I was just sent this in my email. Knowing nothing about it, and unable to comment, it’s here in my Rejected Posts alternative blog. I’m keen to hear what others, familiar with it, think of this 21st Century Cures Act. (It reminds me of the Doors, 20th Century Fox). Actually, I think this was passed in 2016, so it’s not big news. Of course the push for “personalized medicine is part of this, never mind that it hasn’t had much success yet. I would have thought observational studies were at least admissible before. Do they require adjustments for data-dredging? Or not? I’m glad they will mention “pitfalls”. Now I’m going to have to ask my doctor which type of study a drug has had its approval based upon.
Thanks to the 21st Century Cures Act you are no longer bound to traditional clinical trials to prove safety and efficacy — new and exciting alternatives are available to you!
Observational studies are now acceptable in the FDA’s approval process. These studies allow the collection of real-world usage information from patients and physicians. And they allow for the harvesting of data from existing studies — from university research to patient data registries — to use as evidence of a product’s efficacy and safety (my emphasis).
Observational research requires an entirely different set of procedures and careful planning to ensure the real-world evidence collected is valid and reliable.
The 21st Century Take on Observational Studies walks you through everything you need to know about the opportunities and pitfalls observational studies can offer. The report looks at the growing trend toward observational research and how provisions in the 21st Century Cures Act create even more incentives to rely on real-world evidence in the development of medical products. The report covers:
- Provisions of the 21st Century Cures Act related to observational studies and gathering of real-world evidence
- The evolution of patient-focused research
- How observational studies can be used in the preapproval and postmarket stages
- The potential for saving time and money
- New data sources that make observational studies a viable alternative to clinical trials
- How drug- and devicemakers view observational research and how they are using it
Order your copy of The 21st Century Take on Observational Studies: Using Real-World Evidence in the New Millennium and learn effective uses of observational studies in both the preapproval and postmarket phases; how to identify stakeholders and determine what kind of data they needed, and how the FDA’s view on observational research is evolving.
(Sec. 2263) Clinical testing of medical devices or drugs no longer requires the informed consent of the subjects if the testing poses no more than minimal risk and includes safeguards.
I’d like to know what counts as minimal risk. If they don’t require informed consent, then do they require no consent at all? or some kind of minimal consent?
Here’s some info on the bill, scarcely comforting:
Among other measures, the legislation calls for the use of “data summaries” to support the approval of certain drugs for new indications, rather than full clinical trial data. The Cures Act will also allow drug companies to promote off-label uses to insurance companies, allowing them to expand their markets.
Winner: ‘Real world evidence’
The Cures Act directs the FDA to evaluate the use of “real world evidence” for approval of new indications for FDA-approved drugs. Proponents see this as a way to apply useful and timely information learned from observational studies, patient input, anecdotal data, and other research to drug and device approval.
Loser: Randomized clinical trials
Currently the gold standard for testing drugs and devices for safety, the adoption of real world evidence standards may indicate that randomized clinical trials will become less important for drug and device approval.