The ALS Therapy Development Institute (ALS TDI) provides a summary of what is included int the ACT for ALS bill, and what it means for people with ALS.

On December 16, 2021, the United States Senate passed the Accelerating Access to Critical Therapies for ALS Act, more commonly known as the ACT for ALS. It’s a bill that promises to enact sweeping changes in how research for Amyotrophic Lateral Sclerosis (ALS) is funded – and who can access these investigational therapies.

If you have been following ALS advocacy efforts over the past several months, you’ve certainly heard of this bill. Advocates in the ALS community, organizations like I AM ALS and ALSA, and elected officials like senators Chris Coons and Lisa Murkowski have worked tirelessly raising awareness and lobbying members of Congress to get it passed.

On December 23rd, 2021, President Biden officially signed the bill into law. But now, what will actually happen? Much of the discussion around the bill has centered around expanding early access to experimental treatments for people with ALS, but this is only part of what the bill does.

The bill has additional measures that could also help to fund organizations like the ALS Therapy Development Institute (ALS TDI) that are working to invent and discover ALS treatments.

We read through the text of the bill itself in order to provide a summary of the actual text of the law, and what it might mean for people living with ALS.

Grant Research on Therapies for ALS

The ACT for ALS will provide for increased access to investigational treatments through grants for expanded access programs (EAPs) for drugs in phase 3 clinical trials. These grants will be administered by the U.S. Department of Health and Human Services (HHS) and will be available for any drug sponsor or clinical trial site that provides access to treatments to people who are not eligible to enroll in a clinical trial for ALS.

Any company or group applying for one of these grants will need to demonstrate that the data generated from this expanded access program will be used to advance ALS research and will not interfere with the conduct of ongoing clinical trials. The grants will cover the sponsor or site’s expenses related to providing access to the drug to people with ALS that are ineligible for trials. The aim of this program is to incentivize more trial sponsors to provide expanded access, and to ensure that they are able to adequately fund these programs.

Public-Private Research Partnership

Beyond expanded access, the ACT for ALS will also seek to support further research into ALS treatments. One way it will go about this is creating a “public-private partnership,” or an official collaboration between the federal government and private companies, universities, and other research groups. It will be led by both the Food and Drug Administration (FDA) and National Institutes of Health (NIH), as well as private institutions that are yet to be determined.

This partnership will seek to move forward research into both ALS and other rare neurodegenerative diseases by fostering new scientific collaborations between public and private institutions, and is authorized to work to secure additional funding, charitable gifts, and grants to support ALS research. The bill places a deadline of one year for the establishment of this partnership.

FDA Grant Program

The bill also calls for the creation of an additional Rare Neurodegenerative Disease Grant Program under the leadership of the FDA. These grants will be available to research organizations studying ALS, as well as related diseases. They will specifically be allocated for projects aiming to learn more about how these conditions progress, discover new drug targets, and create better and more equitable clinical trial designs.

Other Measures in the Bill

No more than six months after the bill becomes law, the ACT for ALS also calls for the publication of a “Rare Neurodegenerative Disease Action Plan” on the FDA’s website. This plan will outline how the agency will develop further initiatives and enhancements to current programs to accelerate the development of ALS therapies and increase access to investigational therapies.

Finally, to ensure accountability for these programs, the bill calls for the US Government Accountability Office (GAO) to prepare a report analyzing their impact and effectiveness four years from the time it becomes law. In order to pay for these various grants and other initiatives, the bill sets aside $100,000,000 in funding per year for the next five years.

Next Steps

Now, the Department of Health and Human Services will begin to set up the programs outlined in the bill, delegating to FDA and NIH where appropriate. While the bill outlines the funding for these programs, HHS may have to work with Congress, who have not approved a budget beyond February 2022, to make sure those funds are available soon. According I AM ALS’ Director of Science and Policy Dr. Megan Miller, making sure this happens is one of the organizations’ main goals heading into 2022.

“I AM ALS looks forward to working with the Biden administration, HHS, NIH, and FDA,” Dr. Miller said in an interview with ALS TDI, “to make sure to ensure that all pieces of this legislation are established in a timely manner, and that they operate in the best interests of the ALS community.”

ALS TDI believes the passing of ACT for ALS is a testament to the power of the ALS community and will offer hope to many living with ALS. We know that we will need to discover many more drugs to meet the needs of everyone with ALS. So, as we celebrate this accomplishment, ALS TDI will continue to build on this hope by inventing and discovering more drugs for ALS - until everyone living with ALS has access to effective treatments.

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