FDA’s Post

The vast majority of #diagnostictests for #rarediseases are #labdevelopedtests or #LDTs - including most #newbornscreening tests, #genetic tests including #genepanels, #wholegenomesequencing and #wholeexomesequencing, #companiondiagnostics including #biomarkertests, and many #biochemicaltests. For the past 40 + (!) years (since the Medical Device Amendments of 1976), these tests were under FDA's enforcement discretion. #FDA's LDT final rule sets out to change all that. The rule includes narrow exemptions for certain LDTs - including those LDTs that are currently marketed, and those that BOTH address an unmet medical need AND are offered and used in a single health system (whatever that will actually mean, a lot of legal definitions yet to come). BUT, even those exemptions on apply to *some* #regulatoryrequirements such as pre-market clearance or approval (i.e., #510K clearance or #pma). ALL LDTs will have to comply with other requirements including labeling, medical device reporting (#MDR) requirements, and quality system (#QS) requirements around complaint handling. MANY OF THESE REQUIREMENTS ARE SET TO GO INTO EFFECT IN LESS THAN 2 YEARS! Nobody knows exactly how many LDTs are currently offered, which is a big part of the problem - but everyone agrees that there are A LOT of LDTs out there (tens of thousands of them). Webinars such as this one are crucial to help diagnostic labs, healthcare systems, and other key stakeholders navigate these new requirements: two years, thousands and thousands of tests, and a complex healthcare system that for the past 40 + years did not include this kind of oversight ... the challenges are obvious, and time is ticking. What does all this mean for #raredisease patients who need BOTH high-quality, reliable diagnosti tests they can trust AND ready access to these tests; after all, many of our patients already spend 7 years or more on the 'diagnostic odyssey?' Ultimately, time will tell. We at #NORD the National Organization for Rare Disorders are thankful for webinars like this one - but also deeply concerned about how the rule implementation may play out for our community, and what all this may ultimately mean for #raredisease #drugdevelopment (only think about #companion diagnostics and how vital LDTs are to bridge approval of the diagnostic tests there), for #newbornscreening programs, for access to #genetictesting and #precisionmedicine - and, ultimately most importantly, for our patients and their ability to access the #raredisease #treatments they need - when they need them and where they need them.

If you've been following the discussions on the changes to FDA regulations making many LDTs require compliance with the IVD regulations, here is another FDA webinar providing details on achieving compliance.

Key Update from FDA Regarding IVD Regulations: The FDA is moving to amend its regulations to explicitly state that LDT IVDs are devices under the Food, Drug, and Cosmetic (FD&C) Act. Ensure clarity in regulations, as current device definitions do not differentiate between device manufacturers. Phase out the general enforcement discretion approach for Laboratory Developed Tests (LDTs) to uphold the same enforcement approach as for other IVDs. FDA suggests that this has the potential to foster innovative IVD manufacturing. In my view, it could have the opposite effect in clinical trials. Many trial protocols will remove all LDT IVDs because the cost of including them is prohibitive, even though it would potentially have great benefits for the selection of patients. 🗣️ Your thoughts? The window for commenting on this proposed rule is open until 4th Dec 2023. https://lnkd.in/exdDmUrD

ASSESSING REGISTRIES TO SUPPORT DRUG REGULATORY DECISION-MAKING The Cures Act of the 21st Century, enacted on December 13, 2016, aimed to expedite the development of medical products and facilitate quicker and more efficient delivery of innovations and advancements to patients in need. One of its additions was section 505F to the Federal Food, Drug, and Cosmetic Act (FD&C Act). In compliance with the stipulations in section 505F, the FDA established a framework for the Real-World Evidence (RWE) Program, which assesses the potential utilization of RWE to aid in the approval of a new indication for a drug previously sanctioned under section 505(c) of the FD&C Act. Read more at : https://lnkd.in/eTqEh2TA

Small Entity Compliance Guide: Laboratory Developed Tests Today, the U.S. Food and Drug Administration (FDA) issued a Small Entity Compliance Guide (SECG) to assist small entities in complying with FDA regulations as they apply to in vitro diagnostic (IVD) products, including laboratory developed tests (LDTs). The LDT final rule published on May 6, 2024, amends the FDA’s regulations to make explicit that IVDs are devices under the Federal Food, Drug, and Cosmetic Act (FD&C Act) including when the manufacturer of the IVD is a laboratory. Along with this amendment, the FDA finalized a policy under which the FDA will phase out its general enforcement discretion approach for LDTs over the course of four years so that IVDs manufactured by a laboratory will generally fall under the same enforcement approach as other IVDs. The phaseout includes targeted enforcement discretion policies for specific categories of IVDs manufactured by laboratories. https://lnkd.in/e9_kDv-m

🔔 Coming June 26! If you work on prescription drug promotion, don’t miss this webinar! Learn directly from FDA experts about the different categories and types of promotion, as well as the regulatory requirements for each. The discussion will cover the clear, conspicuous, and neutral (CCN) final rule, the five standards associated with the rule, and the rule’s compliance date.  Register ➡

From May 6, 2024, any new laboratory developed tests (LDT) coming into the US market must be cleared through premarket processes. To all companies developing IVD kits and detection kits relying on CLIA labs for market penetration – please rethink your route.   #medicaldevices #IVD #FDA #LDT #LDPConsulting

🔔 Coming June 26! If you work on prescription drug promotion, don’t miss this webinar! Learn directly from FDA experts about the different categories and types of promotion, as well as the regulatory requirements for each. The discussion will cover the clear, conspicuous, and neutral (CCN) final rule, the five standards associated with the rule, and the rule’s compliance date. Register ➡ https://lnkd.in/eyPqVMF4

Today, the FDA published the revised final guidance Application User Fees for Combination Products. This guidance replaces the final guidance of the same title issued in 2005, and includes revisions for consistency with the current user fee programs and revisions to reflect combination product provisions in the FD&C Act (section 503(g)). Combination products may be reviewed in a single application or in separate applications for the constituent parts, as appropriate. This guidance explains that combination products for which a single application is submitted should be assessed by the applicable user fee associated with that particular type of application.  The document also explains how the Agency applies user fees for combination products when separate applications are submitted for the constituent parts.   This guidance should be used in conjunction with the guidance for industry Prescription Drug User Fee Act Waivers, Reductions, and Refunds for Drug and Biological Products and the appropriate medical device user fee amendments (MDUFA) guidance document(s). #fda #userfees #gcp #glp #gmp #ind #bla #ide #combinationproducts

FDA's OPDP has issued the second regulatory action letter of the year. Looking at the recent focus of the agency in the letters of the last two years, a pattern possibly presents itself. Check out my latest posting about FDA #OPDP enforcement and #pharma promotional speech.