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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.

21st Century Cures 2.0 Act

21st Century Cures 2.0 Act H.R. 6000 builds on the successes of the 21st Century Cures Act, passed in 2016, to advance biomedical research, regulatory science, public health, and payment policy innovation so critical for rare disease patients and families.  The draft builds on the framework of the Cures Act and aims to further modernize the nation’s healthcare pipeline in the hopes of avoiding some of the burdens that the system has faced during the COVID-19 pandemic. Some of the proposed areas for policy include public health and pandemic preparedness, healthcare delivery systems, patient engagement in healthcare decision-making, caregiver integration into the care team, modernizing CMS, and increasing diversity in clinical trials.

Additional resources available on RDLA website here
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ABLE Age Adjustment Act 

The ABLE Age Adjustment Act H.R.1219/ S. 331, increases from 26 to 46 the age threshold for tax-favored ABLE (Achieving a Better Life Experience) accounts. (ABLE accounts are designed to enable individuals with disabilities to save for and pay for disability-related expenses. To establish an account, an individual must have a qualifying impairment that began before the individual attained the age threshold.)

Accelerating Kids’ Access to Care Act

The Accelerating Kids’ Access to Care Act HR 3089/S 1544 requires 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. 2144/ S. 1450 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

Access to Rare Indications Act

The Access to Rare Indications Act H.R.6160 will allow Medicare and Medicaid to use sources including compendia, peer-reviewed literature, and the expertise of rare disease specialists to validate that a prescribed therapy meets the medically accepted criteria used to determine insurance coverage for a rare disease patient. The Access to Rare Indications Act will work to ensure that the 1 in 10 Americans who live with rare diseases have the intended benefit of health coverage and can access evidence-based standards of care for their conditions.

This bill recognizes that over 90% of the 7,000 known rare diseases have no FDA-approved treatment, due to a lack of research in the rare disease space, and challenges in finding enough patients to conduct clinical trials. This leaves many rare and ultra-rare disease patients facing serious and life-threatening illness to rely on off-label use of drugs approved by the FDA for more common conditions. To address this problem, the Access to Rare Indications Act aligns coverage in Medicare and Medicaid for off-label rare disease treatment with Medicare’s existing system used to determine coverage for off-label cancer treatment. Additionally, this bill requires private payers to create an expedited review pathway for formulary exception, reconsideration, and/or appeal of any denial of coverage for a drug or biological prescribed for a patient with a rare disorder.

Take Action here
Haystack Project one page explanation here
Patient Advocacy Group letter here

Advanced Research Project Agency–Health (APRA-H) Act

ARPA-H Act H.R. 5585 would establish the new entity within the Department of Health and Human Services (HHS), but not specifically within the National Institutes of Health (NIH), as the White House has proposed. Both the House-passed and draft Senate FY 2022 spending bills include funds to establish ARPA-H within the NIH, although the Senate bill’s explanatory statement notes the committee “remains open” to establishing ARPA-H as a free-standing agency outside of the NIH. This legislation:

Would authorize the creation of a new fund to support ARPA-H

Would require a budget request from the president in future years that would be separate from the rest of the HHS budget

Emphasizes priority of funding to support proposals that would address a disproportionate disease burden and promote health equity

Would establish the ARPA-H director as a presidential appointee with a five-year term

Would establish an advisory committee made up of federal officials from other health and research agencies with appropriate subject matter expertise


Better Empowerment Now to Enhance Framework and Improve Treatments (BENEFIT) Act S. 373/H.R. 4472 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
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BENEFIT Act Policy Primer Video

Diversifying Investigations Via Equitable Research Studies for Everyone (DIVERSE) Trials Act 

The DIVERSE Trials Act H.R. 5030/ S. 2706 requires activities to increase diversity in clinical trials. It also expands reporting by laboratories that test for and diagnose COVID-19 and requires a study on data collection and data sharing during public health emergencies.

Specifically, the Food and Drug Administration (FDA) must issue guidance on decentralized clinical trials to promote meaningful demographic and geographic diversity in patient engagement, enrollment, and participation. Decentralized clinical trials include those executed through telemedicine or other digital technologies to allow for the remote collection and assessment of clinical trial data. The FDA may also work with foreign regulators to harmonize international regulations governing decentralized clinical trials and the use of digital health technology.

In addition, the Department of Health and Human Services (HHS) may support community education, outreach, and recruitment activities for clinical trials of treatments for conditions that disproportionately impact populations underrepresented in clinical trials.

The bill also specifies that drug or device manufacturers may provide, subject to some limits, free digital health technologies and other remuneration to patients in approved clinical trials without violating laws that address fraud and abuse in federal programs.

Furthermore, laboratories that test for and diagnose COVID-19 must report additional demographic data, including information about social determinants of health. HHS must also contract with the National Academy of Medicine to study and propose a design for an interoperable platform to facilitate data sharing during public health emergencies.

Ending the Diagnostic Odyssey Act

The Ending the Diagnostic Odyssey Act S. 2022 allows state Medicaid programs to cover whole genome sequencing services for certain individuals.

Specifically, states may cover such services for individuals under the age of 21 (or a lower age, if the state chooses) and for former foster youth under the age of 26 who (1) have been referred or admitted to an intensive care unit or seen by a medical specialist for a suspected genetic or undiagnosed disease, or (2) are suspected by a medical specialist to have a neonatal- or pediatric-onset genetic disease.

The Centers for Medicare & Medicaid Services may award grants to assist states in developing plans to cover such services.

The HELP Act

The Help Ensure Lower Patient (HELP) Copays Act, H.R. 5801, 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
Take Action. Source:  National Hemophilia Foundation 

Helping Experts Accelerate Rare Treatments (HEART) Act

Helping Experts Accelerate Rare Treatments (HEART) Act H.R. 1184 will ensure that FDA is transparent and open when it reviews medications to treat rare disorders. It will require FDA to publish an annual report summarizing its actions related to designating and reviewing drugs for rare diseases. Further, it will require the use of outside experts, including patient advocates and experts in small population trial design when reviewing these products. The HEART Act calls for these simple and common-sense changes:

  • The FDA must include members of its own Rare Disease Program staff in reviews for drugs to treat rare diseases.
  • FDA must include experts in rare diseases on Advisory Committee panels for rare disease drugs.
  • Each year the FDA must prepare a report showing how many rare disease drug applications were reviewed by each division at the Agency.
  • Members of the patient community must be consulted when devising or reviewing a Risk Evaluation and Mitigation Strategy (REMS) for a rare disease drug.
  • We should review the EU process for approval of rare disease drugs including the use of data from open-label extension studies.

Haystack Press Release here
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Sponsor Rep Paul Tonko webinar about the HEART Act which he introduced to the House on 18 Feb 2021 here
EveryLife Foundation Accelerated Approval resource page here 
Partnership to Fight Chronic Disease Accelerated Approval resource page here

Newborn Screening Saves Lives Reauthorization Act

The Newborn Screening Saves Lives Reauthorization Act, H.R. 482/S. 350 was passed by Congress on June 23rd, 2021 and the Senate legislation is still pending.  

This bill reauthorizes the Health Resources and Services Administration (HRSA) state grants to expand and improve screening programs, provide educational resources to parents and health care providers, and improve follow-up care for infants with a detected condition.  It reauthorizes the Secretary’s Advisory Committee on Heritable Disorders in Newborns and Children which advises the Secretary of Health and Human Services on newborn and childhood screening policies and priorities to enhance state health agencies to ensure screening is available to every eligible infant. Includes the evidence-based federal Recommended Uniform Screening Panel (RUSP).

The bill also aligns statutory requirements for research on non-identified newborn blood spots with federal regulations governing research on human subjects. These blood spots are collected as part of newborn screening programs, and some states make them available for biomedical and public health research. Under current regulations, research on non-identified bio-specimens such as blood spots is not considered to be research on human subjects, to which additional protections apply.

Learn more...
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Orphan Drug Tax Credit Changes

On 19 Nov 2021 the House-passed Build Back Better Act and the current Senate Finance Committee draft contains language that will negatively impact the rare disease community. Proposed changes to the Orphan Drug Tax Credit (ODTC) to limit the credit to the first approval would fundamentally alter the rare disease pipeline and discourage companies from pursuing further clinical programs to test approved therapies in new disease areas. Investment in further research improves the lives of the most vulnerable rare disease patients and offers clinicians and insurers the evidence needed to guide treatment recommendations. 

Our patient communities need more options, more hope, and more investment in rare disease research. The proposed change to the Orphan Drug Tax Credit will not result in any benefit to patients and stands to cause irreparable harm to the progress and innovation in rare disease therapeutic development. 

ODTC Explanation on RDLA Hot Legislative Topics video (start time 24:48)
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Prescription Drug User Fee Act (PDUFA VII)

The FDA released its proposed recommendations for the Prescription Drug User Fee Act VII (PDUFA VII) which included several rare disease priorities including, but not limited to, the establishment of a Rare Disease Endpoint Advancement (RDEA) pilot program, advancing the use of Real-World Evidence (RWE), advancing momentum around Patient Focused Drug Development, with a specific emphasis on understanding patient perspectives in gene and cell-based therapy products, enhancing capacity to review complex or novel clinical trial designs, and enhancing reviews of cell and gene therapy products.

RDLA recommendations here
RDLA Policy Primer here
Take Action

The STAT Act

The Speeding Therapy Access Today (STAT) Act, H.R. 1730/S. 670, is a bipartisan bill that requires and authorizes various actions to accelerate the development of therapies for rare diseases. It was created with the input of the rare disease community aimed at improving the development of and access to therapies for the rare disease community. The centerpiece of the STAT Act is the creation of a Rare Disease Center of Excellence at the US Food and Drug Administration. The STAT Act will:

  • Accelerate rare disease therapy development,
  • Optimize interagency coordination,
  • Advance science-based regulatory policies, and
  • Facilitate access to therapies.

Stay up to date about the STAT Act through EveryLife Foundation website here
Take Action 
STAT Act Policy Primer Video

Telehealth Modernization Act

The Telehealth Modernization Act H.R. 1332/ S. 368 modifies requirements relating to coverage of telehealth services under Medicare.

Specifically, the bill extends certain flexibilities that were initially authorized during the public health emergency relating to COVID-19 (i.e., coronavirus disease 2019). Among other things, the bill allows (1) rural health clinics and federally qualified health centers to serve as the distant site (i.e., the location of the health care practitioner); (2) the home of a beneficiary to serve as the originating site (i.e., the location of the beneficiary) for all services (rather than for only certain services); and (3) all types of practitioners to furnish telehealth services, as determined by the Centers for Medicare & Medicaid Services.


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