Legislative and Policy Priorities to Advance Research

While directly funding research is a core part of the mission of The ALS Association, the organization also advances ALS research by working with Congress and government agencies that play significant roles in accelerating efforts to find a treatment and a cure for the disease.

By partnering with the Federal government, The Association is able to generate significant funding for ALS research, including from the following agencies:

• National Institutes of Health (NIH)
• Centers for Disease Control and Prevention (CDC)
• Department of Defense (DOD)
• Department of Veterans Affairs
• Food and Drug Administration (FDA)

Some key victories for the fight against ALS include the following:

Increasing government research funding

Increased government funding from $15 million annually in 1998, when The Association opened a Public Policy department, to nearly $80 million in 2015. The government has provided more than $950 million for ALS research during that time.

Partnering to Fund ALS Research

• National Institutes of Health: Each year, The Association advocates for increased funding for the National Institutes of Health. In 2016, NIH is expected to provide $52 million for ALS research, the single largest source of ALS research funding in the world. In addition to advocating for increased funding, The Association works with Congress and NIH officials to help direct needed funding to promising areas of ALS research and to encourage collaboration with other federal agencies and the private sector.
• Department of Defense: The Association worked with Congress to establish the ALS Research Program (ALSRP) at the Department of Defense in 2007. The ALSRP is the only ALS research program at the DOD and is focused on translational research, with the specific goal of finding new treatments for the disease. Congress appropriated $7.5 million for the program in 2016, bringing total funding for the program to more than $60 million. This funding has supported 44 ALS research studies which have resulted in 25 peer reviewed publications and, most importantly, the discovery of four potential treatments for ALS.
• Centers for Disease Control and Prevention: The Association advocated with Congress to enact the National ALS Registry Act in 2008, legislation that created the first nationwide ALS patient registry. The registry has become the single largest ALS research project ever created and has enrolled nearly 16,000 people with ALS. The Association also has worked with Congress to secure $58 million for the registry, including $10 million in 2016. The registry has:
  • Funded 13 ALS research projects examining the causes of ALS
  • Generated 30 peer-reviewed publications, more than 50 abstracts/presentations that are advancing our understanding of ALS
  • Helped ALS patients learn about and enroll in more than 20 clinical trials and research studies
  • Launched 17 ALS risk factor surveys to identify potential causes of the disease. People with ALS have completed a total of nearly 60,000 surveys.
  • Established a biorepository that collects blood and tissue samples, including post-mortem samples for use in research. The samples are collected via visits to participants’ homes and are tied to risk factor data collected by the registry. More than 11,000 samples are expected to be made available for research in 2016

Collaborating to Fund Research Projects

The ALS Association’s research program partners directly with the government to fund research projects.

• National Institute of Neurological Disorders and Stroke (NINDS): Currently, we are partnering with the NINDS and contributing $2.5 million dollars to the NeuroLINCS strategic initiative.
• Rare Diseases Clinical Research Network (RDCRN): A partnership with the Office of Rare Diseases, National Center for Advancing Translational Sciences (NCATS) and NINDS fund the CReATe Consortium strategic initiative with The ALS Association investing $1.3 million. As part of CReATe, The ALS Association and partners support an annual biomarker call for abstracts, which seeks the use of bio specimens and clinical data to advance biomarkers for ALS.

Speeding the Development of New Treatments

• Draft Guidance for ALS Drug Development: The Association has launched a groundbreaking effort that has brought the entire ALS community together to create the first patient-focused Guidance for ALS drug development. The Guidance provides the FDA with an ALS community-centered view of how the Agency should approach therapies for ALS with the goals being to increase the efficiency, predictability and speed of the drug development process and deliver promising new therapies to patients as fast as possible.
• Prescription Drug User Fee Act (PDUFA): Every five years, The Association joins with Congress and the FDA to help enact PDUFA. The legislation plays a critical role in providing the FDA with resources to speed drug reviews and also routinely includes other policy changes that help expedite drug development and speed access to new treatments. Recent versions of PDUFA have included provisions to: Strengthen and improve FDA’s Fast Track and Accelerated Approval processes.
• Require FDA to work more closely with patients and patient organizations through Patient Focused Drug Development
• Improve FDA’s capacity to advance rare disease drug development

There is much more work to be done and our more than 17,000 grassroots ALS advocates are critical to the ensuring that Congress and federal agencies continue to be key partners in the search for a treatment and cure.

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