and other life-threatening diseases like DIPG get access to life-saving treatments

WASHINGTON – On June 8, Senators Mike Braun, Kirsten Gillibrand, Lisa Murkowski, Kevin Cramer and Roger Wicker are introducing the Promising Pathway Act, a bill to help Americans with ALS and other life-threatening diseases such as DIPG get early access to promising treatments. 

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Americans diagnosed with ALS are typically given 3 years to live. ALS has no cure, but there are promising treatments. Promising Pathway Act allows ALS patients to receive early access to treatments under a provisional approval while research continues for full FDA approval. The bill will also help patients with other life-threatening such as Diffuse intrinsic pontine glioma (DIPG). Treatments eligible for provisional approval under PPA must have substantial evidence of safety and early evidence of positive therapeutic outcomes. Promising Pathway Act would also open up the door to provisional approval for treating other terminal diseases with limited to no current treatment options. “Americans with life-threatening and terminal diseases like ALS don’t have time to waste on a bureaucratic journey through red tape, only to be denied access,” says Senator Braun. “I’m proud that the Promising Pathway Act is now a bipartisan solution to help these Americans get access to life-saving treatments.” – Sen. Braun

“Patients with life-threatening illnesses can’t wait for years for the FDA to approve new drugs. They need treatment as soon as possible, and this bill would help make sure they get it. The Promising Pathway Act would open a provisional FDA approval pathway for drugs and therapies that treat conditions where the disease progresses rapidly and few to no treatment options exist while continuing to ensure that these new therapies are safe and effective.”—Sen. Gillibrand

“Red tape bureaucracy delays access to treatment for ALS patients, and those with conditions don’t have time to wait. The FDA’s complex approval process for drugs and therapies only adds to the stress of patients and families weighing various treatment options. Removing barriers and establishing a rolling, priority pathway for the approval of lifesaving treatments is the least we can do.”—Sen. Cramer

“I AM ALS appreciates the bipartisan efforts of Senators Braun and Gillibrand with the Promising Pathway Act. We look forward to working with the Senators along with the FDA, clinical experts, and our own community to ensure that people facing rare, rapidly progressing, fatal diseases like ALS always have access to quality treatments pathways.” - Andrea Goodman, I AM ALS CEO

“While the FDA is the gold standard for drug review and approval, the agency can exercise greater flexibility when making decisions for patients facing aggressive terminal diseases like ALS. Given that most people diagnosed with ALS will be dead in three years, patients and their families do not have time to wait for promising therapies through the long trial approval process. The Promising Pathways Act gives patients the opportunity to make decisions critical to their health, where few opportunities currently exist.”—Steve Gleason, Founder of both Team Gleason and Answer ALS and is a living with ALS

"We support Congressional efforts to expedite access to healthcare innovation for vulnerable patients with serious diseases. In our discussions with FDA regarding Barth syndrome, an ultra-rare life-limiting disease, FDA has acknowledged the need for new approval pathways where traditional efficacy studies are not feasible or ethically possible. The Promising Pathways Act is a vital initiative to bridge this gap for those affected.”— Reenie McCarthy, CEO of Stealth BioTherapeutics

“The Promising Pathways Act is a bipartisan solution to delivering high-quality and lower cost drugs to American families. By creating a more streamlined approval pathway for drugs that treat rare diseases, drug developers will be able to more effectively deliver life-saving cures to millions of patients in need of care. This legislation is key to creating a Personal Option that puts people in control of their health care.”— Charlie Katebi, Senior Policy Analyst, Americans for Prosperity

“The Promising Pathway Act represents more than just an accelerated and ethical drug pathway.  For children fighting terminal cancers, the Promising Pathway Act represents the first hope we may have at giving them life.  PPA accelerates cures, refocuses therapies on the correct patients and creates an incentive for companies to invest in rare diseases in ways that we’ve never had before.”—Keith Desseriech, the Cure Starts Now

"We thank Senator Braun and the bipartisan group of cosponsors for their efforts to compassionately remove red tape for those with rapidly progressing terminal illnesses. The Promising Pathway Act is a sensible way to give patients and their families access to additional treatments, innovative drugs or biologics, and hope for the future. There’s a compelling and demonstrated need for expedited access to treatments, and allowing this through the PPA is the right thing to do."—Robin Walker, senior federal affairs director for the Foundation for Government Accountability (FGA)

"The Polen Family wholeheartedly support the Promising Pathway Act and commend Corey for his tireless efforts in shaping this vital legislation. The PPA is a significant step forward in empowering individuals providing opportunities to fight for their lives and to secure access to safe, promising therapies.  We are proud to #StandForCorey in championing this important cause."—The Polen Family

“The Promising Pathway Act offers a transformative solution to the critical challenges faced in accelerating the search for a cure for brain cancer and other deadly diseases. With three decades of experience working closely with brain cancer patients, I have witnessed the hurdles posed by the lengthy and costly journey from lab to patient, as well as the complexities of accessing potentially beneficial drugs that may contribute to breakthrough treatments.”— Al Musella of the Musella Foundation “The Promising Pathway Act empowers the entire healthcare ecosystem to work more closely together to ensure smart and timely review of important new therapies and reinforces the adoption of innovative pre and post-marketing 21st century regulatory science.”— Peter Pitts, President, Center for Medicine in the Public Interest

“We believe this potential pathway may provide timely access for patients grappling with rare, life-threatening diseases by striking a delicate balance between the need for urgency and a strong commitment to ensuring the safety and effectiveness of treatments.”—Meredith Manning, PharmEssentia President, The Americas

"The Promising Pathway Act has the potential to transform the search for cures for deadly diseases by addressing critical challenges to development and patient access. By further streamlining the approval process for rare disease treatments, the Promising Pathway Act empowers drug developers to more efficiently deliver treatments to patients in desperate need. It stands to revolutionize healthcare and provide hope for patients with life-threatening diseases that currently lack suitable treatment options."— Anish Bhatnagar, M.D., Chief Executive Officer of Soleno Therapeutics. 

The Promising Pathway Act has enormous support among patient communities.   

The Promising Pathway Act creates a provisional approval pathway for promising treatments for diseases like ALS that progress rapidly, have few or no treatment options, and result in death. 

BACKGROUND: 

• Promising Pathway Act (PPA): this bill would expedite beneficial outcomes for patients by requiring the FDA to establish a rolling, real-time, priority review pathway for drugs intended to treat, prevent, or diagnose serious or life-threatening diseases or conditions. 
• Under this pathway, FDA would grant time-limited, provisional approval to drugs that demonstrate substantial evidence of safety, and relevant, early evidence of efficacy. Drug sponsors would be allowed to incorporate scientifically-substantiated surrogate endpoints and real-world data to demonstrate the efficacy of the drugs under review. The period of provisional approval is time-limited and effective for a two-year period. 
• Drug sponsors may request provisional approval status renewal for subsequent two-year periods (up to a total of eight years) and can apply for full approval at any time. PPA would require patient registries for all provisionally approved drugs to track patient usage until the drug is fully approved. 
• The FDA would review the drug and renew provisional approval status based on real world data collected in the patient registries—which track patient usage of provisionally approved drugs—until the drug receives full approval or provisional approval expires. Under this provisional approval pathway, those with rapidly-progressing terminal illnesses would have access to drugs that provide their only hope for treatment, and real-world data collected from these patients would be incorporated into the drug approval process.

Original source can be found here