Promising Pathway Act offers hope for those suffering from deadly diseases

More than 5,600 Americans are diagnosed with amyotrophic lateral sclerosis each year–meaning 15 Americans are diagnosed with ALS each day. Because this disease is terminal and patients decline so quickly, there is no ALS survivor community.

Those who are diagnosed with ALS and other devastating disease as well as their loved ones, rely on members of the Congress and others to advocate on their behalf.

In January 2020, I co-founded the first-ever Senate ALS Caucus with my colleague across the aisle, Sen. Chris Coons of Delaware.

The mission of the ALS Caucus is to raise awareness about the difficulties faced by these patients, advance policies that improve their quality of life, and to advocate for investments in research for new treatments and to push for increased access to life-saving treatments for ALS.

The ALS Disability Insurance Access Act was a top priority for the Caucus since its inception. At the end of 2020, it was signed into law by President Donald Trump.

For years, Sens. Sheldon Whitehouse (D-R.I.) and Tom Cotton (R-Ark.) championed the ALS Disability Insurance Access Act. Thanks to their efforts, we were able to give ALS patients and their families more timely access to their Social Security disability benefits and allow patients and their families to focus their precious time where it needs to be—on their loved ones and their battle against ALS.

No longer will these patients have to wait five months to access benefits they earned. Oftentimes, they did not have that time to wait.

By coming together and rolling up our sleeves to advance policies that will improve the quality of life for those fighting ALS, we celebrate these patients: those we have lost, those who are currently fighting this dreadful disease, and those whose journeys have not yet begun.

I’d like to talk about another issue where much more work needs to be done: securing access to treatments for ALS patients and for others suffering other deadly diseases with few treatments.

Promising therapies that have demonstrated clinical safety and efficacy are on the horizon for those with ALS. Failure to approve these safe and effective treatments could mean the difference between life and premature death for those fighting this disease.

Many patients with ALS have been very clear that they are willing to take a higher degree of risk to have earlier access to meaningful treatment.

In September 2019, the Food and Drug Administration issued new guidance on developing drugs for ALS, which touted regulatory flexibility when applying the FDA standard of safety and efficacy to drugs for diseases with serious unmet medical needs.

Unfortunately, the FDA’s guidance has been an empty promise, and patients with ALS still lack the flexible regulatory pathway needed to grant them access to promising treatments. 

That is why I have reintroduced the Promising Pathway Act, the legislative solution needed to give those struggling with life-threatening illnesses like ALS a fighting chance to receive access to timely, meaningful treatments.

The Promising Pathway Act would require the FDA to establish a rolling, real-time, priority review pathway to evaluate provisional approval applications for drugs intended to treat life-threatening illnesses such as ALS.

Under this pathway, provisional approval would be granted by the FDA to drugs demonstrating substantial evidence of safety and relevant early evidence of positive therapeutic outcomes.

Critically, it would grant patients timely access to promising treatments that are covered by their insurance.

Americans with life-threatening diseases don’t have time to wait for the slow pace of Washington, D.C., and for millions of families fighting terminal illnesses, access to experimental treatments is their only hope.