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.

Medtech mergers and acquisitions (M&A) and venture capital (VC) showed signs of life in 2024, contributing to an overall optimistic outlook for the sector this year despite lingering headwinds.

Strategic investments are expected to continue as medtech companies innovate, particularly in areas such as AI-driven diagnostics, wearables and remote monitoring devices, and advanced surgical technologies.

Private, venture-backed M&A activity for medical devices—which picked up in the second half of last year and started 2025 strong with two ten-digit acquisitions and two spin-offs by strategics—could continue rising amid a more deregulatory backdrop under the new presidential administration.

Still, challenges persist that could slow growth. Early-stage VC deals in the sector have faced difficulties, and private M&A exit timelines have increased. Uncertainty regarding the path of interest rates and the broader economy also muddy the outlook.

Read the full insight here.

On January 7, 2025, the FDA issued a draft guidance called Considerations for the Use of Artificial Intelligence to Support Regulatory Decision-Making for Drug and Biological Products. The document clarifies how sponsors, manufacturers, and other industry developers should approach artificial intelligence (AI) to support safe, effective development and marketing of AI-based tools.

The guidance discusses the use of AI models in the nonclinical, clinical, post-marketing, and manufacturing phases of the drug product life cycle, where the specific use of the AI model is to produce information or data to support regulatory decision-making as it relates to safety, efficacy, or the quality of the product. It does not cover AI use in drug discovery or operational efficiencies that do not affect patient safety, drug quality, or study reliability.

Read the full alert here.

Goodwin’s Life Sciences team is excited to host its Annual Rare Disease Symposium in Boston on February 5, 2025. Participants are invited to join for an afternoon of engaging fireside chats, inspirational presentations, and networking with peers in the rare disease community.

Please see the agenda below and register to attend in-person or via our virtual webinar to join us.

Agenda

12:00 PM – 1:00 PM EDT | Welcome & Networking Lunch

1:00 PM – 4:30 PM EDT | Rare Disease Symposium Program

• The Patient View
  • David Downing, GRIN1 Dad
  • Jaime McHugh, Rare Disease Mom and NORD Running for Rare Champion
• The Research View
  • Dr. Shira Rockowitz, PhD, Data Science Director, Boston Children’s Hospital, Children’s Rare Disease Collaborative Co-Leader
  • Dr. Piotr Sliz, PhD, Vice President, Chief Research Information Officer & Associate Professor, Boston Children’s Hospital, Children’s Rare Disease Collaborative Co-Leader
• The FDA View
  • Amy Rick, Director of Strategic Coalitions for FDA’s Rare Disease Innovation Hub
• The Policy View
  • Karin Hoelzer, Senior Director, Patient Advocacy, BIO
  • Jack Kalavritinos, Founder, JK Strategies and the Washington Health Innovation Council, and Former Director, HHS Office of Intergovernmental & External Affairs
  • Judy Stecker, SVP, Burson, and Former HHS Deputy Chief of Staff for Strategy & Operations – Rare Disease Parent & Founder, Wheeler’s Warriors
• The View from the National Organization for Rare Disorders
  • Pamela Gavin, Chief Executive Officer, NORD
• The View from the Rare As One Network
  • Heidi Bjornson-Pennell, Senior Program Manager, Science in Society, and Lead, Rare As One Network
• The Biotech CEO View
  • Paula Ragan, PhD, CEO, X4 Pharmaceuticals

4:30 PM – 5:30 PM EDT | Networking Reception

We look forward to kicking off Rare Disease Month with you!

As discussed in a prior Goodwin Alert, the US Food and Drug Administration (FDA) recently released Draft Guidance for designating a platform technology for drug development pursuant to § 560k of the Federal Food, Drug, and Cosmetic Act. The platform technology program was included as part of the PREVENT Pandemics Act “to bring significant efficiencies to the drug development or manufacturing process.” Specifically, a platform technology must have the “potential to be incorporated in, or utilized by, more than one drug without an adverse effect of quality, manufacturing or safety.”

Read the full insight here.

The Bioresearch Monitoring (BIMO) Program, operated by the U.S. Food and Drug Administration (FDA), conducts on-site inspections and data audits in order to effectively monitor the compliance of all FDA-regulated research.

As a follow up to our July 2023 post, we highlight the most common violations identified in Fiscal Year (FY) 2023, in addition to those observed thus far in FY 2024.  BIMO conducted 1073 inspections in FY 2023.  The majority of these inspections (approximately 79%) were of drug, biologic, or medical device study clinical investigators, institutional review boards (IRBs), sponsors, clinical research organizations (CROs), and sponsor-investigators.  Some of the most common inspection outcomes are highlighted in our alert linked below. Our methodology included a search of FDA’s Warning Letter database for FY 2023 and 2024, to date, for letters issued by BIMO and the Center for Drug Evaluation and Research, the Center for Biologics Evaluation and Research, and the Center for Devices and Radiological Health to IRBs, CROs, clinical investigators, sponsors, and sponsor-investigators.

Read the full alert 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.

In newly released Draft Guidance from the U.S. Food and Drug Administration (FDA) entitled, Platform Technology Designation Program for Drug Development, the FDA addresses its new designation program for platform technologies, which is intended to bring efficiencies to drug development, manufacturing, and review processes for applications that incorporate designated platform technologies.

Read the full alert here.

On May 6, 2024, following more than a decade of discourse with interested stakeholders on potential approaches to regulation of laboratory developed tests (LDTs), the U.S. Food and Drug Administration (FDA) published its final rule setting forth its framework for oversight of LDTs. The final rule and accompanying policy to phase out the agency’s general policy of “enforcement discretion” for LDTs comes roughly six months after FDA published its proposed rule that outlined the agency’s proposed approach to increasing oversight over LDTs. As detailed in our prior analyses of the proposed rule (see here and here), FDA proposed to implement a phaseout policy that would, across five stages and within four years, apply to clinical laboratories offering tests as LDTs the same regulatory requirements applicable to in vitro diagnostics (IVDs).

The proposed rule received more than 6,500 comments, and while FDA did not change its amendments to the regulation or meaningfully modify the phaseout timeline, FDA has significantly modified its phaseout policy to extend full or partial enforcement discretion to additional categories of LDTs, creating a framework whereby the agency intends to take a more targeted enforcement approach, particularly in the near-term, to addressing LDTs.

You can read our more in our Insight, where Steven Tjoe, Matt Wetzel, and Sukrti Thonse highlight the key features of the final rule and five-stage phaseout policy. Be sure to bookmark our dedicated LDT Resource Page to stay informed on the latest news and analyses on LDTs.

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.