Rare Disease Policy
If you are interested in participating in advocacy training, check out the EveryLife Foundation advocacy toolkit here or consider joining Rare Disease Legislative Advocates (RDLA) Young Adult Representatives (YARR). Learn more about YARR here.
Federal Level Advocacy
- Legislative Scorecard
- Check out your legislators score for insights on your legislator’s voting record on rare disease legislation here
- Find your Congressional Representatives here
- Find your US Senators here
State Level Advocacy
- The RareAction Network ® (RAN) here
- Rare Disease Advisory Council (RDAC) here
- Find your State Legislators here
Legislation to Watch
People with Barth syndrome and their families are the experts about the impact of living with this condition. Your voice matters! Your journey gives you the power to shape public policy to improve the lives of people living with Barth syndrome. Below you will find legislation that is important to our rare disease community.
Accelerating Kids’ Access to Care Act
The Accelerating Kids’ Access to Care Act HR 4758/S 2372requires state Medicaid programs to establish a process through which qualifying out-of-state providers may enroll as participating providers without undergoing additional screening requirements. Among other things, a qualifying provider must (1) serve minors, or adults whose condition began as a minor; and (2) have previously been screened for Medicare participation or for participation in the Medicaid program of the state in which the provider is located.
Access to Genetic Counselor Services Act
Access to Genetic Counselor Services Act H.R. 3876/ S. 2323 is proposed to address the significant barriers Medicare beneficiaries encounter in accessing genetic counselor services, and these barriers worsened during the COVID-19 pandemic. Recent evidence also finds disparities to accessing genetic counseling services based on social determinants of health and geographic location. This bill provides for coverage under Medicare of genetic counseling services that are furnished by genetic counselors. Covered services include those services, as well as incidental services and supplies, that would otherwise be covered under Medicare if provided by a physician.
Stay up to date about the Access to Genetic Counselor Services on National Society of Genetic Counselors website here
Access to Genetic Counselor Services Act Policy Primer Video
The BENEFIT Act
Better Empowerment Now to Enhance Framework and Improve Treatments (BENEFIT) Act S. 526/H.R. 1092 requires the Food and Drug Administration (FDA) to consider relevant patient-focused drug development data, such as data from patient preference studies and patient-reported outcome data, in the risk-benefit assessment framework used in the process for approving new drugs.
After a new drug application has been approved, the FDA's public statement about how it used patient experience data shall include a description of how such data was considered in the risk-benefit assessment framework. This action would send an important signal to all stakeholders that patient experience and PFDD data will be fully incorporated into the agency’s review process and will encourage such entities to develop scientifically rigorous and meaningful tools and data.
Parent Project Muscular Dystrophy one page explanation here
Take Action
BENEFIT Act Policy Primer Video
Creating Hope Reauthorization Act of 2024
The Creating Hope Reauthorization Act (H.R. 7384/ S. 4583) reauthorizes the Rare Pediatric Disease Priority Review Voucher (PRV) program for six years, ensuring this vital incentive that has no cost to taxpayers can continue to spur innovation in rare diseases that disproportionately affect children.
The proposed legislation aims to amend the Federal Food, Drug, and Cosmetic Act to extend the authority of the Secretary of Health and Human Services to issue priority review vouchers, which are designed to incentivize the development of treatments for rare pediatric diseases. Specifically, the bill modifies Section 529(b)(5) of the Act by extending the expiration date for issuing these vouchers from September 30, 2024, to September 30, 2028. Additionally, it extends the deadline for the submission of applications that can qualify for these vouchers from September 30, 2026, to September 30, 2030. These changes are intended to provide a longer timeframe for pharmaceutical companies to develop and seek approval for treatments targeting rare pediatric conditions, thereby encouraging continued investment and innovation in this critical area of public health.
Program now set to expire on December 20, 2024
Ask your Senator to cosponsor here
EveryLife Foundation Creating Hope Reauthorization Act: Why PRV is important to people with rare diseases here
EveryLife Foundation Creating Hope Reauthorization Act: One pager here
Health Equity and Accountability Act of 2024
The Health Equity and Accountability Act H.R.9161 /S.4773 is a comprehensive initiative to advance health equity by improving data collection, amending laws, and funding culturally and linguistically appropriate healthcare services to address disparities and improve outcomes for minority groups, with additional provisions for nutrition assistance, school meals, and clinical trial diversity.
The HELP Act
The Help Ensure Lower Patient (HELP) Copays Act, S. 1375/ H.R. 830, is a two-part solution bill that requires health insurance plans to apply certain payments made by, or on behalf of, a plan enrollee toward a plan's cost-sharing requirements. Specifically, plans must apply third-party payments, financial assistance, discounts, product vouchers, and other reductions in out-of-pocket expenses toward the requirements. The HELP Copays Act clarifies the ACA definition of cost sharing to ensure payments made “by or on behalf of” patients count towards their deductible and/or out-of-pocket maximum. It closes the EHB loophole to ensure that any item or service covered by a health plan is considered part of their EHB package and thus cost sharing for these must be counted towards patients’ annual cost sharing limits.
HELP Copays Act One Pager
ORPHAN Cures Act
The "Optimizing Research Progress Hope And New Cures Act" or "ORPHAN Cures Act" S. 3131/ H.R. 5579 proposes amendments to title XI of the Social Security Act to enhance and clarify the exemption of orphan drugs from the Drug Price Negotiation Program. Specifically, it amends Section 1191(e) to exclude any period during which a drug was considered an orphan drug in calculating its approval time for price negotiation purposes. Additionally, it broadens the definition of orphan drugs to include those treating one or more rare diseases or conditions, rather than only one.
Patient Access Act of 2024
The bill H.R. 9184 amends the Social Security Act to allow drug manufacturers to cover travel and lodging costs for patients under specific conditions, exempting these arrangements from antikickback sanctions, and requires a GAO report on the impact and oversight of such arrangements. The proposed legislation seeks to amend Title XI of the Social Security Act to allow certain travel and lodging arrangements between drug manufacturers and individuals prescribed those drugs to be exempt from antikickback and other sanctions. The amendment introduces a new subsection to Section 1128B, detailing the conditions under which these arrangements are permissible. These conditions include the provision of payment or reimbursement by the manufacturer for transportation, lodging, meals, or other incidental expenses related to the administration of the drug, provided the individual does not have access to these services at no cost from the administering facility. The arrangement must not be advertised, and the manufacturer must ensure that the individual is informed only after the drug is prescribed. The travel and lodging must be practical, convenient, and reasonable, with costs not exceeding the per diem rate established by the General Services Administration. Eligibility for these arrangements is limited to individuals with income not exceeding the median income for their region and who live more than an hour's drive or 50 miles from the nearest facility administering the drug. The manufacturer must offer these arrangements regardless of the facility or the individual's insurance status and must not seek reimbursement from federal health care programs. Additionally, the legislation mandates a study by the Government Accountability Office (GAO) to be submitted to Congress within three years of enactment. This report will analyze the impact of these arrangements on access to therapies, socioeconomic disparities, utilization of biosimilars and generics, tax benefits for manufacturers, and the efficacy of oversight to prevent waste, fraud, and abuse.
Pediatricians Accelerate Childhood Therapies Act of 2023
The Pediatricians Accelerate Childhood Therapies Act of 2023 H.R. 4714/ S. 2345 is designed to amend the Public Health Service Act, enabling the Director of the National Institutes of Health (NIH) to provide awards to outstanding early-career scientists, including physician-scientists, in pediatric research. This research spans basic, clinical, translational, and pediatric pharmacological areas. The Act emphasizes support for researchers from historically underrepresented groups and institutions that enhance diversity in research and higher education. It identifies key pediatric research priorities such as mental and behavioral health, childhood cancer, precision medicine, rare diseases, and fetal development. The Act ensures that awards will augment existing pediatric research funds, covering research activities, mentorship, and training to promote research independence. It also establishes the NIH Pediatric Research Consortium, led by the Director of the Eunice Kennedy Shriver National Institute of Child Health and Human Development, to coordinate pediatric research across NIH institutes and centers, identify research gaps and opportunities, and foster the next generation of pediatric researchers. The Consortium will consult with external pediatric research experts and report biennially to Congress on research projects, strategic initiatives, career development awards, and demographic data of awardees to ensure broad representation and impact in pediatric research.
Promising Pathways Act 2.0
Promising Pathways Act 2.0 S. 4426 establishes a conditional approval pathway for drugs treating rare, progressive, and serious diseases, with provisions for priority review, real-world evidence use, mandatory observational registries, and insurance coverage without cost-sharing, while integrating these changes into existing health care laws and setting specific timelines for implementation and reporting.
The legislative text introduces amendments to the Federal Food, Drug, and Cosmetic Act to establish a conditional approval pathway for certain drugs and biological products intended for individuals with rare, progressive, and serious diseases. The primary objective is to expedite the availability of treatments for conditions with substantial unmet medical needs. The legislation outlines a process where drug sponsors can apply for conditional approval, which includes priority review and the potential for other designations and benefits, such as tax credits and fee waivers. Drugs eligible for this pathway must treat rapidly progressive, terminal diseases or rare conditions that significantly impact lifespan or quality of life. The approval process requires evidence of safety and preliminary effectiveness, with provisions for rolling, real-time review and the use of real-world evidence.
The legislation mandates the establishment of observational registries for patients treated with conditionally approved drugs, ensuring data collection on treatment outcomes and side effects. Conditional approval is granted for two years, with the possibility of up to three renewals, not exceeding a total of eight years. The Secretary of Health and Human Services has the authority to withdraw conditional approval based on safety data, fraudulent application information, or ineligibility. The bill also includes provisions for labeling, informed consent, and the transition to full approval under existing drug approval statutes.
Funding and reimbursement provisions are included, requiring private health insurers and federal health care programs to cover conditionally approved drugs without imposing cost-sharing requirements. The bill also amends existing laws to integrate the new conditional approval pathway, ensuring that conditionally approved drugs are recognized and reimbursed under various health care programs.
The legislation sets specific timelines for the establishment of processes and standards, such as the approval of observational registries, data submission standards, and the appeals process for withdrawal of conditional approval. Additionally, the Secretary is required to submit biennial reports to Congress on the status and impact of conditionally approved drugs, including patient treatment data and registry information.
Note this is an amended version of Promising Pathways H.R. 4408 / S. 1906
RARE Act
The RARE Act, H.R. 7383/ S. 1214 modifies the Federal Food, Drug, and Cosmetic Act to limit drug exclusivity to specific approved uses or indications within rare diseases, rather than the entire disease category.
The proposed legislation aims to amend the Federal Food, Drug, and Cosmetic Act to impose specific limitations on the exclusive approval or licensure of drugs designated for rare diseases or conditions. The primary objective is to refine the criteria for exclusivity by specifying that it applies to the same approved use or indication within a rare disease or condition, rather than the broader category of the same disease or condition. This change is reflected in several modifications to Section 527 of the Act, including adjustments to subsections (a), (b), and (c)(1), where the language is altered to focus on the "same approved use or indication" rather than the "same rare disease or condition." Additionally, a new subsection (f) is added to define "approved use or indication" as the use or indication approved under section 505 of the Act or licensed under section 351 of the Public Health Service Act for a drug designated for a rare disease or condition. These amendments are applicable to any drug designated under section 526 of the Federal Food, Drug, and Cosmetic Act, irrespective of the designation or approval date.
Safe Step Act
The Safe Step Act, H.R 2630/S. 652 seeks to modify the Employee Retirement Income Security Act of 1974, introducing a requirement for group health plans or health insurance issuers with step therapy protocols to establish a transparent and accessible process for exceptions. These protocols currently require patients to try and fail preferred medications before receiving coverage for a prescribed alternative. The bill outlines a clear exceptions process, including criteria such as treatment ineffectiveness, potential severe consequences, contraindications, adverse reactions, or the need to maintain medication stability. It details the procedure for submitting exceptions, evaluation criteria, required documentation, and provisions for expedited review in urgent cases. Information on the exceptions process must be easily accessible online, and if an exception request meets the criteria, coverage for the requested medication is mandated under the plan's terms, with decisions made within 72 hours for standard requests and 24 hours for expedited requests. The legislation also specifies its applicability to any group health plan or health insurance coverage that includes a medication step therapy protocol and makes a clerical amendment to reflect this requirement. Additionally, it mandates that these changes apply to plan years beginning at least 6 months after the Act's enactment, with the Secretary of Labor required to issue final regulations within 6 months of enactment to implement these provisions.
