Last month, GlaxoSmithKline (GSK), the global pharmaceutical company based in the U.K., made a key move into an industry-leading position to reform the clinical trial process that tests new drugs on human subjects. The company in October opened its files making available to outside researchers detailed data from its own clinical trials, just after the industry announced it would write a common data standard to cut the costs of these expensive studies.
Clinical trials consume some 40 percent of the pharma industry's R&D spending, which is forcing the industry to find better ways of testing drugs that save time and money without compromising patient safety. And fixing the clinical trial process is more than just an pharma industry issue, but also one of economic and health care policy.
The President's Council of Advisors on Science and Technology (PCAST) issued a report in September calling for a doubling of the output of medicines in the next 10 to 15 years, and identified the inefficient clinical trial process a barrier to meeting that goal. PCAST member Ed Penhoet, a director of the venture capital firm Alta Partners, told a Brookings Institution conference on 19 November that the commission plans to brief the president next week on the report.
Testing drugs with humans calls for large, complex, and expensive research projects that come with a host of scientific, regulatory, and ethical requirements. The length of time needed for clinical trials varies depending on the uniqueness of the drug, complexity of the disease, and time needed for treatment. But is not unusual for all phases of clinical trials of a new drug to last 10 years or more.
Trials typically follow three phases, with the early phases testing new drugs for safety, moving into later phases that test with large samples for efficacy against placebos or existing drugs. Strict testing protocols are needed to ensure the effects on patients happen because of the drugs being tested and not extraneous factors.
The conduct of clinical trials, as the PCAST report notes, resemble more of a medieval guild than a 21st-century scientific endeavor. Most clinical trials today are designed as one-off exercises, recruiting scientific teams and sites from scratch. Institutional review board protocols -- rules governing the conduct of clinical trials for dealing with human subjects -- vary from one institution and trial to another, and by themselves can take months to write.
The trials are organized to deliver findings about the test drug for regulatory approvals and management decisions, with little concern for longer term utility. As a result, the information technology used in clinical trials is rarely standardized, making it difficult to integrate data across trials.
The biopharmaceutical industry, with its affiliates in government and academia, in recent years started various initiatives to cut the time and cost of clinical trials, but a new industry project called TransCelerate BioPharma, appears ready to take on the systemic problems. TransCelerate, announced in late September, is a not-for-profit consortium of 10 pharmaceutical companies including GSK, to speed the development of new drugs, focusing particularly on the clinical trial process.
TransCelerate aims to create common methods for clinical trials, providing a standard organizational and data framework and thus making these studies less of a one-off exercise. The group plans to deliver common tools for assessing patient risk, standard criteria and processes to qualify medical centers for trials, common data reporting standards (working with an industry standards group), a uniform model for supplying comparator drugs for head-to-head trials, and a shared portal for researchers and staff.
GSK, in an 11 October announcement -- about two weeks after TransCelerate's unveiling -- went further, making it easier to access detailed data from its clinical trials. The data to be made available will cover all of GSK's trials, with patient identification removed, including those where the results did not lead to a new drug.
Researchers seeking these data will first need to register and apply for access to the database, with requests reviewed by an independent panel. GSK already publishes summary results of 4,500 clinical trials that the company says attracts some 10,000 visitors a month.
GSK's press release announcing this initiative calls it "a step towards the ultimate aim of the clinical research community developing a broader system where researchers will be able to access data from clinical trials conducted by different sponsors." When considered in the context of TransCelerate, GSK seems to be pushing its system for clinical trial data as the de facto standard for the pharmaceutical industry.
Familiarity breeds success
TransCelerate plans to work with the Clinical Data Interchange Standards Consortium, a cross-industry group devising data standards for clinical trials, of which GSK is a member. Since GSK is making its patient-level clinical trial data available well before standards are written, however, researchers will likely get to know GSK's system, using their analytical tools with GSK's data format, far in advance of any industry-blessed standard.
With clinical researchers needing their systems to work with GSK's format, one could reasonably expect software companies serving the industry to respond to that need. Software developers are usually the companies that translate standards into real-world implementations, and they frequently take part when industry standards are developed.
As a result, end-users -- the clinical trial researchers -- and software companies will likely be familiar with GSK's data format, once standards development begins. While a new data standard will probably reflect industry needs overall and not just one company, GSK's system could well be the starting point of discussions leading to the standard.
Information-intensive industries, such as banks and airlines, learned decades ago that control of information systems provides an enormous competitive advantage in those industries. American Airlines, for example, used its SABRE system to achieve an early advantage with travel agencies. GSK may be trying something similar with clinical trials.