Cures 2.0 – Details of what’s in store for Real-World Evidence

CONTEXT:  This article provides a nice summary and a bit more detail on the draft legislation that will eventually become Cures 2.0.  As we reported in June, the draft legislation is now out for review and discussion.  From a Real-world research perspective there are two key areas of note.  Firstly grants for novel trial designs and other innovations in drug development contains specific wording to prioritise the use of digital health technologies and real-world evidence.  Secondly there is a specific section dedicated to increasing the use of real-world evidence with 3 key measures;  1. Cures2.0 directs the Secretary of HHS to issue guidance within 6-months of enactment on the use of RWE in evaluating drug safety and effectiveness. Such guidance should consider: (1) special and underrepresented populations; (2) acceptable endpoints and outcomes measures; (3) data quality standards; (4) data transparency requirements; and (5) study design considerations. 2. Cures 2.0 also directs the Secretary of HHS to, within 1 year of enactment, identify approaches for HHS to use real world evidence in research and regulation and purchase of health care products and services.3.  And finally Cures 2.0 directs the Secretary of HHS to establish a Real World Evidence Task Force.  All welcome news.  There are a couple of other points to note here.  Cures 2.0 also calls for increased diversity in clinical trials.  This is a topic we have covered several times as RWE studies show time and again how minorities are missing out on the treatments once they are approved, because of the lack of representation at the approval stage.  This author is very happy to see this issue being addressed directly within Cures 2.0.

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1. “Notable changes include:. Emergency Use Authorizations during a public health. emergency:. The bill makes clear that under certain conditions, diagnostic. tests used during a declared public health emergency, such as the. ongoing pandemic, may be authorized for emergency use and do not. need to be approved by FDA before they are used to diagnose an. emerging threat.” 

2. “The draft builds. off of Reps. Upton and DeGette’s landmark 21st Century Cures. Act, which was enacted in 2016, and includes a number of bipartisan. provisions intended to support, and increase access to, of the bills are “must pass” items required to. be taken up by Congress, and with a summer and fall legislative. calendar expected to be dominated by infrastructure, appropriations. and the National Defense Authorization Act (NDAA), it is unlikely. that either will receive significant committee consideration this. year.” 

3. “Like last year’s iteration, the VALID Act of 2021 would. unequivocally give FDA authority to regulate diagnostic tests and. most of their constitutive components under the Federal Food, Drug,. and Cosmetic Act by creating an entirely new product category,. in vitro clinical tests (IVCTs), for all in vitro. diagnostics (IVD) and Lab Developed Tests (LDTs).” 

4. “Definition of “High Risk” IVCTs:. The current draft modifies the definition of a. “high-risk” IVCT in several ways, including: 1) it would. only apply to those uses “intended” by the test. developer, 2) “potential” has been dropped as qualifier. from “unreasonable risk” for serious harm or death from. an inaccurate result, and 3) a delay of life-supporting or. life-sustaining medical treatment caused by an inaccurate result. must now be “significant.”” 

5. “Definition of “Low Risk” IVCTs (which are exempt from. pre-market review):. The current draft modifies the definition of a. “low-risk” IVCT in the following ways: 1) it too only. applies to tests when used as “intended” by the. developer, 2) allows for additional circumstances (such as the. availability of confirmatory tests), and 3) makes clear that. appropriate “mitigating measures” may overcome potential. “serious adverse health consequence[s].”” 

Source URL: https://www.mondaq.com/unitedstates/healthcare/1088288/the-valid-act-21st-century-cures-20-what-industry-needs-to-know-