The Rare Disease Legislative Advocates (RDLA) held a webinar on December 3rd, 2020. This event focused on the organization’s goal of passing Cures 2.0, a bill that would help further improve national infrastructure surrounding medical research and enhance rare disease patient engagement and drug development for rare disease. Cures 2.0 is meant to be a successor legislation to the 21st Century Cures Act, which was signed into law in the final remaining weeks of 2016 under the Obama administration.
Annie Kennedy, the Everylife Foundation’s Chief of Policy and Advocacy, says that the 21st Century Cures Act has played a critical role in advancing the development of new rare disease treatments at a faster pace. These enhancements have come in the form of increased funding among others, but a major concern is that many of the latest and most advanced therapies that have been developed since the passage of the law have been extremely expensive, and in fact include the most expensive medicines in the world. This has caused problems with access, and Cures 2.0 aims to help mitigate this issue.
This may look like investigating new models for payment and establishing a fleshed out legal framework for them.
Lisa Schill, VP of the RASopathies Network, says that 21st Century Cures helped improve the FDA’s ability to support rare disease drug development. Unfortunately, no rare disease-related legislation has passed at the federal level since.
Jennifer Dexter of the National Health Council outlined several goals of Cures 2.0. Some of these include:
- Prioritizing the patient voice/perspective
- Improving sustainability, affordability, and equity
- Enhancing digital health
- Covering the newest treatments
- Improving inclusion for patient families and caregivers
Data from patients will be critical to lowering costs, removing access barriers, and personalizing care.
Robert Falb of the Alliance for Regenerative Medicine says that Cures 2.0 also should help improve the playing field for regenerative medicines, of which only four or five have been FDA approved so far. Regenerative medicines include gene therapies, cell therapies, and tissue therapies, which are often developed with the intent to cure a given disease. Cures 2.0 also includes improvements to medicine manufacturing and newborn screenings.
The Everylife Foundation for Rare Diseases has also called for several other provisions including:
- Guaranteed access to whole genome sequencing and other sequencing procedures to those living with undiagnosed rare genetic conditions.
- Establishing a Rare Disease Center of Excellence at the FDA.
- Reducing delays to access following a drug’s approval.
- Establish a clearer framework for the tailored evaluation of rare disease treatments.
- Ensuring the broadest possible inclusion criteria in trials while accounting for scientific and clinical integrity.
