Regulatory Resources

Regulators hold enormous amounts of data, and some are making them publicly accessible.  Some of the data regulators hold are those submitted to them by sponsors (e.g. clinical study reports), whereas other types of regulatory data are produced by regulators in the course their work (e.g. FDA medical officer reviews).

This webpage describes the types of data regulators hold, and how you can obtain them.

US Food and Drug Administration

• FDA reviews in Drug Approval Packages (for drugs).  Drug “approval packages” (also called “action packages”) contain multiple reviews regulators have written in the course of evaluating a sponsor’s application. Medical officer reviews and statistical officer reviews are often an important source of both clinical trial data and FDA analyses/reviews of those data, and other reviews from other scientific disciplines (e.g. clinical pharmacology, toxicology) are also available.  FDA publishes these on its Drugs@FDA website.  For instructions and a video on how to use Drugs@FDA, we recommend two articles: Ladanie et al. (2018) and Turner (2013). In 2019, the FDA announced a decision publish “integrated reviews” in lieu of separate reviews by individual scientific disciplines, a move that has been criticized by academics and researchers (including from the RIAT Support Center).
• FDA reviews (for biologics).  Similar to the Drugs@FDA, FDA makes some of its reviews (e.g. clinical reviews) available for vaccines and other biologics. These can be found by looking for “Supporting Documents” under the product’s webpage. FDA maintains a list of licensed biological products with supporting documents here.
• Pediatric data.  Additional FDA medical, statistical, and clinical pharmacology reviews of some pediatric studies are made available across multiple pages on FDA’s website: Pediatric Studies BPCA (2002-2007), Pediatric Studies BPCA and PREA (2007-2012), and Pediatric Studies BPCA and PREA (2012 – Present).  These studies apparently appear on this section of FDA’s website (and not Drugs@FDA) because the pediatric studies were conducted under the BPCA (Best Pharmaceuticals for Children Act) or PREA (Pediatric Research Equity Act).
• Advisory committee meeting materials (for drugs, biologics, devices, and more).  The FDA has a system for receiving external advice on its regulatory functions.  For example, before deciding whether to license a new drug or new indication of an approved drug, the FDA may convene an advisory committee.  Agendas, transcripts, and presentation materials (e.g. slides submitted by both the sponsor and the FDA) for these meetings are made publicly available, and can be another source of regulatory data valuable to RIATers.  Unfortunately, advisory committee meeting materials are difficult to find as there is no central database of these materials. Documents are instead linked from the advisory committee meeting webpage on FDA’s website.  Therefore knowledge of which advisory committee is relevant to the medical product you are interested in, and the date(s) that committee met to discuss that medical product are two important details needed to discover all available advisory committee meeting materials.  If you do not know whether the medical product you are interested in was discussed at a FDA advisory committee, searching the web can help (for example, a search for “oseltamivir FDA advisory committee” reveals a webpage that mentions a November 18, 2005 Pediatric Advisory Committee meeting; ultimately, after following the links from FDA’s website to the pediatric advisory committee, to past meeting materials, one can find all the materials from the Nov 18, 2005 meeting).
• Clinical study reports and other trial data. The FDA does not routinely release clinical study reports or other trial data, even in response to a freedom of information request.  We are only aware of one occasion where some parts of a CSR were released in response to a FOIA request, and a second occasion when CSRs were released under FOIA but only after the requestors sued. In January 2018, the FDA announced a limited pilot program to publish some CSRs, however in March 2020, after receiving feedback about this pilot program, the FDA decided to conclude the pilot.

European Medicines Agency

The EMA has two major policies that enable access to clinical study reports and other regulatory documents: Policy 0043 and Policy 0070.

• Policy 0043 governs the agency’s approach to the retrospective release of certain documents (some people call these “legacy” documents) when in the agency’s possession.  The policy was open to anybody, irrespective of citizenship, since 2010, but as of June 15, 2018, the EMA will no longer process access to documents requests issued from outside the EU. In colloquial terms, Policy 0043 is the agency’s freedom of information policy. Any document in the agency’s possession can be requested including Clinical Study Reports, other parts of the Common Technical Document including Modules 2.5 and 2.7, and Periodic Safety Update Reports.  Requests for documents under Policy 0043 should be submitted by filling out this simple online form on EMA’s website.  Your request will be assigned to the Access To Documents (ATD) team at EMA and given a tracking number.
  • RIAT Support Center advice on requesting trial data from EMA under policy 0043:  The more specific you are about what you are requesting, the quicker it will be handled by EMA.  For example, are you interested in receiving the CSR with all or just some appendices? Knowing the names of the appendices therefore becomes important to facilitate clarity with EMA regarding the scope of your request.  However, the difficulty is that requestors often do not know what EMA holds. A good way to start is first familiarize yourself with the layout of a clinical study report by looking at samples and the ICH E3 document.  Then, in your query to EMA, ask whether the agency can list its holding of the drug/biologic of interest.  A simple query, “Dear EMA, I am interested in obtaining clinical data for the drug X. Can you tell me for which trials you hold clinical study reports, and for each trial, which sections of clinical study reports you hold?” is a straightforward way to inquire about EMA’s CSR-related holdings for a given medicine.
• Policy 0070 – EMA are prospectively making available documents relating to compounds on which it has made a decision under the centralised procedure since 1 January 2015 here. This program is now known as Clinical Data Publication. Anyone can register and view the documents but download of documents is reserved to those domiciled in the EU. What’s on offer? The typical document package for download includes various trial documents: one or more CSR main bodies, protocols and statistical analysis plans and Modules 2.5 and 2.7 of the Common Technical Document, affording an overview of the study program and its rationale. The anonymisation report completes the set. This is a slim, sponsor-generated document telling you what has been redacted/anonymised, how and why, in the released version of the trial documents.

In addition to these sources, European Public Assessment Reports (EPARs) are detailed assessments of all medical and veterinary compounds assessed the European regulator EMA and on which a decision has been made. EPARs are readily accessible with no restrictions and contain details of EMA’s scientific assessment of the index compound.

Therapeutic Goods Administration (Australia)

Australian Public Assessment Reports for prescription medicines (AusPARs) are modelled on EPARs. As with an EPARs, an AusPAR provides the rationale for the regulatory decisions made by the Australian regulator Therapeutic Goods Administration (TGA). In contrast to EPARs, AusPARs are written for each market application and are prepared by TGA in conjunction with the sponsors and are tens of pages long, although they may contain attachments. Useful information such as timelines and a summary of the evidence development programme are also included in the document.

Health Canada

Clinical trial data for drugs and medical devices. Health Canada posts clinical trial data to its website and it can be freely downloaded by anybody around the world.  This practice, which began in 2019, applies to drugs and medical devices and has made Health Canada arguably the world’s most transparent regulator, and the only regulator proactively sharing data on medical devices. “Clinical information,” as Health Canada calls the data, is proactively released following completion of Health Canada’s regulatory review, and occurs whether or not the regulator approves or rejects a sponsor’s application. The transparency policy that set this in motion is being phased in for different submissions and product types over four years.

For previously reviewed drugs or medical devices, you can request data from Health Canada by submitting a request.  (One does not need to be a Canadian citizen.)

Prior to these advances, Health Canada only shared clinical trial data if the requestor was prepared to sign a confidentiality agreement, which permits the requestor to publish research results based on the data, but not the data itself. However the legality of requiring a confidentiality agreement was successfully challenged in a 2017-2018 lawsuit brought by Peter Doshi (of the RIAT Support Center) against the Canadian government.  For those interested, we wrote a blog about the process of getting clinical trial data and another blog following the court’s decision in Doshi’s favor.

Other regulatory documents. Health Canada also shares its Summary Basis of Decision documents which are issued after a new drug or medical device is approved.  These documents are authored by the regulator and explain the scientific and benefit/risk information that was considered prior to approving the product.

Finally, the Health Canada website also makes available product monographs which are a product’s prescribing information, analogous to “drug labels” or “package inserts” as they are known in other countries.

Pharmaceuticals and Medical Devices Agency (Japan)

The Japanese medicines regulator PMDA makes a variety of regulatory data publicly available.  While most is in Japanese, the agency does post English translations of regulatory review reports (similar in scope to FDA reviews) for some (but not all) drugs, medical devices, and regenerative medicines that can be a valuable source of information about trials.