On April 18, U.S. Food and Drug Administration (FDA) Commissioner Marty Makary announced plans to roll-out a new approval pathway for rare disease drugs. Commissioner Makary’s comments build on sentiments expressed across both the patient community and industry that rare disease drug development needs greater regulatory flexibility in order to speed access to treatments for patients with no or limited options. This is an initiative that has also been trumpeted by Janet Woodcock, former Principal Deputy Commissioner and Acting Commissioner of the FDA, in her work since retiring from the FDA. Prior legislative proposals (including the “Promising Pathway Act” proposed in 2024) have attempted to create a time-limited conditional approval pathway in the rare disease space, and Commissioner Makary’s remarks may signal a renewed push for action.

In last week’s interview, Commissioner Makary emphasized the following potential eligibility factors in how he is thinking about a new “conditional” approval pathway: rare conditions affecting only a small number of people, where a randomized clinical trial has not been conducted and is not feasible, but where a “plausible mechanism” physiologically exists. Commissioner Makary also noted that post-approval monitoring of adverse events and other data may be an important tool to support more flexible regulatory decision making about drug approvals.

Whether and when the FDA or Congress will take further steps in outlining a conditional approval pathway, and what form that outline may take (e.g., Agency guidance, expansion of the current accelerated approval authorities, or new legislation), remains unclear at this time. This is an area rare disease researchers and developers should monitor for developments, including any opportunities to provide comments to the FDA on its potential plans.

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 2025 updates here.

Attendees at this year’s symposium were optimistic about the potential for progress, citing momentum from new FDA initiatives, growing legislative support, and increased global innovation in research and development. These efforts, alongside increased patient advocacy and a presidential administration focused on speeding patient access, could lead to significant advances in rare disease treatments and cures in 2025.

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Based on recent policy signals and statements from incoming administration officials, a picture of potential regulatory and policy changes that could affect biotech, pharmaceutical, and medical device companies in coming months and years is emerging.

Anticipated changes span multiple regulatory fronts: a revamped approach to antitrust review at the Federal Trade Commission (FTC), continued momentum on biosecurity measures, and a fundamental rethinking of agency regulation to streamline “red tape” and accelerate patient access to innovative treatments. The Trump administration’s stated focus on “making America healthy again” suggests a broader transformation in how healthcare is delivered and regulated, with emphasis on nutrition, prevention, longevity, enhanced physician autonomy, and a more holistic approach to health to reduce the burdens of chronic disease.

While some changes may create opportunities for innovation and growth, others could pose compliance and operational challenges. Understanding these emerging dynamics will be crucial for industry stakeholders as they position themselves for success under the new administration.

The following six sections are based on discussions from a regulatory panel held on January 15 at the Goodwin + KPMG 6th Annual Symposium, which was held during the 2025 JPM Healthcare Conference.

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On January 7, 2025, FDA announced the availability of a final guidance document titled “Communications From Firms to Health Care Providers Regarding Scientific Information on Unapproved Uses of Approved/Cleared Medical Products.” The final guidance supersedes the agency’s revised draft guidance of the same title issued in October 2023 (see our analysis of the draft guidance here) and includes several key updates, including further describing scientific standards for appropriate source publications, providing additional examples of the separate dissemination of information on approved and unapproved uses in different scenarios, and expanding the section on firm-generated presentations with further context on what is permitted and what would be viewed as inappropriate when an SIUU communication includes a source publication and firm-generated content.

Several of these updates appear to be responsive to comments from industry stakeholders on the draft guidance. For example, the draft guidance stated that source publications for SIUU communications should describe “scientifically sound” studies and analyses that provide “clinically relevant” information. Multiple commenters requested that the “clinically relevant” and “scientifically sound” concepts be either removed or more clearly defined. The final guidance no longer contains the “clinically relevant” terminology, but provides some further recommendations on what constitutes a “scientifically sound” study or analysis, noting for example that certain early-phase studies could meet this standard.

Similar to the draft guidance, the final guidance document is written in a question and answer format and addresses: (1) what firms should consider when determining whether a source publication is appropriate to be the basis for an SIUU communication; (2) what information should be included as part of an SIUU communication; (3) how SIUU communications should be presented (e.g., the format and accompanying disclosures); and (4) recommendations for specific types of materials (including reprints and clinical reference resources). The final guidance includes a new question and answer focusing specifically on recommendations for firm-generated presentations.

The final guidance also provides an expanded list of examples of communication techniques that FDA regards as “encouraging” an unapproved use of a medical product. In addition to celebrity endorsements, premium offers, and gifts (which were noted in the draft guidance), the final guidance identifies emotional appeals unrelated to scientific content, promotional tag lines, and jingles, along with “calls to value” that “pre-judge the benefit(s) of the medical product for individual patients” (e.g., “Click here to start improving your patients’ lives today”), as techniques that would take a firm-generated presentation outside the scope of the guidance’s enforcement policy.

FDA has submitted the guidance to the Office of Management and Budget for review and clearance of certain information collection provisions contained in the guidance. As such, the final guidance is not for current implementation, but we expect to see a Federal Register notice about the final guidance’s applicability once this administrative step is complete.

Please contact any of the authors or your Goodwin attorney if you have any questions about this final guidance.

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 2025 updates here.

On October 4, 2024, a US House version of the revised Promising Pathway Act (PPA) 2.0 was introduced, sponsored by Rep. Bruce Westerman (R-AR). The bill (H.R.9938) mirrors a US Senate version that was introduced in May 2024 (S.4426) that would authorize the US Food  and Drug Administration (FDA) to grant time-limited conditional approval to drugs for rapidly progressive, terminal diseases with substantial unmet need for treatments that are eligible for the Orphan Drug Act and result in a substantially shortened lifespan, substantial reduction in quality of life, or other substantial adverse health effects.

Read the full insight here.

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 Q4 2024 updates here.

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 Q3 2024 updates here.

In Draft Guidance published this week by the U.S. Food and Drug Administration (FDA), Guidance for Industry – Processes and Practices Applicable to Bioresearch Monitoring Inspections, the Agency provides some wisdom on best practices for responding to Form FDA 483s, albeit in the context of its Bioresearch Monitoring (BIMO) program inspections, but very much translatable to any Form FDA 483 response. FDA notes the following best practices:

A response should demonstrate the establishment’s acknowledgment and understanding of FDA’s observations. It should also demonstrate the establishment’s commitment to address the observations, including a commitment from senior leadership.

Responses should be well-organized and structured to:

• • • Address each observation separately
    • Note whether the establishment agree(s) or disagree(s), and why
    • Provide both corrective and preventive actions and timelines for completion
    • Provide both completed and planned actions and related timelines
    • Provide a method of verifying or monitoring the effectiveness of the actions
    • Submit documentation (e.g., training, Standard Operating Procedures (SOPs), corrective action plans, records, etc.)

Importantly, FDA also states that timely Form FDA 483 responses that include “appropriate corrective and preventive actions could impact FDA’s determination of the need for subsequent Agency action.” FDA encourages responses within 15 business days after the end of an inspection and, helpfully, notes that any responses received within that window “will be considered before further Agency action or decision.” Interested stakeholders may submit comments here on FDA’s Draft Guidance until August 5, 2024.

Please contact Julie Tibbets or any member of our Life Sciences Regulatory & Compliance practice with questions on FDA’s Draft Guidance or on responding to Form FDA 483s.