The second iteration of the 21st Century Cures discussion draft comes with some added attractions – most specifically a bi-partisan authorship.
Hill chatter made it clear that Rep. Frank Pallone was holding back any “D” support until the draft called for additional NIH funding. And, lo and behold, that language is now Title I -- front and center. Whether or not that’s a boondoggle is another discussion (for another time).
As to the actual updated discussion draft, it’s better than its predecessor in many ways, not the least of which is its recognition of the FDA as part of the solution rather than part of the problem -- and that real world data should play a role in informing agency decisions. Efficacy and effectiveness.
Some items of particular interest:
Section 1121: Clinical Trial Data System
This section would create a third party scientific research sharing system for trials solely funded by the federal government in order to allow the use and analysis of data beyond each individual research project.
Section 1141: Council for 21st Century Cures
This section would establish a public-private partnership to accelerate the discovery, development, and delivery in the United States of innovative cures, treatments, and preventive measures for patients.
Section 2001: Development and Use of Patient-Experience Data to Enhance Structured Risk-Benefit Assessment Framework
Because no one understands a particular condition or disease better than patients living with it, this section would require FDA to establish a structured framework for the meaningful incorporation of patient experience data into the regulatory decision-making process, including the assessment of desired benefits and tolerable risks associated with new treatments.
Section 2062: Utilizing Evidence From Clinical Experience
This section would require FDA to establish a program to evaluate the potential use of evidence from clinical experience to help support the approval of a new indication for a drug and to help support or satisfy post-approval study requirements. In parallel, FDA would identify and execute pilot demonstrations to extend existing use of the Sentinel System to support these efforts.
Section 2082-2083: Expanded Access
This sectionwould place transparency requirements on certain drug companies regarding their expanded access programs (programs for patients to access drugs before they are approved) and require FDA to finalize guidance regarding how it interprets and uses adverse drug event data resulting from drug use under such expanded access programs.
Section 2101: Facilitating Dissemination of Healthcare Economic Information
This section would add clarity and facilitate dissemination of healthcare economic information, as defined in the section, to payers, formulary committees, or other similar entities.
Section 2263: Reagan Udall Foundation
This section would ensure that the Reagan Udall Foundation has access to the expertise and human capital it needs to fulfill its statutory mission of advancing FDA’s scientific priorities.
The full draft can be found here.
The section-by-section draft can be found here.
And the one-pager here.
Many items of interest – and the devil is in the details. Stay tuned.