To ensure access to and proper treatment for people with rare diseases, legislation should be enhanced. For example, amyotrophic lateral sclerosis, or Lou Gehrig’s disease, is a gradually progressive neurodegenerative illness of the central nervous system accompanied by damage to motor neurons (Kiernan, et al., 2021). The mentioned disease is characterized by the variability of the debut of motor impairment and the nature of the progression of the pathological process. Thus, public law has been passed on this issue, which should be considered.
Summary of the Act
It is essential to specify that H.R.3537 – Accelerating Access to Critical Therapies for ALS Act is a federal law passed in December 2021 and signed by the President of the United States. The intended target population is people with ALS and other rare neurodegenerative diseases. (All information for S.352 – Health Care Improvement Act of 2021, n.d.) The bill was introduced by Mike Quigley, a representative of Illinois’ 5th electoral district and a Democrat. Significantly, the legislation had 331 cosponsors, including 186 democrats and 145 republicans (All information for S.352 – Health Care Improvement Act of 2021, n.d.). This bill aims to introduce a grant system for the treatment of neurodegenerative diseases such as amyotrophic lateral sclerosis, also known as ALS or Lou Gehrig’s disease. Hence, the Department of Health and Human Services should provide grants to organizations that can conduct scientific research (Kiernan, et al., 2021). This study would include providing access to experimental methods that can assist in treating ALS for people who are not eligible to participate in clinical trials.
However, another provision of the law is that the FDA should offer grants to public and private organizations. This is intended to provide coverage for the research and development of medications to diagnose or treat ALS and other rare neurodegenerative disorders. The Accelerating Access to Critical Therapies for ALS Act also established a Public-Private Partnership for Neurodegenerative Diseases between the National Institutes of Health, the FDA, and at least one eligible entity (All information for S.352 – Health Care Improvement Act of 2021, n.d.). A corporate entity includes an institution of higher education or a nonprofit research organization. The bill empowers the Partnership to support ALS therapies’ development and regulatory review. At the same time, the FDA is required to publish on its website a five-year action plan to promote the advancement of drug development that enhances or prolongs the lives of people with rare neurodegenerative diseases.
The Reason for Choosing the Bill
I selected H.R.3537 – Accelerating Access to Critical Therapies for ALS Act because it is an essential component of progress toward finding a cure for rare neurodegenerative illnesses. In my opinion, one of the significant obstacles to the development of treatments for neurodegenerative diseases is the insufficient set of biomarkers and related data on the clinical progression of the disease. Consequently, physicians do not have enough information to screen the population, diagnose patients, and monitor disease progression, which prevents them from producing the proper medication (Kim, et al., 2020). Thus, through research, pharmaceutical firms will be enabled to obtain data that will accelerate the invention of new effective drugs.
Hence, the legislation passed is an essential measure to advance the development of treatments for a vulnerable group of patients. Moreover, its value is that it funds, coordinates, and supports research on non-proliferative neurodegenerative diseases. Thus, it is expected that scientific studies will produce a formula for an effective cure and provide help to thousands of patients around the world in a few years.
All information for S.352 – Health Care Improvement Act of 2021. (n.d.). Congress. Gov. Web.
Kiernan, M. C., Vucic, S., Talbot, K., McDermott, C. J., Hardiman, O., Shefner, J. M., A., Al-Chalabi, W., Huynh, M., Cudkowicz, P., Talman, L. H., Van den Berg, T., Dharmadasa, P., Wicks, C., Reilly & Turner, M. R. (2021). Improving clinical trial outcomes in amyotrophic lateral sclerosis. Nature Reviews Neurology, 17(2), 104-118. Web.
Kim, G., Gautier, O., Tassoni-Tsuchida, E., Ma, X. R., & Gitler, A. D. (2020). ALS genetics: gains, losses, and implications for future therapies. Neuron, 108(5), 822-842. Web.