On June 24, 2026, the Office of Inspector General (“OIG”) of the Department of Health and Human Services (“HHS”) issued a Request for Information (“RFI”) seeking stakeholder input on payments received by individuals in connection with clinical trial participation. Specifically, OIG is seeking to identify ways it might: (i) modify or add new safe harbors to the Federal anti-kickback statute (“AKS”) or exceptions to the civil monetary penalty provision prohibiting inducements to beneficiaries (the “Beneficiary Inducements CMP”); or (ii) issue or revise guidance addressing arrangements intended to facilitate clinical trial participation while safeguarding against fraud and abuse.

The RFI follows on the heels of HHS’s Operation TrialBlazer initiative, which has identified patient access and engagement as a priority area for revitalizing U.S. clinical research. That initiative has already acknowledged that trial participation imposes real financial burdens on patients (i.e. things like cost-sharing, unplanned tax consequences from trial-related payments, and potential effects on program eligibility (e.g., Medicaid)).  Accordingly, OIG appears to be leveraging this RFI to explore whether the fraud and abuse rules themselves serve as a form of barrier or burden to Americans’ – and especially government beneficiaries’ – ability to access clinical research.

Current OIG Thinking and Issue at Hand

• The AKS and the Beneficiary Inducements CMP were both written broadly, and neither statute carves out an exception for remuneration tied to clinical trial participation. As a result, OIG currently treats remuneration provided to clinical trial participants, including cost-sharing waivers, transportation, childcare expenses, and stipends, as potentially falling within the broad reach of the AKS and the Beneficiary Inducements CMP, meaning such payments may be considered presumptively suspect unless they fit within an existing safe harbor or exception.
• OIG has issued 10 favorable advisory opinions over the past two decades allowing for certain forms of cost-sharing waivers for clinical trial participants, but has not addressed other forms of remuneration to clinical trial participants. Further, while these opinions establish that OIG is comfortable with cost-sharing relief in the right circumstances, the advisory opinions themselves are fact-specific and binding only on the requesting party.

OIG’s Request

Rather than proposing specific regulatory text, OIG has opted to cast a wide net.  The RFI poses 14 questions spanning from whether remuneration works as an enrollment tool in the first place to the mechanics of a potential safe harbor (i.e. value caps, permissible payors, IRB oversight, and clinical research phase-specific distinctions).  OIG has asked that commenters back their positions with data, studies, or concrete examples where possible.  In other words, a generalized comment expressing support for “more flexibility” is unlikely to carry the same weight as specifics tied to actual trial experience.

The RFI poses 14 specific questions to stakeholders, and OIG asks that commenters support claims with relevant data, studies, analyses, and other citations.

Topics covered include:

• Whether offering Federal health care program enrollees remuneration actually facilitates clinical trial participation;
• Whether the AKS or Beneficiary Inducements CMP are perceived as barriers to providing appropriate remuneration;
• What categories and levels of remuneration (e.g., travel, lodging, childcare, stipends, time compensation) are useful to facilitate participation, and which carry heightened fraud and abuse risk;
• What value caps, limits, or restrictions on who may provide remuneration would guard against fraud and abuse;
• The role of Institutional Review Boards (IRBs) as a safeguard over the type, amount, and frequency of remuneration;
• Whether remuneration considerations differ across trial phases (Phase 1–4) and types of trials (e.g., government-sponsored vs. industry-sponsored);
• Whether safeguards are needed to prevent participants from being steered toward non-trial items or services offered by the party providing the remuneration;
• Whether limitations on advertising remuneration are needed to protect trial integrity;
• What additional or modified safe harbors or CMP exceptions may be necessary, and what key provisions they should include; and
• Whether OIG could address some issues through guidance (e.g., Special Advisory Bulletins, FAQs) rather than formal regulation.

Stakeholder Considerations

OIG has signaled openness to addressing some issues through a Special Advisory Bulletin or FAQ rather than formal rulemaking. Given the length of the rulemaking process, stakeholders may consider advocating for interim guidance as the most pragmatic path to near-term certainty for sponsors currently designing or enrolling trials.

This RFI is a particularly timely opportunity for rare disease-focused sponsors, sites, and patient advocacy organizations.  For example, qualified treatment centers for certain rare disease therapies are often limited to a small number of sites nationally (or globally), and participants frequently face significant travel and lodging burdens, caregiver support needs, and other questions about remuneration that OIG can squarely address through this guidance.  Rare disease stakeholders may be best positioned to give OIG concrete, data-supported input on categories like qualified-treatment-center travel support and caregiver assistance that general commentary from more geographically distributed therapeutic areas may not capture.

Next Steps

Stakeholders wishing to submit comments must do so no later than August 24, 2026 at 5:00 p.m. ET (60 days from the Federal Register publication date of June 24, 2026).

Interested stakeholders should reach out to Matt Wetzel (mwetzel@goodwinlaw.com) and Amelia Nell (anell@goodwinlaw.com) with any questions or assistance with preparing and submitting a response to OIG’s RFI.

On April 15, 2026, the U.S. Food and Drug Administration (“FDA”) announced that it will convene a public meeting of the Pharmacy Compounding Advisory Committee (“PCAC”) on July 23-24, 2026, to consider whether to recommend seven (7) peptide bulk drug substances for inclusion on the list of substances that may be used in compounding under Section 503A of the Federal Food, Drug, and Cosmetic Act. Five (5) additional peptide and peptide-derived substances are also slated for separate PCAC evaluation through early 2027. On the same date, FDA also republished its interim 503A Bulks List, indicating its intent to remove 12 peptides from its Category 2 list (“Bulk Drug Substances that Raise Significant Safety Concerns”).  This removal took effect following a seven calendar-day notice period.

These FDA actions follow comments in February by HHS Secretary Robert F. Kennedy Jr. on a show hosted by Joe Rogan, during which he expressed concerns about the gray-market nature of the current supply of certain peptide products and that he hoped there would be a pathway for consumers to obtain these products from “ethical suppliers”.

The seven (7) peptide bulk drug substances expected to be considered by the PCAC in July, together with the uses identified for evaluation, are:

July 23, 2026:

• BPC-157 (free base and acetate): ulcerative colitis
• KPV (free base and acetate): wound healing and inflammatory conditions
• Thymosin Beta-4, Fragment (LKKTETQ)(“TB-500”) (free base and acetate): wound healing
• MOTs-C (free base and acetate): obesity and osteoporosis

July 24, 2026:

• Emideltide (DSIP) (free base and acetate): opioid withdrawal, chronic insomnia, and narcolepsy
• Semax (free base and acetate): cerebral ischemia, migraine, and trigeminal neuralgia
• Epitalon (free base and acetate): insomnia

The peptide substances scheduled for consideration at the July 23-24, 2026, PCAC meeting are among those identified for removal from Category 2. FDA has also identified five (5) additional peptide and peptide-derived substances, including Cathelicidin LL-37, Dihexa acetate, GHK-Cu (injectable), Mechano Growth Factor (PEG-MGF), and Melanotan II, for removal from Category 2 and for later consultation with the PCAC at an advisory committee meeting expected before February 2027.

For those substances removed from the Category 2 list, such removal does not, in itself, establish eligibility for compounding under Section 503A.  The regulatory status of these substances remains subject to further evaluation by FDA. FDA had previously identified a range of potential safety considerations associated with these substances, including risks related to immunogenicity, peptide-related impurities, and, in some cases, limited human exposure data. Against that backdrop, FDA’s removal of these substances from the Category 2 list and its decision to seek PCAC input regarding their potential inclusion on the 503A bulks list suggests that the Agency is continuing to evaluate how such considerations should be weighed in light of other available data.

Inclusion of any substance on the 503A bulks list historically has been implemented through rulemaking, with advisory committee input forming part of that process. Although PCAC’s recommendations are not binding, they generally have informed FDA’s ultimate determinations. Accordingly, the upcoming PCAC deliberations may provide an important indicator of how FDA intends to approach these substances going forward.

FDA has also opened a public docket (FDA-2025-N-6895) in connection with the upcoming PCAC meeting. Comments submitted by July 9, 2026 will be provided to the Committee for consideration, and the docket will remain open for submissions through July 22, 2026.

If you have any questions or would like to submit a comment to the public docket, please contact the authors or the Goodwin attorney with whom you typically work.

On July 10, 2025, the U.S. Food and Drug Administration (FDA) announced publication of over 200 complete response letters (CRLs) issued in response to applications submitted to FDA for approval of drugs or biologics between 2020 and 2024. The FDA has described this move as a step toward the Agency’s “broader initiatives to modernize and increase transparency.”

CRLs are formal communications sent to applicants when the FDA has completed its review of an application but determined that it cannot approve the application in its current form. Until now, the Agency has only made CRLs available as part of larger approval package files on the Drugs@FDA online database (i.e., after product approval). While the CRLs released this week continue to be limited to approved products—and have been redacted to remove trade secrets and confidential commercial information—the FDA has, for the first time, provided these documents in a central database on openFDA. A few key highlights:

• While many of these CRLs have already been disclosed as part of the “Other Action Letters” section of publicly posted drug approval packages, some have not.
• There are multiple CRLs for supplemental New Drug Applications (sNDAs) that had not yet been disclosed, reflecting the fact that approval packages for sNDAs are not consistently posted in the same manner as original NDA approvals.
• Some of these CRLs were issued for products approved before 2020, suggesting that the CRL database scope may exceed the time frame identified in the FDA’s announcement.
• At least one CRL has been posted for a product approved as recently as June 2025. For this product, no other portions of the approval package (beyond the label and approval letter) have yet been posted on Drugs@FDA.

Notably, the FDA’s announcement references a 2015 analysis conducted by FDA researchers, which found that sponsor disclosures of CRLs did not consistently provide full detail regarding the Agency’s specific concerns. The FDA’s highlighting of this finding, coupled with the Agency’s statement that it plans to publish additional CRLs from its archives, warrants attention from sponsors, especially public company sponsors.

Sponsor disclosures regarding CRLs are always closely scrutinized, and the FDA’s move to (1) centralize and regularly release CRLs, and (2) publish additional CRLs (e.g., those for sNDAs, or very recently approved products) is likely to invite further scrutiny—by investors, analysts, competitors, and patient communities. Sponsors should prepare disclosures around receipt of a CRL with the expectation that the CRL itself will become public upon approval of an application. Even where a product is ultimately approved, third parties may make comparisons between a sponsor’s characterization of a CRL and the later-posted CRL itself.

According to the FDA, publication of CRLs is just one step in the Agency’s broader transparency push. Our team will continue to monitor the frequency and scope of additional releases, as well as any opportunities for interested stakeholders to provide comments or feedback to FDA on its plans.

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.

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.

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.