Political Advocacy Program Proposal
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POLITICAL ADVOCACY: THE RARE ACT
Political Advocacy Program Proposal
Steven Kristofer Hoskins
Herzing University
HA610-8: Health Policy and Management
G. Stan Reeley, Ph. D.
October 22, 2023
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POLITICAL ADVOCACY: THE RARE ACT
Rare diseases, while individually uncommon, collectively affect millions of people
worldwide. These conditions often result in significant suffering, but due to their rarity, they
receive limited attention and research funding. The Recognizing and Advancing Rare Diseases
Excellence (RARE) Act is a piece of legislation sponsored by Senator Tammy Baldwin with the
aim of addressing the unique challenges faced by individuals living with rare diseases. Rare
diseases, often characterized by their low prevalence in the population, pose significant hurdles
in terms of diagnosis, treatment, and research (
Boycott, K. M., Lau, L. P., Cutillo, C. M., &
Austin, C. P., 2019)
. This summary will examine the key provisions of the RARE Act, discuss
the potential benefits it offers to patients, and advocate for its widespread support and passage.
The RARE Act, introduced by Senator Tammy Baldwin in 2019 and reintroduced in
2021, is primarily designed to establish a Rare Disease Center of Excellence within the National
Institutes of Health (NIH). This center would serve as a hub for coordination and research on rare
diseases, fostering collaboration between researchers, healthcare providers, and patient advocacy
groups. It aims to expedite the development of diagnostics and therapies for these conditions,
address the shortage of medical professionals with expertise in rare diseases, and promote greater
awareness and understanding of rare diseases within the health care system (Congress.gov,
2023).
This act has the goal of allocating additional funding for research into rare diseases. It
promotes reforms to streamline the drug approval process for rare disease treatments, making it
more efficient while maintaining rigorous safety standards. This change could potentially
expedite the availability of life saving therapies. The RARE Act recognizes the importance of
patient advocacy and support by providing resources to establish patient registries and strengthen
patient engagement in research and clinical trials. The legislation includes provisions for
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POLITICAL ADVOCACY: THE RARE ACT
educating healthcare providers about the complexities of diagnosing and treating rare diseases,
which will lead to quicker diagnosis and improved outcomes. The RARE Act also promotes
public awareness campaigns to help people understand the unique challenges faced by this
population and the importance of rare disease research (
Seoane-Vazquez, E., Rodriguez-
Monguio, R., Szeinbach, S. L., & Visaria, J., 2018).
One of the key features of the RARE Act is the creation of a comprehensive, publicly
accessible, and up-to-date Rare Disease Database. This database would contain information
about the prevalence, natural history, and available therapies for rare diseases affecting
Americans. It is intended to be a valuable resource for patients, caregivers, and health care
professionals, aiding in early diagnosis and informed decision making. The Act also seeks to
establish a network of rare disease research consortia, bringing together experts in the field to
share knowledge and collaborate on clinical trials (
Fermaglich, L. J., & Miller, K. L., 2023)
.
My position on the RARE Act is one of wholehearted support. Rare diseases affect
approximately 30 million Americans, many of whom face significant delays and diagnosis,
limited treatment options, and a lack of medical expertise. The rare act addresses these issues
comprehensively by promoting research, raising awareness, and fostering collaboration. This
legislation recognizes the fundamental right of individuals living with rare diseases to access
accurate information, quality care, and effective treatments. Furthermore, it acknowledges the
potential for scientific breakthroughs in rare disease research to benefit not only those with rare
conditions but also individuals with more common diseases, as discoveries in rare disease
pathways often have broader applications (
Adachi, T., El-Hattab, A. W., Jain, R., Nogales
Crespo, K. A., Quirland Lazo, C. I., Scarpa, M., Summar, M., & Wattanasirichaigoon, D., 2023)
.
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4
POLITICAL ADVOCACY: THE RARE ACT
The RARE Act is not only a matter of compassionate care but also a strategic investment
in the future of healthcare. It encourages innovation, reduces the burden on the health care
system by preventing misdiagnoses and unnecessary tests, and supports the development of
precision medicine. Its emphasis on interdisciplinary collaboration ensures that the knowledge
and expertise of different stakeholders in rare disease management are leveraged to their full
potential. As a result, this legislation is not only a moral imperative but also a wise and forward-
thinking policy.
To underline the importance of the RARE Act, it is worth considering the perspectives of
industry leaders and experts in the field. Dr. Stephen Groft, the former Director of the National
Center for Advancing Translational Sciences (NCATS) at the NIH, has stated that the RARE Act
“will advance the progress in rare diseases by creating a home for collaboration among the many
agencies and programs.” Such endorsement from a prominent figure in the rare disease research
community underscores the potential impact of this legislation on the field (
Boycott, K. M., Lau,
L. P., Cutillo, C. M., & Austin, C. P., 2019)
.
Additionally, a study published in the Orphanet Journal of Rare Diseases titled “The
Impact of Rare Diseases: A Systematic Review” highlights the substantial socioeconomic burden
of rare diseases. The study found that individuals with rare diseases often face delayed diagnosis,
misdiagnoses, and a lack of effective treatments, leading to increased healthcare costs and
reduced quality of life. The RARE Act directly addresses these challenges by focusing on
improving diagnosis, treatment, and research, thus offering a pathway to alleviate the economic
burden on both patients and the healthcare system (
Adachi, T., El-Hattab, A. W., Jain, R.,
Nogales Crespo, K. A., Quirland Lazo, C. I., Scarpa, M., Summar, M., & Wattanasirichaigoon,
D., 2023)
.
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POLITICAL ADVOCACY: THE RARE ACT
To take action, you should contact your representatives. Write to or call your senators and
representatives to express support for the RARE Act. Encourage them to prioritize this important
legislation. Spread awareness by using social media, community events, and other platforms to
raise awareness about this act and the challenges faced by those with rare diseases. Join or
support patient advocacy groups working to advance the cause of rare diseases. These groups
often have valuable insights and can amplify our collective voice. Keep informed about the
progress of the RARE Act and other legislative efforts related to rare diseases.
In conclusion, the RARE Act is a crucial piece of legislation aimed at addressing the
challenges faced by individuals living with rare diseases. It provides a comprehensive approach
to research, diagnosis, and treatment, and its potential benefits extend beyond the rare disease
community to the broader healthcare system. My position on this legislation is one of
enthusiastic support, and I urge fellow citizens, healthcare professionals, and lawmakers to join
in advocating for the passage of the rare act. By doing so, we can make significant strides toward
improving the lives of those with rare diseases comma advancing medical knowledge comma
and building a more equitable and efficient healthcare system. Rare diseases should no longer be
ignored. They deserve the recognition and excellence the RARE Act promises to provide.
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POLITICAL ADVOCACY: THE RARE ACT
References
Adachi, T., El-Hattab, A. W., Jain, R., Nogales Crespo, K. A., Quirland Lazo, C. I.,
Scarpa, M., Summar, M., & Wattanasirichaigoon, D. (2023). Enhancing Equitable Access to Rare
Disease Diagnosis and Treatment around the World: A Review of Evidence, Policies, and
Challenges.
International journal of environmental research and public health
,
20
(6), 4732.
https://doi.org/10.3390/ijerph20064732
Boycott, K. M., Lau, L. P., Cutillo, C. M., & Austin, C. P. (2019). International
collaborative actions and transparency to understand, diagnose, and develop therapies for rare
diseases.
EMBO molecular medicine
,
11
(5), e10486.
https://doi.org/10.15252/emmm.201910486
Fermaglich, L. J., & Miller, K. L. (2023). A comprehensive study of the rare diseases and
conditions targeted by orphan drug designations and approvals over the forty years of the Orphan
Drug Act.
Orphanet journal of rare diseases
,
18
(1), 163.
https://doi.org/10.1186/s13023-023-
02790-7
Seoane-Vazquez, E., Rodriguez-Monguio, R., Szeinbach, S. L., & Visaria, J. (2018).
Incentives for orphan drug research and development in the United States.
Orphanet journal of
rare diseases
,
3
, 33.
https://doi.org/10.1186/1750-1172-3-33
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