Goodwin’s Life Sciences Regulatory & Compliance practice puts the life sciences regulatory environment “under a microscope” to spot the latest trends, legal developments, and happenings.

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 will update this list on an ongoing basis throughout the year.

WHAT WE ARE TRACKING

Expand the sections below to learn more about trending topics in Q2 2024 and beyond.

Preclinical and Clinical Research

How companies develop their products:

• We are monitoring the BIOSECURE Act (and its corollary in the Senate), including the potential implications for research-stage companies that leverage technology described in the legislation. (Client Alert available here.) 
• We are monitoring new bipartisan legislation from Reps. Anna Eshoo (D-CA) and Michael McCaul (R-TX), the Innovation in Pediatric Drugs Act of 2023, which would increase funding for pediatric trials and require drugs for rare diseases to be studied in children. The legislation would also grant the FDA authority to penalize companies that do not complete required pediatric studies. The House Energy and Commerce Committee held a meeting on this legislation on February 29, 2024.
• We are watching whether Congress will advance legislation extending the sunset date of the FDA’s Rare Pediatric Disease Priority Review Voucher program. Under the current statutory sunset provisions, after September 30, 2024, the FDA may award a voucher for an approved rare pediatric disease product application only if the sponsor has rare pediatric disease designation for the drug and that designation was granted by September 30, 2024. After September 30, 2026, the FDA may not award any rare pediatric disease priority review vouchers. Companies holding or pursuing rare pediatric disease designations should be mindful of these upcoming deadlines.
• We continue to watch for the FDA’s implementation of its new clinical trial Diversity Action Plan authority for Phase 3 or pivotal study drug and biologic developers and device developers conducting clinical trials (whether under investigational device exemptions or not), pursuant to section 3601 of the Food and Drug Omnibus Reform Act of 2022 (FDORA). The FDA missed the deadline set in FDORA of December 29, 2023. Any FDA guidance may inform not only trial design and recruitment planning moving forward for sponsors but also updates to public company product development risk factors.
• We continue to watch for the FDA’s implementation of a new provision of the Federal Food, Drug, and Cosmetic Act that allows the FDA to designate a “platform technology” that has the potential to be incorporated in or used by more than one drug. Under section 2503 of FDORA, the FDA may expedite the development and review of a subsequent application for a drug that incorporates or uses a designated platform technology and must issue draft guidance on the new designation program within one year of enactment of FDORA. The head of the FDA’s Office of Therapeutic Products has previously noted the potential opportunity the designation can offer cell and gene therapy sponsors. The FDA missed the deadline to issue draft guidance by December 29, 2023, on the implementation of this new designation program, including examples of drugs that can be manufactured using platform technologies and information about the submission and review of designation requests, among other things. We continue to monitor for its release, because it may inform sponsor product development activities as well as updates to public company product development risk factors.
• We continue to monitor implementation by the Medicines and Healthcare products Regulatory Agency (MHRA) of legislation to reform the regulatory framework for clinical trials in the UK. On March 21, 2023, the MHRA published its response to the public consultation that it conducted at the beginning of 2022 on legislative proposals concerning the regulation of UK clinical trials. The MHRA has confirmed that the new legislation will ensure that patients and their safety are the focus of all clinical trials, bring the benefits of clinical trials to everyone, create a proportionate and flexible regulatory environment, cement the UK as a destination for international trials, and provide a framework that is streamlined, agile, and responsive to innovation. On October 12, 2023, the MHRA introduced a Notification Scheme in the UK, which ensures that initial clinical trial applications for certain Phase 3 and Phase 4 trials are approved by the MHRA within 14 days (instead of the statutory 30 days), provided that the sponsor shows that the trial meets the MHRA’s inclusion criteria.
• We are monitoring implementation of Executive Order 14117 and a related Advance Notice of Proposed Rulemaking issued by the DOJ. They target foreign access to “bulk sensitive personal data” of US persons by countries of concern (i.e., the People’s Republic of China [including Hong Kong and Macau], Russia, Iran, North Korea, Cuba, and Venezuela) and certain entities and individuals connected to these countries. The proposed DOJ framework would prohibit or restrict certain transactions through which a country of concern or covered person could access human genomic data, personal health data, or biometric identifiers, among other types of data, subject to proposed volume thresholds ranging from 100 to 1 million US persons. These rules could implicate commercial relationships with counterparties with ties to the listed countries, including for clinical trial data depending on the thresholds to be determined in the rulemaking. We continue to monitor these developments, which may inform updates to public company regulatory risk factors, particularly for companies with connections to China or another country of concern. (Client Alert available here.)

FDA, EMA, and MHRA Approvals

Deciding whether a company can market or sell a specific product:

Pharmaceuticals and Biologics

• We are actively monitoring further developments related to the FDA’s January 2024 safety labeling updates for serious risks of T-cell malignancy following BCMA-directed or CD19-directed genetically modified autologous chimeric antigen receptor (CAR) T cell immunotherapies. Life sciences companies developing genetically modified autologous CAR T cell therapies should consider whether updates to disclosures regarding their product’s side effects may be warranted, including the potential for the FDA to take regulatory action based on their product’s proposed mechanism of action and tolerability profile.
• We continue to monitor the implementation of the European Commission’s legislative proposals — Regulation 2023/0131 and Directive 2023/0132 — to replace the current EU regulatory framework for all medicines (including those for rare diseases and for children). The proposals, announced on April 26, 2023, aim to reduce costs, expedite the introduction of new medicines, and prevent medicine shortages. Read our summary of these proposals here. The legislative proposals have been under consideration by the European Parliament. On March 19, 2024, the European Parliament’s Committee on Environment, Public Health and Food Safety adopted its position on the proposals, which include a number of amendments to the original proposals. The European Parliament is scheduled to debate and vote on the amended proposals during a session on April 10 and 11, 2024. Even if the European Parliament approves the amended proposals, this is subject to further review and potential amendment by the European Council.
• We continue to monitor the implementation of the Windsor Framework that will apply as of January 1, 2025, in the UK. On February 27, 2023, the UK government and the European Commission announced a political agreement in principle to replace the Northern Ireland Protocol with a new set of arrangements, the Windsor Framework. This new framework fundamentally changes the existing system under the Northern Ireland Protocol, including the regulation of medicines in the UK. In particular, the MHRA will be responsible for approving all medicines destined for the UK market (that is, Great Britain and Northern Ireland), and the European Medicines Agency (EMA) will no longer have a role in approving medicines destined for Northern Ireland. The MHRA will grant a single UK-wide marketing authorization for all medicinal products sold in the UK, enabling medicines to be sold in a single pack and under a single authorization throughout the UK. The Windsor Framework was approved by the EU-UK Joint Committee on March 24, 2023, meaning the UK government and the EU will enact legislative measures to bring it into law.
• We continue to monitor the implementation of the International Recognition Procedure (IRP), effective January 1, 2024, in the UK, which replaces the European Commission Decision Reliance Procedure. The IRP allows the MHRA to consider the expertise and decision-making of medicines regulators in Australia, Canada, the EU, Japan, Singapore, Switzerland, and the US when approving a new medicine. The decentralized and mutual recognition reliance procedure (MRDCRP), which allows the MHRA to have regard for approvals in the EU, will be incorporated under the umbrella of the IRP.

Medical Devices and Medtech

• In September 2023, the FDA published a trio of draft guidances related to its efforts to “strengthen and modernize” the 510(k) Program. The draft guidances address two foundational issues of concern with the 510(k) process — best practices for selecting a predicate device, and when clinical data is needed in a 510(k) — in addition to providing updated performance testing recommendations for manufacturers of 510(k) implant devices. See our summary of these guidances here.
• We continue to monitor the implementation of new cybersecurity authorities for software-containing or internet-connected devices, enabling the FDA to require sponsors to develop cybersecurity plans for post-market implementation, as mandated in section 3305 of FDORA. The FDA issued its guidance on its “refuse to accept” policy for cyber devices and related systems in March 2023, but this policy expired on October 1, 2023, after the FDA published its final guidance, “Cybersecurity in Medical Devices: Quality System Considerations and Content of Premarket Submissions,” in September 2023. In March 2024, the FDA issued a draft guidance with select updates to the September 2023 cybersecurity guidance related to cybersecurity considerations for cyber devices and for documentation in device premarket submissions. The FDA has scheduled a webinar to discuss the draft guidance on April 30, 2024. The draft guidance is open for comment through May 13, 2024.
• We continue to monitor the impact of Regulation (EU) 2017/745 (the European Medical Device Regulation, or MDR), which has been applicable since May 26, 2021, in the EU. The MDR requires manufacturers to submit all applications for medical devices that they want to transition to the MDR by May 26, 2024, and they need to have a signed contract with an EU Notified Body for review and conformity assessment by September 26, 2024. The MDR also requires the use of the EU’s medical device database, EUDAMED, which effectively outlines the life cycle of medical devices by collating and processing information about those medical devices and related companies, such as the manufacturers of those devices. The mandatory use of EUDAMED will start when the entire system (including all six modules) is declared fully functional, which is expected in the fourth quarter of 2027.

In Vitro Diagnostic and Laboratory Testing

• On October 3, 2023, the FDA published a proposed rule to make explicit that laboratory-developed tests (LDTs) are devices regulated by the FDA. At the same time, the FDA is proposing a policy to, upon finalization of the proposed rule, phase out its current general enforcement discretion approach so that LDTs would generally fall under the same enforcement approach as in vitro diagnostic (IVD) tests that the FDA currently actively regulates. Comments on the proposed rule were due December 4, 2023. The FDA announced in its Fall 2023 Unified Agenda its plans to finalize the LDT proposed rule by April 2024. On March 1, 2024, the Office of Information and Regulatory Affairs (OIRA), Office of Management and Budget (OMB), and Executive Office of the President received the final version of the rule for administrative review. You can read more about the proposed rule at our LDT Resource Page.
• We are monitoring legislative interest in diagnostics policy. In March 2023, the Verifying Accurate Leading-edge IVCT Development Act of 2023 (the VALID Act) was introduced in the US House of Representatives by Reps. Larry Bucshon (R-IN) and Diana DeGette (D-CO). However, Congressional interest in diagnostics policy has largely fallen off after the VALID Act was not included in the 2023 omnibus bill at the end of 2022. On March 21, 2024, the House Committee on Energy and Commerce Subcommittee on Health held a hearing, “Evaluating Approaches to Diagnostic Test Regulation and the Impact of the FDA’s Proposed Rule.” Stakeholders at the hearing unanimously expressed a preference for a legislative approach to diagnostic test regulation. On the Senate side, in March 2024, Senator Bill Cassidy (R-LA) issued a request for information (RFI) on test regulation. Responses to the RFI are due by April 3, 2024.
• We are monitoring the FDA’s efforts to reclassify most IVDs that are currently class III (high risk), including infectious disease and companion diagnostic IVDs, into class II (moderate risk). According to a press release dated January 31, 2024, the FDA intends to propose reclassification of IVDs for which it believes there is sufficient information to establish special controls that, in combination with general controls, provide a reasonable assurance of safety and effectiveness for the tests.
• We are monitoring a European Commission legislative proposal, Regulation 2024/0021, to extend the time frame under which certain in vitro diagnostic medical devices are required to transition to Regulation (EU) 2017/746 (IVDR). The IVDR has been applicable since May 26, 2022, in the EU, and the proposal was published on January 23, 2024, to give companies more time to transition to the IVDR, under certain conditions. The European Commission has provided the proposal to the European Parliament and the European Council for their consideration.

Commercial Requirements

How companies manufacture, promote, and monitor their approved products:

• We continue to monitor recent bills introduced in the House and the Senate, Further Strengthening America’s Supply Chains and National Security Act, sponsored by Rep. Michael Waltz (R-FL) and Sen. Marco Rubio (R-FL). The draft legislation seeks to decrease US dependency on foreign supply chains, and specifically would require drug makers to share additional information with the FDA about where pharmaceutical ingredients are sourced. While no action has occurred on these bills since their introduction and referral to their respective House and Senate committees, given other legislative and executive actions focused on dealings with foreign adversaries, this measure may be one to watch.
• Industry members and their supply chain partners continue to enjoy a one-year stabilization period, until November 27, 2024, to allow trading partners to implement, troubleshoot, and mature their electronic interoperable systems to meet the requirements of the Drug Supply Chain and Security Act (DSCSA). This stabilization period is intended to avoid disruption to the supply chain and ensure continued patient access to drug products as trading partners work to fully implement the enhanced drug security requirements of the DSCSA. The FDA also issued an immediately effective compliance policy guidance regarding the enforcement of requirements for enhanced drug distribution security. It also issued a revised final compliance policy guidance that describes the FDA’s policies regarding enforcement requirements for wholesale distributors and dispensers to verify a product’s identifier in certain circumstances. As the year progresses, companies and their trading partners must finalize implementation of electronic interoperable systems.
• In February 2024, the FDA published its final rule harmonizing its device good manufacturing practice requirements to align more closely with the international consensus standard for devices by converging with the quality management system (QMS) requirements used by other regulatory authorities from other jurisdictions. The final rule will take effect February 2, 2026.
• We are monitoring the evolving situation and the FDA’s ongoing evaluation of quality and performance issues related to certain plastic syringes manufactured in China. In March 2024, the FDA issued (i) an updated safety communication warning of issues related to certain plastic syringes manufactured in China; and (ii) a number of warning letters related to the sale and distribution of unauthorized plastic syringes made in China that have not been cleared or approved by the FDA.

Coverage and Reimbursement

Whether government or commercial payers are willing to pay for a product, how much they are willing to spend, and under what circumstances:

Pharmaceuticals and Biologics

• Via our Inflation Reduction Act webpage, we continue to follow how the Inflation Reduction Act of 2022 (IRA) will affect reimbursement for pharmaceutical and biologic products. This includes the continued and ongoing price negotiations for the first 10 drugs selected, which we anticipate concluding in Q3 2024. We are also actively tracking ongoing constitutional challenges to the IRA’s drug price negotiation program. While there also continue to be a host of legislative proposals regarding the IRA, as of Q2 2024, none appear to be likely to pass through Congress in the near term.
• We continue to monitor the backlash from the December 2023 National Institute of Standards & Technology proposed framework for how government agencies can implement “march-in” rights under the Bayh-Dole Act and the White House statement released on the same day indicating that it plans to use such march-in rights to combat pharmaceutical pricing, signaling that the government will consider marching in and assigning drug patents to others to the extent that a particular drug has been funded by taxpayers and the government considers the price to be too high. See “March-In” Rights Under the Bayh-Dole Act: Implications for Pharmaceutical and Medical Technology Companies.”
• We continue to monitor the Department of Health and Human Services’ (HHS) proposed rule that would modify the current alternative-dispute-resolution process under the 340B Drug Pricing Program. The program has been the subject of much scrutiny and disagreement among pharmaceutical manufacturers (who are obligated to cap prices significantly for patients of qualified covered 340B public health entities) and the public health entities whose patients benefit from reduced 340B pricing. The proposed rule would simplify and expedite the process by which covered entities and drug manufacturers resolve disputes about overcharging, duplicate discounts, or drug diversion.
• We are monitoring any future litigation developments in the Genesis Health Care v. Becerra ruling in November 2023 that deemed the interpretation of eligible 340B patients by the Health Resources and Services Administration (HRSA) to be too narrow. In response to this case, the HRSA announced a new webpage with program policies, guidance documents, and regulations for stakeholders to assist 340B covered entities with the meaning of “eligible patient” under the 340B program. Here is a link to the webpage for 340B Patient Definition Compliance Resources.
• Congress is considering numerous proposed pharmacy benefit manager (PBM) reforms in both the House and the Senate. Legislative proposals include but are not limited to: eliminating rebates; divorcing service fees from the price of a drug, discount, or rebate; prohibiting spread pricing; limiting administrative fees; requiring PBMs to report formulary placement rationale; promoting transparency; and many others. PBM reform and transparency measures have become a key policy topic in Washington and continue to be front and center for policy makers.
• We continue to monitor a proposed rule issued by CMS in 2023, “Misclassification of Drugs, Program Administration and Program Integrity Updates Under the Medicaid Drug Rebate Program (MDRP)” (the Proposed Rule). Among other topics, the Proposed Rule focuses on enhancing MDRP administration by incorporating policies that would facilitate more accurate and consistent drug information reporting, data collection, and operations. One of the most significant proposals is to revise the definition of “best price” to provide that manufacturers must aggregate cumulative discounts, rebates, or other arrangements to determine the final price realized by the manufacturer for a particular unit of a drug. The CMS fact sheet is available here. Comments on the Proposed Rule were due by July 25, 2023.
• We are monitoring the implementation of Regulation (EU) 2021/2282 (the HTA Regulation) on health technology assessment (HTA), which came into force on January 11, 2022, in the EU and will apply as of January 12, 2025. An HTA is a multidisciplinary process that summarizes information about the medical, social, economic, and ethical issues relating to the use of a health technology in a systematic, transparent, unbiased, and robust manner. Currently, and until the applicability of the HTA Regulation in 2025, a manufacturer, after obtaining the respective authorization for health technologies, must apply to different HTA agencies in various EU member states before new health technologies are broadly accessible. The HTA Regulation aims to harmonize various procedures and standards by ensuring health technology developers can submit only once, at the EU level, any information, data, analyses, and other evidence required for the contemplated joint clinical assessment.

Medical Devices and Medtech

• We continue to monitor developments related to CMS’s proposed coverage regulations from 2023 that would permit earlier coverage of medical technology that has received FDA breakthrough designation. We anticipate a final rule sometime in 2024.

Compliance, Fraud and Abuse, and Transparency

Companies’ interactions with their customers and other stakeholders:

• The DOJ has taken a greater interest in pharmaceutical companies’ sponsored-genetic-testing programs that help in confirming diagnoses and vetting patients for appropriate treatment. The DOJ settled an anti-kickback statute case against Ultragenyx in December 2023, and recent reports indicate that drug company Biomarin has also received DOJ subpoenas related to its own sponsored-testing program

Privacy

Companies’ interactions with patient data:

• With companies collecting patient data on a global basis, we closely follow the latest privacy law developments around the world. In the EU, the UK, and Switzerland, we are tracking evolving case law and guidance surrounding the EU and UK General Data Protection Regulation and the Swiss Federal Act, as well as implications for companies collecting and handling patient data. In the US, the post-Dobbs era is marked by intense regulatory focus on health data. This includes state consumer privacy legislation protecting sensitive health information, health-data-specific laws such as the state of Washington’s My Health My Data Act that comes into force on March 31, 2024 (see Goodwin’s client alert for more information), and a wave of Federal Trade Commission enforcement actions against digital health providers. In addition, on March 18, 2024, the Health and Human Services Office for Civil Rights (“HHS-OCR”) revised its December 2022 Bulletin on tracking technologies, seemingly expanding the circumstances in which HHS-OCR would consider individually identifiable information concerning an unauthenticated visitor to the website of a covered entity or business associate to be Protected Health Information (PHI) (see Goodwin’s client alert for more information).
• The European Council and European Parliament have reached a provisional agreement on the European Health Data Space (EHDS). The European Commission published a proposal for the EHDS back in May 2022 and now we’re reaching the final stages. The main objectives of the EHDS is to improve individual access and control over patient health data in the EU and to facilitate cross-border research and innovation in health. The provisional agreement will need to be endorsed by the European Council and European Parliament before it is formally adopted. See Goodwin’s blog posts here and here for further details.
• On July 10, 2023, the European Commission adopted an adequacy decision for the new EU-US Data Privacy Framework (DPF), the revamped transatlantic framework designed to support transfers of personal data from the EU to US companies that self-certify compliance with the DPF’s privacy requirements. This has replaced the prior, invalidated Privacy Shield Framework. See Goodwin’s blog post for more information. This is an important update for life science companies because the previous Privacy Shield Framework excluded key-coded patient data from its scope, which prevented sponsors, clinical research organizations (CROs), and other parties involved in transfers of patient data to the US from certifying to the old framework. However, the new DPF has extended its framework’s protection to key-coded patient data processed under a clinical trial, allowing sponsors, CROs, and researchers to certify to the DPF. Separately, the UK and the US have finalized a UK-US adequacy agreement, which took effect on October 12, 2023. The UK-US adequacy agreement opens up transatlantic data flows between the UK and the US.

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