Several years ago, in FDASIA, Congress added two sentences the a key section of FDA law (FFDA Section 505(d)), instructing the agency to use structured risk benefit in decision making. Now, Congress proposes to revise that language a little. Continued after the break.
The key sentences at Federal Food and Drug Act - 505(d) - today stand as they were created by FDASIA(here):
The Secretary shall implement a structured risk-benefit assessment framework in the new drug approval process to facilitate the balanced consideration of benefits and risks, a consistent and systematic approach to the discussion and regulatory decisionmaking, and the communication of the benefits and risks of new drugs. Nothing in the preceding sentence shall alter the criteria for evaluating an application for premarket approval of a drug.
For a blog about how this language got into FDASIA, see (here). For an FDA deck on the topic, (here).
What's New Now?
The May 13, 2015 release of the House 21st Century Cures Bill (Section 2001, here) deletes the above two sentences and replaces them with language that is grammatically more clear:
Redline as:
(1)
The secretary shall implement a structured risk-benefit assessment framework in the new drug approval process
(A) To facilitate the balanced consideration of benefits and risks, and
(B) to develop and implement a consistent and systematic approach to the discussion of, regulatory decision making with respect to, and the communication of, the benefits and the risks of new drugs.
(2) Rule of construction. Nothing in paragraph (1) shall alter the criteria for evaluating an application for premarket approval of a drug.
This is the Paragraph that Defines "Substantial Evidence" Standards of the FDA
Comparisons are sometimes made between the FDA decision-making process (safe and effective) and the CMS decision-making process (reasonable and necessary.) Congress provides no statutory guidance on how CMS should define reasonable and necessary, but, in sentences just preceding the structured risk/benefit quotations above, Congress does define "substantial evidence" for the purpose of FDA decision-making on new drugs. Congress defines as follows at 505(d):
Comparisons are sometimes made between the FDA decision-making process (safe and effective) and the CMS decision-making process (reasonable and necessary.) Congress provides no statutory guidance on how CMS should define reasonable and necessary, but, in sentences just preceding the structured risk/benefit quotations above, Congress does define "substantial evidence" for the purpose of FDA decision-making on new drugs. Congress defines as follows at 505(d):
The term "substantial evidence" means evidence consisting of adequate and well-controlled investigations, including clinical investigations, by experts qualified by scientific training and experience to evaluate the effectiveness of the drug involved, on the basis of which it could fairly and responsibly be concluded by such experts that the drug will have the effect it purports or is represented to have under the conditions of use prescribed, recommended, or suggested in the labeling or proposed labeling thereof. If the Secretary determines, based on relevant science, that data from one adequate and well-controlled clinical investigation and confirmatory evidence (obtained prior to or after such investigation) are sufficient to establish effectiveness, the Secretary may consider such data and evidence to constitute substantial evidence for purposes of the preceding sentence.
Footnote.
In July 2015, BIO released a paper called "Life Cycle Perspective for Structured Risk Benefit Design," here.
