Pharma Trade Group Giants Update Principles on Clinical Data Sharing

Peter O'Donnell, ACT's Brussels Correspondent

Research-based pharmaceutical companies in Europe and the US have updated their commitment to share clinical trial data.¹ They first published their so-called “Principles for Responsible Clinical Trial Data Sharing” in 2014 as a response to growing pressure from healthcare campaigners, academia, and politicians for greater transparency. Since then, the companies belonging to the two trade associations involved—the European Federation of Pharmaceutical Industries and Associations (EFPIA) and the Pharmaceutical Research and Manufacturers of America (PhRMA)—say they have developed their own practices for data sharing that go beyond their initial commitments. The statement of principles is accordingly being updated and reworded to reflect that.

The new version of the principles also emerges alongside new legislation—and notably, in Europe, the increasing impact of the 2014 clinical trials regulation, as that comes progressively into effect, bringing with it specific new transparency requirements for trial sponsors, particularly in respect of the prompt publication of results.

The modified industry document reflects the—voluntary and carefully caveated—approach of biopharmaceutical companies that claim to enhance public health “through responsible sharing of clinical trial data”in a way that safeguards patient privacy, and that at the same time respects national regulatory systems and maintains incentives for investment. The underlying tensions between conflicting goals in disclosure are unmistakeable.

Sharing data with researchers will take place, according to the new version of the principles, upon request—but only from “qualified scientific and medical researchers”—and consist of patient-level clinical trial data, study-level clinical trial data, and protocols from clinical trials in patients for medicines and indications approved in theUS and the EU, and only“as necessary for conducting legitimate research.” Requests will be reviewed to determine whether they meet the criteria, with the participation of a scientific review board that includes scientists and/or healthcare professionals who are independent of the company concerned. Any patient-level data that is shared will be anonymizedand companies will not be required to provide access to patient-level data if there is a reasonable likelihood that individual patients could be re-identified.

Data requestors will be required to document “the legitimacy of the research question and the qualifications of the requestor,”including a description of the data being requested, the hypothesis to be tested, the rationale for the proposed research, the source of any research funding, and“any potential conflicts of interest, including potential competitive use of the data.” In a supplementary note to the principles, the trade associations insist that “in a sustainable research ecosystem, companies must be certain that their proprietary information will remain secure from disclosure to competitors.”

Researchers who are provided access to company data will be expected to publish the results of their analysis so that it may be peer-reviewed and to consider publication via an open access format. And there are further constraints: researchers must agree not to transfer the shared data or information to parties not identified in the research proposal, use the data for purposes not contained in the research proposal, or seek to re-identify research participants.

Public access will be “enhanced,” say the principles, by companies providing synopses of clinical study reports (CSRs) following approval of a new medicine or new indication for an approved drug in the US and EU. But “appropriate redaction” will be employed so the publication is “consistent with the need to protect patient privacy, publication rights, and confidential commercial information.” Requests for full CSRs, and for patient-level andstudy-level data, will be subject to review. Biopharmaceutical companies also undertake to“work with regulators” to adopt mechanisms for providing a factual summary of clinical trial results and make the summaries available to research participants.

All company-sponsored clinical trial results should be considered for publication in the scientific literature irrespective of whether the results of the sponsors’ clinical trials are positive or negative so that it may be peer-reviewed. Companies should consider using an open access format. At a minimum, results from all Phase III clinical trials and any study results of significant medical importance should be submitted for publication. This commitment also pertains to investigational medicines whose development programs have been discontinued.The principles also confirm the industry view that “all company-sponsored clinical trial results should be considered for publication in the scientific literature irrespective of whether the results of the sponsors’ trials are positive or negative so that it may be peer-reviewed.” At a minimum, the new version continues, results from all Phase III clinical trials and any trial results of significant medical importance should be submitted for publication.

Many of these principles and commitments might be considered unremarkable, since for the most part they coincide with established views of sound science. The fact that they have to be proclaimed at all—and with such a pretension to virtue—is indicative of how far the world still is from a genuinely transparent drug development system.

Peter O'Donnell is a freelance journalist who specializes in European health affairs and is based in Brussels, Belgium

Reference

1. https://efpia.eu/media/qndlfduy/phrmaefpiaprinciplesforresponsibledatasharing2023.pdf