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Cure Sanfilippo lends support to multiple provisions of Cures 2.0 Act

July 16, 2021

The 21st Century Cures 2.0 Act legislation from U.S. Representatives Diana DeGette (D-Colorado) and Fred Upton (R-Michigan) could have a profound impact on the speed of therapy development and diagnostic access for patients with rare diseases.

“Many organizations have been monitoring the legislation and rallying the patient advocate community about how to make sure our voices are heard as this legislation is taking shape,” said Foundation President Glenn O’Neill. “We appreciate and are proud to lend the Foundation’s support to their advocacy efforts to create change that benefits all patients with rare diseases.”

Creation of Neuroscience Center of Excellence within FDA

Earlier this year, Cure Sanfilippo Foundation joined more than 95 other organization and industry partners in signing on to a letter from the American Brain Coalition asking U.S. Reps. DeGette and Upton to include language in the Cures 2.0 legislation establishing a Neuroscience Center of Excellence at the Food and Drug Administration (FDA).

As a result of the collective advocacy, U.S. Reps. DeGette and Upton included language requiring the FDA to create a new center of excellence focusing on high prevalence and burdensome diseases, like those affecting the brain and central nervous system, in their June 22 discussion draft of 21st Century Cures 2.0 legislation that  closely mirrors the ABC proposal.

Cure Sanfilippo is joining the ABC’s new sign-on letter emphasizing support for the new language in the Cures 2.0 draft.

Improved diagnostic services to shorten the “Diagnostic Odyssey”

Cure Sanfilippo has also joined a letter led by the Genetic Alliance, regarding language in the Cures 2.0 legislation to expand access to diagnostic services for children who are suspected of having rare genetic disorders.

The letter asks for two changes: 1) Allow any interested and eligible state to participate in the demonstration program; and 2) Focus the demonstration program on the newest diagnostic modalities: next-generation sequencing (NGS) clinical services.

The letter points out that a demonstration program to evaluate and demonstrate the value of single-gene testing or genetic panel tests is unnecessary because their value is already understood by public and private payers and are widely covered.

“However, many children suffer from undiagnosed neonatal-onset or pediatric-onset diseases, the causes of which are unknown. In a world with more than 6,000 known genetic disorders, and new disorders being discovered on a regular basis, access to the newest next-generation sequencing (NGS) diagnostic tests can mean the difference between receiving an answer in days or weeks, or never receiving an answer at all,” states the sign-on letter.

Focusing a demonstration project on next-generation sequencing clinical services would build a robust body of evidence on how next-generation sequencing delivers value in our health care system and delivers more timely and comprehensive answers to patients and families in desperate need.

Earlier support for Cures 2.0 legislation

Cure Sanfilippo also joined a the Genetic Alliance’s June 2020 letter to U.S. Reps. DeGette and Upton, asking that states be able to conduct a pilot program to increase the Federal Medical Assistance Percentage rate to provide Whole Genome Sequencing clinical services for children on Medicaid with a disease that is suspected to have a genetic cause.

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