21st Century Cures II -- Frank and Earnest

  • by: |
  • 04/29/2015

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.

CMPI

Center for Medicine in the Public Interest is a nonprofit, non-partisan organization promoting innovative solutions that advance medical progress, reduce health disparities, extend life and make health care more affordable, preventive and patient-centered. CMPI also provides the public, policymakers and the media a reliable source of independent scientific analysis on issues ranging from personalized medicine, food and drug safety, health care reform and comparative effectiveness.

Blog Roll

Alliance for Patient Access Alternative Health Practice
AHRP
Better Health
BigGovHealth
Biotech Blog
BrandweekNRX
CA Medicine man
Cafe Pharma
Campaign for Modern Medicines
Carlat Psychiatry Blog
Clinical Psychology and Psychiatry: A Closer Look
Conservative's Forum
Club For Growth
CNEhealth.org
Diabetes Mine
Disruptive Women
Doctors For Patient Care
Dr. Gov
Drug Channels
DTC Perspectives
eDrugSearch
Envisioning 2.0
EyeOnFDA
FDA Law Blog
Fierce Pharma
fightingdiseases.org
Fresh Air Fund
Furious Seasons
Gooznews
Gel Health News
Hands Off My Health
Health Business Blog
Health Care BS
Health Care for All
Healthy Skepticism
Hooked: Ethics, Medicine, and Pharma
Hugh Hewitt
IgniteBlog
In the Pipeline
In Vivo
Instapundit
Internet Drug News
Jaz'd Healthcare
Jaz'd Pharmaceutical Industry
Jim Edwards' NRx
Kaus Files
KevinMD
Laffer Health Care Report
Little Green Footballs
Med Buzz
Media Research Center
Medrants
More than Medicine
National Review
Neuroethics & Law
Newsbusters
Nurses For Reform
Nurses For Reform Blog
Opinion Journal
Orange Book
PAL
Peter Rost
Pharm Aid
Pharma Blog Review
Pharma Blogsphere
Pharma Marketing Blog
Pharmablogger
Pharmacology Corner
Pharmagossip
Pharmamotion
Pharmalot
Pharmaceutical Business Review
Piper Report
Polipundit
Powerline
Prescription for a Cure
Public Plan Facts
Quackwatch
Real Clear Politics
Remedyhealthcare
Shark Report
Shearlings Got Plowed
StateHouseCall.org
Taking Back America
Terra Sigillata
The Cycle
The Catalyst
The Lonely Conservative
TortsProf
Town Hall
Washington Monthly
World of DTC Marketing
WSJ Health Blog