On April 29, 2024, the U.S Food and Drug Administration (FDA) announced its final rule on Laboratory Developed Tests (LDTs). This final ruling amends the FDA’s regulations to make explicit that in vitro diagnostic products (IVDs), including those manufactured by laboratories, are devices under the Federal Food, Drug, and Cosmetic Act (FD&C Act). Alongside the amendment, FDA issued its policy to phase in regulatory requirements for certain LDTs over the course of four years.

The FDA will host a webinar to provide an overview of the final rule on May 14, 2024. A link to register can be found here. The final rule is expected to have profound effects on many LDT developers. Goodwin’s Life Sciences Regulatory & Compliance Team are ready to work with clients to navigate the challenges that the final rule may pose. Our breakdown and analysis of the rule will be upcoming on Goodwin’s LDT Resource page.

In April 2023, we published an alert in relation to two European Commission legislative proposals: new Regulation 2023/0131 and new Directive 2023/0132, to replace the current EU regulatory framework for all medicines (including those for rare diseases and children). On April 10, 2024, the European Parliament adopted its position on the European Commission’s legislative proposals with respect to (i) Regulation 2023/0131 that can be found here and (ii) Directive 2023/0132 that can be found here. For certain key areas covered in the proposed EU legislation, we have set out a brief summary of (i) the European Commission’s original proposals and (ii) the European Parliament’s proposed amendments. You can read more here.

Goodwin’s Rare Disease Initiative hosted its Annual Rare Disease Symposium in Boston on March 13, 2024. Participants were invited to join for an afternoon of engaging and inspirational conversations led by Julie Tibbets, Matt Wetzel, and Danielle Lauzon, in addition to networking with peers in the rare disease community. The program included speakers covering the patient, advocacy, policy, research, and CEO perspectives.

For more event highlights and key takeaways from our speakers, please visit the Goodwin Rare Disease Symposium 2024 page.

As the life sciences, medtech, and diagnostic industries continue to expand and grow increasingly complex, so does the legal, regulatory, and compliance landscape. To help companies and investors navigate the many evolving and emerging laws and regulations across pharmaceuticals, biologics, medical devices, diagnostics, and laboratory testing, our Life Sciences Regulatory & Compliance team has provided an overview of key developments. We update and publish a quarterly tracker detailing these developments. You can read about the Q2 2024 updates here.

On March 1, 2024, the Office of Information and Regulatory Affairs (“OIRA”), Office of Management and Budget (“OMB”), Executive Office of the President received the final version of FDA’s rule on regulation of laboratory developed tests (“LDTs”) for administrative review.  Having swiftly moved to OIRA review in under 5-months from the publication of the proposed rule and under 3-months from the end of its comment period, the rule has undoubtedly been a top priority for the FDA.  Further, as of the date of this post, OIRA has scheduled four back-to-back meetings with interested stakeholders, all of which are to be held the week of March 18th.  All of this signals that the final rule remains on track for potential issuance in April 2024, the target date for final action on the rule as we previously discussed here.

Further, on March 14, 2024, the House Energy and Commerce Committee Chair and Subcommittee on Health Chair announced a subcommittee hearing titled “Evaluating Approaches to Diagnostic Test Regulation and the Impact of the FDA’s Proposed Rule.”  The hearing is scheduled for Thursday, March 21, 2024 at 10:00 AM ET.  Additional information on attending or viewing the hearing is available here.

Be sure to bookmark our dedicated LDT Resource Page to stay informed on the latest news and analyses on LDTs.

Goodwin’s Life Sciences team will be hosting an upcoming event in our Boston office on March 13, 2024 to spotlight the critical work being done to address the 7,000+ rare diseases that impact more than 300 million people globally.

Join us in person in our Boston office or attend virtually for our Annual Rare Disease Symposium on March 13, 2024. Look forward to an afternoon of engaging fireside chats, inspirational presentations, and networking with your peers in the rare disease community. This year’s program will include speakers covering the patient, advocacy, policy, research, and CEO’s perspectives.

The U.S. Food and Drug Administration (FDA) recently issued a draft guidance, “Master Protocols for Drug and Biological Product Development”, that echoes and builds on principles that the Agency previously set forth in guidance for COVID-19 master protocols (2019), master protocols in oncology (2022) and clinical trials for multiple versions of cellular or gene therapy products (2022). The draft guidance offers numerous (and at times very detailed) recommendations to facilitate the design, efficient analysis of data, and regulatory review of clinical trials conducted under such master protocols.

As a starting point, this draft guidance defines several key terms, including the types of trials that can be conducted under a master protocol:

Master protocols offer sponsors the ability to streamline drug development through shared control groups, study infrastructure and oversight. However, these protocols also involve increased complexities and design challenges that generally require a higher degree of coordination. Here, we highlight some key design and analysis considerations addressed in the draft guidance:

Randomization

Sponsors should consider allocating more subjects to control arms than for each individual drug arm to increase power and reduce the risk of a poorly or highly performing control arm. For a platform trial, a sponsor should create a plan for changes to the randomization ratios that can occur as products enter and exit a platform trial. In umbrella or platform trials comparing different drugs, the sponsor should ensure that the randomization process prevents subjects from being randomized to drugs they are not eligible to receive given each drug’s exclusion criteria.

Informed Consent

Sponsors should cover all treatment arms in their informed consent and obtain consent prior to randomization. In a platform trial where drugs are entering and exiting the study, consent forms should be modified accordingly to reflect the drugs currently under evaluation. FDA also recommends the use of a central IRB to review informed consent forms, the protocol, and other relevant documents for monitoring of a trial conducted under a master protocol.

Blinding

Given the potential for different administration methods for various drugs included in umbrella or platform trials, unique blinding challenges may arise and sponsors should discuss their proposed approach to blinding with FDA early in the planning stage.

Safety Data

Safety data from a master protocol can be considered part of overall safety database but data from other sources may be needed to support approval. The type of master protocol and the drugs being evaluated will impact the approach to safety data collection. FDA also recommends that a data monitoring committee (DMC) or other independent, external entity review accumulating safety and efficacy data to minimize inadvertent dissemination of information that could pose risks to trial integrity.

Regulatory Review Considerations

Each master protocol should be submitted as a new IND, and FDA recommends that the sponsor request a pre-IND meeting to discuss the protocol and other IND submission details.  Given the potentially rapid pace of changes in a master protocol, the draft guidance recommends specific procedures for protocol amendments, including cover letters for each protocol amendment that update on the status of each drug and notifying the RPM at least 48 hours before submitting any protocol amendment that could substantively affect the master protocol.  The IND should also include a well-designed communication plan to facilitate timely and effective communication between multiple stakeholders, including rapid communication of serious safety information and protocol amendments to investigators and FDA.

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Comments on this draft guidance are due February 22, 2024. Please contact the authors or your Goodwin attorney with any questions or if you would like to submit a comment.

As the life sciences, medtech, and diagnostic industries continue to expand and grow increasingly complex, so does the legal, regulatory, and compliance landscape. To help companies and investors navigate the many evolving and emerging laws and regulations across pharmaceuticals, biologics, medical devices, diagnostics, and laboratory testing, our Life Sciences Regulatory & Compliance team has provided an overview of key developments. We update and publish a quarterly tracker detailing these developments. You can read about the Q1 2024 updates here.

On December 6, 2023, the Office of Information and Regulatory Affairs (“OIRA”) released the Fall 2023 Unified Agenda of Regulatory and Deregulatory Actions (the “Agenda”), a semiannual compilation of information regarding regulations under development by federal agencies. In its preamble, the Department of Health and Human Services (“HHS”) notes that the regulatory actions forecasted for the Agenda reflect the priorities of HHS Secretary Xavier Becerra and the Biden-Harris Administration, HHS, and the U.S. Food and Drug Administration (“FDA”).

As we analyzed in detail in recent articles (see here, here and here), the proposed rule for laboratory developed tests (“LDTs”) was released in October 2023.  Citing factors including “extensive background of public comment on this topic” and “the public health benefits of proceeding expeditiously,” FDA declined to extend the 60-day comment period, which closed on December 4, 2023.  FDA received over 6,000 comments from individual citizens, laboratories, academic medical centers, and other industry stakeholders.  As part of the Agenda, FDA has updated the target date for final action on the LDT proposed rule to April 2024.

FDA is under no obligation to publish the LDT rule according to the schedules reflected in the Unified Agenda.  If the rule and related LDT policy are finalized as proposed by April 2024, high-risk LDTs may be called-in for premarket review as early as October 1, 2027. Subsequently, low-to-moderate risk LDTs may be called-in for premarket review as early as April 1, 2028.

To stay informed on the latest news and analysis affecting LDTs, be sure to bookmark our dedicated LDT Resource Page.

Two recent federal court cases signal new significant developments with respect to the 340B Drug Pricing Program. Specifically: (1) new federal district court litigation challenging a recent HRSA Notice involving 340B Program “child site” registration and eligibility; and (2) a court decision in other litigation that implicates the scope of the 340B “eligible patient” definition. Details regarding these developments are in the client alert.

Read the client alert here.