GSK to Shine Light on Raw Clinical Trial Data

By Marie Powers
Staff Writer

GlaxoSmithKline plc (GSK) took extraordinary steps toward data transparency Thursday, disclosing plans to accelerate its "open innovation" approach to R&D by allowing researchers access to detailed patient-level data underlying clinical trials both in approved medicines and failed investigational compounds.

The London-based pharma also said it will make its library of potential tuberculosis (TB) compounds freely available to researchers and increase funding for its Open Lab for independent researchers in Tres Cantos, Spain.

CEO Andrew Witty unveiled the measures at a meeting in London hosted by the Wellcome Trust.

Currently, GSK posts summary information on protocols at the outset of each trial and shares abridged findings – positive or negative – on its clinical study register, a freely accessible website, and on clinicaltrials.gov. In addition, the company takes requests for clinical trial data on a case-by-case basis and seeks publication of drug trial results, regardless of outcome, in peer-reviewed scientific journals, according to company spokeswoman Melinda Stubbee.

The new transparency initiative goes much further. Beginning in January 2013, GSK will allow independent researchers to sift through anonymous, raw patient data and conduct their own analyses of clinical trials results both in approved medicines and halted compounds. To keep the lawyers at bay, researchers will be required to submit requests, which will be vetted for scientific merit by an independent panel of experts. When approved, access will be granted via a secure website.

The goal is to help researchers examine and combine data from different studies to further their own research, understand drug mechanisms of action in different patient populations and help optimize drug development.

The pharma said its move was designed to help the research community develop "a broader system" where scientists could access data from clinical trials conducted "by different sponsors."

Data requests will be evaluated for their relevancy to medical science or patient care, clear scientific rationale, appropriate and scientifically valid analytical approach and publication plan, according to Stubbee. Researchers will be required to sign a data-sharing agreement that will include requirements to use the data only for the stated purpose and not to attempt to identify individuals.

In broad terms, the data-sharing agreement will include language giving GSK a royalty-free, worldwide, nonexclusive license to use intellectual property created by the research, Stubbee said.

"It's hard to speculate how that principle will apply to hypothetical situations, as the facts of a given situation may differ," she told BioWorld Today.

GSK did not specifically seek input from biotech partners, patient advocacy groups or third-party researchers or discuss the proposed measures with other pharmas, according to Stubbee. Instead, the initiative "is a result of listening to others and being part of discussions generally on the potential benefits and concerns – for example, issues related to patient privacy and confidentiality," she said.

GSK Puts Sanctions Behind It, Moves On

The transparency initiative is all the more remarkable coming from GSK, which recently ran afoul of the U.S. Department of Justice (DOJ) for practices conducted in secret. In November 2011, the company reached an agreement in principle with the DOJ resolving investigations into its U.S. sales and marketing practices, the possible inappropriate use of the nominal price exception under the Medicaid rebate program and the development and marketing of diabetes drug Avandia (rosiglitazone). The settlement, which included civil and criminal liabilities, was finalized in July, with GSK agreeing to pay a whopping $3 billion.

At the time the DOJ agreement was signed, a chastened Witty said GSK had already improved its U.S. compliance programs with a larger staff and strengthened employee training programs. The company also initiated an incentive compensation system for sales representatives who work directly with health care professionals based on quality of service rather than individual sales targets.

In a statement about the transparency initiatives, Witty acknowledged "the complexity of the science and the scale of the challenge" prevent any single pharma or biotech from solving serious global health problems. By focusing on collaboration and "being more open with our clinical trial data, we also hope to help further scientific understanding."

Although skeptics weighed in on social media, Stubbee said the company has no hidden agenda.

"We hope that this extra step in providing access to our data will demonstrate our commitment to transparency, enable the research community to learn from what we know with the aim of improving patient care and offer patients and health care professionals more confidence in the data that support our medicines," she said.

Christopher P. Austin, director of the National Center for Advancing Translational Sciences (NCATS), said companies and researchers must "work as a team and openly share information" to improve the drug development pipeline.

"Crowdsourcing data and knowledge to the broader research community enables the best scientific ideas to emerge," Austin added, noting NCATS is leading similar collaborative initiatives with various stakeholders to increase the efficiency of translational research and to accelerate the pace at which new treatments are delivered to patients. (See BioWorld Insight, Sept. 4, 2012, and BioWorld Today, Dec. 28, 2011, May 4, 2012, and June 21, 2012.)

In making its TB compound library freely available, GSK scientists screened the company's entire library of more than 2 million compounds for any that may inhibit TB bacteria, identifying some 200 promising hits that could serve as starting points to develop TB drugs.

The process represents the first public disclosure by a pharma of proprietary compounds with potential activity against TB.

Stubbee said the findings were submitted to a peer-reviewed journal. Coinciding with the paper's publication, the data will go live on the European Bioinformatics Institute portal.

Similarly, in 2009 GSK made 13,500 experimental malaria compounds, their chemical structures and associated assay data freely available to the public. The company and Alnylam Pharmaceuticals Inc. launched a so-called "patent pool" providing researchers free access to intellectual property, discovered compounds and technologies in an effort to discover new drugs for neglected diseases. GSK pledged access to 800 granted or pending patents, while Cambridge, Mass.-based Alnylam contributed 1 ,500 RNA interference patents to the effort, overseen by the nonprofit BIO Ventures for Global Health. (See BioWorld Today, Jan. 21, 2010.)

GSK also said Thursday it will double the funding for its Open Lab with an additional £5 million (US$8 million) investment. Since the Open Lab was established in 2010 in conjunction with the neglected diseases initiative, 16 research projects have been launched in indications such as TB, malaria, Chagas disease, leishmaniasis and sleeping sickness.