Legal & Regulatory Aspects of Rehab - Health Related Agency Report

What I learned from this assignment: 

Completing this assignment taught me a great deal about the complex work that goes into healthcare policy, especially for populations facing rare diseases. By analyzing the Rare Disease Advisory Council’s (RDAC) report, I learned how legislative actions, such as Indiana’s House Enrolled Act 1201, directly lead to real-world healthcare initiatives. It was eye-opening to see how the RDAC organized itself, collaborated with multiple agencies, and addressed barriers like insurance hurdles and lack of access to specialists. I also gained a deeper appreciation for how pediatric cancer research remains critically underfunded despite its importance, and how coordinated advocacy and collaboration between academic institutions and biotechnology organizations can drive innovation in rare disease treatment.

This assignment also shifted my perspective on healthcare policy by showing me that improving access and care for rare disease patients requires far more than just scientific advancements; it demands systemic policy reforms, public advocacy, and dedicated funding. The detailed recommendations from the RDAC, such as mandating pediatric specialists for peer review processes and supporting school reintegration for pediatric cancer patients, highlighted for me the importance of addressing both medical and social dimensions of healthcare. I realized that real change happens when a multidisciplinary group — including policymakers, patient advocates, researchers, and insurers — work together toward a common goal. Overall, this assignment made me more aware of the critical need for both targeted reforms and broader discussions about healthcare as a basic human right.

Health Related Agency Report Morgen Frausto 

Legal & Regulatory Aspects of Rehab  

Prof. Joseph B. Walterman, J.D. 

What department, council, or board submitted the Report? (Not just ISDH, include  the name of the dept., council, or board) What is the date of the report? 

This report is from the Rare Disease Advisory Council (RDAC), and it was published on  June 14, 2024. The main policy recommendations of this report are "to improve patient access to  and quality of rare disease specialists; affordable and comprehensive health care coverage;  relevant diagnostics; timely treatment; and other needed services for patients with rare diseases." 

What is the “policy issue” focused on in the Report? (e.g., smoking cessation; HIV services; pregnancy terminations) or What state law required the Report to be written? 

The report was mandated by House Enrolled Act (HEA) 1201, enacted in 2023 by the  Indiana General Assembly. Its policy focus is on improving care, research, and support for rare  diseases, with a particular emphasis on pediatric cancer. 

Summarize the report which should be at least 1 page long. What information did the Report contain? If there are data, objectives to accomplish, current results, you should  summarize any/all of those. It is expected your summary would be informative enough that  any other student would have a good idea what the report is about and what information it  contained. 

The Rare Disease Advisory Council (RDAC) was established by the Indiana General  Assembly in 2023 to assess and improve healthcare policies and services for individuals with  rare diseases. Since its inception, the RDAC has met monthly, beginning on September 26, 2023,  with the primary goal of identifying barriers to healthcare, promoting research, and providing 

policy recommendations to state agencies, insurers, and legislators. In its initial activities, the  Council adopted bylaws and electronic attendance policies to streamline its operations and  collaborated with several state agencies, including the Indiana Department of Insurance, Indiana  Department of Health, and the Family and Social Services Administration, to ensure a  coordinated approach to addressing the challenges faced by patients with rare diseases. 

One of the key initiatives undertaken by the RDAC was the development of a survey  instrument designed to gather data on the specific challenges encountered by rare disease  patients and their families. Additionally, the Council formed various subcommittees, one of  which is focused on pediatric cancer research, to delve deeper into specialized areas of concern.  The RDAC also engaged in public advocacy, exemplified by its issuance of a letter in support of  continued funding for paid family caregivers of medically fragile children. 

Among the recommendations put forward by the RDAC are several measures aimed at  strengthening healthcare support for pediatric cancer patients. These include increasing funding  for innovative research and treatments for pediatric cancers, fostering collaborations between  research institutions and the Indiana Biosciences Research Institute (IBRI), and establishing a  Pediatric Oncology Working Group composed of specialists to provide recommendations on  treatments and insurance coverage. The Council also advocates for reforms in prior authorization  and peer review policies by mandating that insurance companies utilize pediatric subspecialists  as peer reviewers for rare disease cases. Additionally, it recommends that each pediatric cancer  center be assigned a state-subsidized learning specialist or school liaison to aid in the  reintegration of children into school settings and calls for insurance coverage for fertility 

preservation procedures for pediatric cancer patients. To ensure the long-term sustainability of its  initiatives, the RDAC is seeking continued state support and funding for dedicated staff. 

The report further highlights Indiana’s status in pediatric cancer research, noting the  significant strengths of institutions such as the Indiana University School of Medicine (IUSM)  and Riley Hospital for Children. Indiana is positioning itself as a national leader in pediatric  oncology research, with ongoing projects at the Indiana Biosciences Research Institute (IBRI)  and active partnerships with Notre Dame University and Purdue University. This underscores the  state’s commitment to advancing healthcare for children affected by cancer. 

What new information did you learn by reading this Agency Report? This response should  be at least a paragraph. 

One key takeaway is that pediatric cancer research is significantly underfunded compared  to adult cancers. The report states that only 4% of federal cancer research funding is allocated to  childhood cancers, despite the significant long-term health impacts on survivors. Additionally, I  learned that insurance barriers, such as prior authorizations, delay life-saving treatments and  potentially affect patient outcomes. Another insight is the multidisciplinary approach required for  rare disease treatment. The RDAC comprises physicians, researchers, patient advocates, insurers,  and policymakers who are all working collaboratively to improve healthcare delivery. The report’s  emphasis on collaborations between academic institutions and biotech companies also highlights  Indiana’s potential to lead innovative drug development in the future. Another new insight is  the multidisciplinary approach required for rare disease treatment. The RDAC  includes physicians, researchers, patient advocates, insurers, and policymakers, all working to 

improve healthcare delivery. The focus on collaborations between academic institutions and  biotech companies highlights Indiana’s potential to lead innovative drug development. 

How did the Report help you have a different perspective on the specific policy issue about  which the Report was written? This response should be at least a paragraph. 

The report broadened my perspective on rare disease healthcare by highlighting the  

multifaceted efforts undertaken by the RDAC. I was previously unaware of the extensive impact  the Council has on shaping healthcare policies in Indiana and addressing the complex challenges  faced by rare disease patients. By detailing the collaborative work with various state agencies  and the formation of specialized subcommittees, the report illustrated the comprehensive  approach needed to tackle such intricate issues. Learning about the specific recommendations,  such as reforms in insurance practices and the establishment of dedicated pediatric oncology  groups, gave me hope for meaningful improvements in healthcare. It also made me appreciate  the critical role of coordinated policy efforts in advancing healthcare services, research, and  patient support. 

State some piece of information that is not in the Report that you believe would have made  the Report more informative or persuasive. 

While the report is comprehensive in many respects, it would have been more  informative with the inclusion of a discussion on “Making Healthcare a Right in Indiana.” This  topic, though challenging and not easily resolved within a short timeframe, could provide a  broader context for the RDAC’s recommendations by linking them to a larger vision of universal  healthcare access. Addressing this issue might help underscore the importance of sustainable,  long-term healthcare reforms. Such a discussion would add an extra layer of depth to the report 

and make the case for transformative changes in the state's healthcare policies even more  persuasive. 

Reference 

Indiana General Assembly. (2024). Rare Disease Advisory Council Annual Report. Retrieved  from https://iga.in.gov/publications/agency_report/Rare%20Disease%20Advisory%20Council% 20Annual%20Report.pdf