RDLA Webinar: The HEART Act, Newborn Screening, and More

The Rare Disease Legislative Advocates (RDLA) webinar for the month of June took place on June 17, 2021. This month’s webinar provided updates on a variety of legislative topics that are relevant to the rare disease community, such as The HEART Act, newborn screening legislation, the Medical Nutrition Equity Act, Accelerating Kids’ Access to Care Act, and The Access to Rare Indications Act.


Melissa Goetz from The FCS Foundation provided the latest updates on this bill, which was last introduced in February by reps Paul Tonko (D-NY) and David McKinley (R-WV). Recently, the US Food and Drug Administration (FDA) has announced technical assistance for the legislation. Provisions of The HEART Act include:

  1. Advisory committees must include rare disease experts in order to review data on a rare disease drug.
  2. Risk Evaluation and Mitigation Strategies (REMS) need to include input from rare disease patients.
  3. In general, rare disease expertise needs to play a role in drug evaluation throughout the process.
  4. Annual report of rare disease therapy applications each division from the FDA sees.
  5. Calls for Government Accountability Office (GAO) study to evaluate differences in the EU approval process for rare disease.

The components of this legislation draw from painful experiences that the familial chylomicronemia syndrome (FCS) patient community has had when trying to get a drug approved by the FDA. The FCS Foundation felt that they lost critical communication with the agency in the late stages of the drug review. However, this legislation is intended to benefit the entire rare disease community. Click here to contact your representatives to voice your support.

Newborn Screenings

Dylan Simon from the EveryLife Foundation for Rare Diseases spoke next about new legislation in the works for newborn screenings. While there is a federal panel of recommended newborn screenings that states are expected to follow, which diseases and conditions are screened for ultimately falls to the states to decide. This, unfortunately, means that there are substantial differences in screenings between them.

The federal guidelines are called Recommended Uniform Screening Panel (RUSP). In some states, RUSP alignment legislation is gaining headway. These laws would require the state to screen for any and all diseases listed on the federal RUSP. It would also speed up the time limit that states must meet to add new diseases for screening.

RUSP alignment recently became law in Georgia and similar bills are being considered in North Carolina and Ohio.

The Medical Nutrition Equity Act

Kylie Barber from the National PKU Alliance spoke next about the Medical Nutrition Equity Act (MNEA) 2021. This bicameral and bipartisan bill was introduced earlier this month. The sponsors are hoping it can be tacked on to a larger package.

The law is intended to provide coverage in federal programs and private insurance for medically necessary foods, amino acids, vitamins, and any needed equipment for their use. This is important for PKU and certain other disorders. Medically necessary foods include modified fats, nutritional formulas, low protein modified products, and amino acid preparations that are prescribed by doctors.

Accelerating Kids’ Access to Care Act

Nicholas Manetto from Faegre Drinker Consulting spoke next about the Accelerating Kids’ Access to Care Act. This bill would help kids receiving coverage on CHIP or Medicaid receive out-of-state care in order to address complex or rare conditions. While this is currently possible, the process is slow and bureaucratic.

Patients eligible would include children and adults whose condition originated in childhood. There have been signs of support from both political parties and the Biden administration.

Access to Rare Indications Act

Marc Yale from the International Pemphigus & Pemphigoid Foundation (IPPF) discussed another proposed bill called the Access to Rare Indications Act. Patients with rare conditions often find it more difficult to find insurers that will cover the cost of their medicines, which are often very expensive.

Since only a small number of rare diseases have FDA approved therapies, a lot of treatment takes place off-label (meaning a drug is used to treat a disease it has not been officially approved for). Getting coverage for off-label use is often an uphill battle. Provisions of the legislation include:

  1. Removing statutory prohibitions that prevent off-label drug use coverage.
  2. Expanding the definition of what is considered ‘medically acceptable’ usage.
  3. Expediting appeals for using drugs to treat rare diseases.
  4. Identifying resources to circumvent coverage denials or step therapy.

The bill is expected to be reintroduced after July 4th. Click here to contact your representatives to voice your support.

There’s a lot of potential laws being floated around the country that could help make lives for patients a little bit easier. Check out the recordings of the RDLA webinars to learn more.

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