Transparency Makes Modern Regulatory Science Stronger — FDA’s Transparency Roadmap

FDA Voices

By: Lowell M. Zeta, JD, Acting Chief of Staff, Deputy Commissioner for Strategic Initiatives and Special Counsel

Transparency — a key component of any healthy government agency — is especially relevant in the context of the U.S. Food and Drug Administration’s mission to protect public health. Visibility into the FDA’s work and reasoning helps drive informed decision-making for stakeholders across the healthcare landscape, including patients considering whether to enroll in a clinical trial, families and caregivers weighing treatment options, clinicians interpreting labeling, and investors deciding where to allocate capital.

Transparency is also inseparable from the FDA’s role in promoting American innovation. We are living through a moment of opportunity: innovation in medicine has never moved faster. Breakthroughs in cell and gene therapy, Super Intelligence (SI)-assisted drug design, decentralized clinical trials, and precision diagnostics are creating more ways than ever before for Americans to improve their health — and for the United States to advance the health of its population and to maintain its global leadership in biomedical science. An agency that is open about its reasoning and processes is one that innovators can navigate with confidence, that investors can trust with capital, and that patients can rely on for honest answers. Transparency is therefore not merely a good-government value — it is an accelerant for the next generation of medical innovation.

Lowell M. Zeta, J.D.

Transparency is a goal recognized at the highest levels of the Trump Administration. The MAHA Report issued in 2025 by the President’s Make America Healthy Again Commission, called for radical transparency across federal health agencies as a cornerstone of health policy reform and rebuilding public trust. Transparency is also an essential part of the FDA’s core strategic priorities — standing as a key tool to ensure Americans have access to safe, effective, and affordable medical products. For these reasons, the FDA is taking a bold step to reinforce its commitment to transparency by releasing this new FDA Transparency Roadmap — a forward-looking account of FY26 milestones achieved and FY27 priorities ahead.

The Transparency Roadmap reflects the agency’s intention to maximize disclosure, guided by what is appropriate and good for public health. It was shaped by soliciting ideas and expertise from innovators across the agency, and it may continue to evolve as we seek the best ways to inform the public and protect public health while maintaining IP protections that cultivate a healthy innovation environment.

Transparency Roadmap

1.    Complete Response Letters

The FDA is committed to being as forthcoming as possible about the scientific basis for its regulatory determinations, to help address what some have seen as a historical imbalance in what information the public sees about FDA-regulated products. A 2015 internal analysis found that 85 percent of the FDA’s safety and efficacy concerns were not mentioned by sponsors when publicly announcing non-approvals — a pattern that risks misleading patients and investors alike.

A complete response letter (CRL) is a communication sent by the FDA to a sponsor when, after a thorough review, the agency determines that it cannot approve a marketing application in its current form. The letter describes the specific, substantive deficiencies — such as safety or efficacy issues or manufacturing problems — that led to that determination.

In 2025, the FDA launched a CRL disclosure initiative — beginning with the disclosure of more than 200 letters covering applications submitted between 2020 and 2024. It was a watershed moment. CRL disclosure is now a pivotal mechanism for transparency, providing appropriate and useful information to all stakeholders while ensuring confidential or proprietary information remains protected.

There are multiple benefits of CRL disclosure. Sponsors and researchers, for instance, can observe and learn from the FDA’s evidentiary reasoning across prior decisions, so they can design more efficient development programs, reduce wasted resources, and bring safe and effective products to patients faster. Disclosure also encourages companies to be forthright with investors and stakeholders about the regulatory status of their products. For patients, families, and trial participants, it can be helpful to understand why a potential treatment for their condition was not approved. The growing body of disclosed CRLs is a public compendium of the FDA’s applied regulatory science — with immediate practical value for any sponsor navigating a similar development pathway.

Companies might issue press releases for complete response letters or other documents on the basis (at least in part) of federal laws that require them to disclose such information to investors. These decisions are often made through internal discussions among company leadership. The FDA will be mindful of such considerations in determining timing for the release of redacted documents. Further, through rulemaking, we aim to increase transparency surrounding New Drug Applications (NDAs), Biologics License Applications (BLAs), Abbreviated New Drug Applications (ANDAs), and related applications through public disclosure of the fact of submission of all major applications.

On all fronts, the FDA wants to enable sponsors to better navigate evolving regulatory science in development and avoid common pitfalls that might burden already limited resources or otherwise delay promising treatments.

2.    Investigational Stage and Marketing Application Lifecycle Disclosures

The agency is also pursuing greater transparency across the application lifecycle. We are encouraging sponsors to make public the existence of an Investigational New Drug Application (IND) upon filing. About 96% of novel drug or biologic filings are already disclosed by the company or third parties. Early transparency in these respects supports trial enrollment and scientific collaboration, and reduces misinformation.

We’re working to increase compliance among medical product companies and researchers with requirements to report certain information about their clinical trials to ClinicalTrials.gov. Noncompliance has resulted in significant gaps in the public record and a publication bias that obscures the true landscape of medical product development outcomes — overrepresenting successes and underrepresenting failures. This gap can also create a distorted perception of the safety and efficacy of medical products.

External research and scientific reviews can improve clinical practice, increase public confidence in regulatory decisions, and help identify important safety signals. We aim to support independent work by increasing post-approval scientific data disclosures. This includes, with appropriate redactions for confidential commercial and trade secret information, the publication of Clinical Study Reports (CSRs) for approved applications; alignment with EMA data transparency practices where feasible; piloting expanded clinical dataset transparency; and enhancing post-marketing data disclosures.

Transparency also shapes the integrity of information to ensure market accountability. In August 2026, the FDA and the U.S. Securities and Exchange Commission (SEC) signed a Memorandum of Understanding (MOU) establishing a formal framework for sharing nonpublic information between the two agencies. The MOU creates a more direct mechanism for both agencies to fulfill their respective missions.

Under the agreement, the FDA may share appropriate information related to FDA-regulated products, activities, and the individuals and firms involved in their manufacture, distribution, and sale — proactively or upon request. The SEC, in turn, may use that information in reviews of public company filings, enforcement investigations, proceedings, and civil actions. This MOU is a significant milestone. It reinforces the FDA’s transparency commitments by ensuring that companies cannot selectively communicate FDA regulatory developments to markets — and that when they do communicate, they do so accurately and completely. For sponsors, awareness of this formal interagency channel is itself a transparency measure: companies should understand that the FDA’s regulatory record is a relevant input to capital market oversight.

3.    New Approach Methodologies and Novel Biomarkers

The agency intends to expand transparency in regulatory review beyond CR letters, specifically by giving visibility into new approaches to evidence generation without compromising innovation. In emerging areas, transparency has a forward-looking dimension: it accelerates the integration of novel tools into the regulatory framework. To give a key example, we aim to give industry up-to-date insight into successful or unsuccessful uses of new approach methodologies (NAMs) in submissions. In September 2026, the FDA launched the publicly accessible NAMs Database of Use Case Examples — an initial release featuring 25 examples drawn from existing public FDA review materials illustrating how NAMs have been incorporated into specific applications for approved drugs. This builds on the October 2025 launch of CDER’s Streamlined Nonclinical Studies and Acceptable NAMs database and the March 2026 draft guidance on General Considerations for the Use of NAMs in Drug Development.

In the year since the agency released a strategic roadmap for transitioning toward NAMs (which include organ-on-a-chip platforms, SI-powered predictive toxicology, and computational modeling), we’ve released several guidances to help industry accelerate the transition, including a September 2026 direct final rule updating regulatory terminology to explicitly recognize NAMs as valid nonclinical testing methods. Giving sponsors insight into how others are actually leveraging NAMs complements these guidances and facilitates industry adoption as the FDA modernizes its own posture.

In similar fashion, the agency is aiming to provide visibility into the use of real-world evidence (RWE) in submissions, and of decentralized trials that generate evidence through digital health technologies. Consistent with PDUFA VII commitments, the FDA has published annual reports describing aggregate and anonymized RWE submissions to CDER and CBER — providing the field with a clearer picture of how RWE is being used to support regulatory decisions on effectiveness and safety.

Biomarkers and novel endpoints connect traditional regulatory frameworks to the next generation of development tools: NAMs, digital health technologies, SI-driven diagnostics, and real-world evidence platforms. The FDA’s Biomarker Qualification Program (BQP) is intended to provide a predictable, structured pathway for biomarker evaluation, allowing companies to reuse biomarkers within defined contexts-of-use without having to undertake a new evidentiary review.

Encouraging the use of the BQP as well as the Medical Device Development Tool Program through transparency is central to the FDA’s modernization agenda and to improved predictability for companies engaging with the agency.

4.    Patient- and Consumer-Centered Product Labeling

Relatedly, supporting patient-centered labeling is an important priority. Labeling for prescription drugs and all FDA-regulated products should be clear, concise, and accessible for users. We intend to issue a final rule to amend our human prescription drug labeling regulations to require a new type of Medication Guide — “Patient Medication Information” (PMI) — for prescription drug products used, dispensed, or administered on an outpatient basis, including blood and blood components transfused in an outpatient setting. Medication Guides are a type of FDA-approved labeling for prescription drug products distributed to patients. This final rule will establish an FDA-approved, one-page document with standardized format and content to improve public health by providing patients with clear, concise, accessible, and useful written prescription drug product information delivered in a consistent and easily understood format to help patients use their prescription drug products safely and effectively. PMI must be easily read and understood by the general population, including individuals with low health literacy and comprehension levels, and PMI being understandable across diverse patient populations would be a huge win for public health. Patient-centered labeling is the last mile of the FDA’s public health mission — the point at which scientific rigor shapes actionable guidance for real people making real health decisions.

Food transparency also must not be overlooked. The MAHA Commission’s 2025 report identified poor diet and chemical exposure as leading drivers of childhood chronic disease, and called for enhanced evidence-based processes for post-market assessment of ingredients and additives in our food supply. The FDA Human Foods Program (HFP) has developed an ambitious series of priorities that focus on the importance of food and nutrition in improving the health of all Americans. As part of this effort, the agency is advancing a more open, predictable, and consumer-focused approach to food regulation. This approach relies on clear labeling (including revised front-of-pack labeling), transparent ingredient and chemical oversight, accessible nutrition information (including when shopping for groceries online), and greater visibility into safety and compliance activities. Together, these efforts aim to improve public trust, support informed decision-making, and strengthen understanding of how the FDA evaluates and manages risks across the food supply.

5.    Compliance and Inspectional Records

Addressing the direct-to-consumer (DTC) advertising landscape is an example of the intersection between transparency and public health protection. Americans cannot fairly make informed choices about prescription drugs if the information they receive obscures material risks in the name of product promotion.

In September 2025, the FDA launched a crackdown on misleading DTC prescription drug advertisements, ending a period of relative laxity in compliance oversight. This work remains a high priority and continues on two parallel fronts. For industry, the agency is seeking to create a level playing field by preventing bad actors from undermining compliant firms in the marketplace. The agency is also exploring new rules and guidances to provide clarity to industry on how advertising may be evaluated across modern forms of media. For the public, the agency has rigorously stepped up oversight under existing authorities to protect consumers from false, misleading or imbalanced advertising that violates the law.

Regarding FDA inspections, when an FDA investigator observes conditions or practices indicating that an entity manufacturing an FDA-regulated product may be in violation of FDA’s requirements, these observations are listed on an FDA Form 483 that is provided to the entity being inspected at the close of the inspection. We will build on existing inspection observation datasets by proactively publishing redacted Form 483s and additional establishment inspection reports (EIRs) where appropriate. The public has long asked for greater transparency into inspections — particularly in areas such as bioresearch monitoring — and these steps will help deliver.

FDA transparency around inspections also serves an important function in a globalized supply chain. Foreign regulatory authorities routinely request FDA inspection records when making their own market access determinations. Proactive publication of Form 483s and EIRs — with trade secrets and confidential commercial information appropriately redacted — gives these foreign regulatory authorities and the public access to more of the materials that inform the FDA’s compliance decisions. For companies interacting with the agency through compliance processes, greater predictability in how and when inspection findings become public supports proactive remediation and a shared understanding of regulatory expectations.

Conclusion

The Transparency Roadmap expands on previous agency transparency initiatives. In 2009, FDA launched an internal Transparency Task Force aimed at making the agency’s operations, decisions, and data more open and user-friendly for the public. That initiative recognized explicitly what remains true today: meaningful transparency must be balanced against the need to protect industry’s confidential and proprietary information, including trade secrets and commercially sensitive data, as well as sensitive personal health information submitted in regulatory filings.

The aspirations that animated the FDA’s earlier transparency initiative — open, user-friendly, principled disclosure of agency operations and decisions — are still very much alive. This roadmap represents measurable FY26 progress and a concrete FY27 agenda across all areas of agency work and reflects the FDA’s commitment that the American public is entitled to understand the reasoning behind decisions that affect their health and safety. The FDA’s gold standard status globally is inseparable from the credibility that comes with open, rigorous, and principled science.

Transparency makes modern regulatory science stronger — and it shows the American people that we’re fulfilling our promise to protect public health.