Elements of a Clinical Trial Transparency Policy - Requirements and Expectations

Previous article in the series Elements of a Trial Transparency Policy – Overview

Summary of Requirements and Expectations

In order to develop a comprehensive transparency policy, it is essential to know the requirements of various international organizations.

International Committee of Medical Journal Editors (ICMJE)

The ICMJE requires that all protocols for trials included in a manuscript must be posted on a recognized public registry by the start of enrollment; otherwise, the manuscript will be rejected.

Additionally, the ICMJE requires that sponsors provide a “Data Sharing Statement” with the manuscript that describes in detail the commitment to sharing clinical data related to the trial(s) in the manuscript, including:

• Will individual patient data (IPD) be made available?
• What data will be shared, and what other documents will be made available?
• When will it be available?
• Who may request the data and for what types of analyses?
• How can the data be accessed?

NOTE: A lengthy list of journals that are not ICMJE members also follow these requirements.

The Declaration of Helsinki

The World Medical Association (WMA) Declaration of Helsinki requires that every research study involving human subjects be registered in a publicly accessible database before recruitment of the first subject and states that researchers have a duty to make publicly available the results of their research. In effect, this means that all trials in humans (including Phase 1 healthy volunteer trials) must be registered and results must be disclosed. Additionally, the declaration now requires that all medical research subjects should be given the option of being informed about the general outcome and results of the study, which is generally understood to mean providing trial participants with plain (lay) language summary of results.

Industry groups

Industry groups such as the Biotechnology Innovation Organization (BIO), the Pharmaceutical Research and Manufacturers of America (PhRMA), the European Federation of Pharmaceutical Industries and Associations (efpia), and the Association of the British Pharmaceutical Industry (ABPI) have made disclosure commitments on behalf of their members.

The PhRMA/efpia principles for responsible data sharing include:

• Sharing clinical trial data and documents with qualified scientific and medical researchers upon request
• Working with regulators to provide a factual summary of clinical trial results to patients who participate in clinical trials
• Making the synopses of clinical study reports for clinical trials in patients submitted to the Food and Drug Administration, European Medicines Agency, or national authorities of EU member publicly available upon the approval of a new medicine or new indication
• Publishing clinical trial results regardless of the outcome; at a minimum, results from all Phase 3 clinical trials and clinical trial results of significant medical importance should be submitted for publication

For BIO members, the principles include:

• A commitment to register all company-sponsored clinical trials conducted on patients on an appropriate registry such as ClinicalTrials.gov or the European Clinical Trials Database (EudraCT)
• Disclosing results for approved products or after discontinuation of development
• Submitting for publication in the scientific literature, or otherwise make available to the scientific community, results of all Phase 3 trials and clinical studies of significant medical importance regardless of whether their outcomes are positive or negative
• For their approved medicines, sponsors will fulfill qualified requests from medical and scientific researchers for additional clinical trial data (e.g., clinical study reports, patient-level clinical datasets, clinical study designs, protocols, etc.) beyond those shared proactively with the public.
• Providing factual summaries of clinical trials to research participants

Transparency advocates

Regular publications in medical journals and articles in the general media assess and occasionally criticize the state of clinical trial disclosure. Increasingly, these publications analyze the disclosure practices of individual sponsors, publish their findings with details of specific trials and companies.

The ongoing scrutiny of disclosure practices is shaping the transparency policies of a growing number of trial sponsors. By pointing out potentially noncompliant trials, these trackers are also informing inspections and compliance audits, as well as impacting public perception and reputation.

Patient groups

While no single advocacy group represents all patients, common clinical transparency expectations include the public disclosure of:

• Protocol summary information for all trials conducted in patients with up-to-date site contact & enrollment information, plain language information describing the trial, and clear inclusion/exclusion criteria
• Plain (lay) language summaries (PLS) for all interventional trials
• Study synopses (CSRs) or basic results for all interventional trials
• Informed consent forms (ICF)
• Links to publication

Next article in the series: Protocol Disclosure Recommendations