Drugmakers are trying to win drug approvals by parsing vast data sets of electronic medical records, shifting away from lengthy, and costly, clinical trials in patients.
Pfizer Inc., PFE 0.67% Johnson & Johnson JNJ 0.49% and Amgen Inc. AMGN 0.50% are among the drugmakers that have submitted the data-mining analyses to the U.S. Food and Drug Administration in seeking approval to sell new medicines or for new uses for older ones. The FDA has approved new uses for breast cancer, bladder cancer and leukemia drugs in part based on the data.
For the companies, the use of real-world data can cut costs and shorten drug-development times. Instead of finding trial subjects, companies simply mine hospital and doctor files for cases where patients already took a drug in routine medical care, looking for changes in blood pressure, tumor size and other readings to see if the medicine is helping or causing a side effect.
This real-world evidence is sometimes used in lieu of a clinical trial’s control arm, to compare outcomes for past patients who got a standard treatment against people who are taking a new drug in a clinical trial. Such analyses can take months, compared with years for prospective clinical trials.
Some doctors worry about forsaking clinical trials, which are carefully designed and conducted in patients to get a sound read on a drug’s safety and efficacy. Health records often have errors, the skeptics say, and even an analysis of records that are error-free doesn’t have the same scientific rigor as a clinical trial in sizing up a drug.
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Data from a clinical trial “are invaluable because it gives you a sense of, ‘In perfect circumstances, did the intervention work?’” said Dr. Joseph Ross, professor of medicine and public health at Yale School of Medicine.
Supporters of real-world analyses say it is unlikely they would replace standard clinical trials needed for the initial approval of a new drug. Instead, data-mining could augment such trials or be done instead of trials for secondary approvals of a new use, when patients are already taking the drug for an unapproved use and can be tracked.
Clinical trials have been a bedrock of medical testing for decades. The most reliable ones randomly assign some subjects to take an experimental drug, while other subjects get a placebo or older drug.
But for rare diseases especially, it can take awhile to even enroll enough patients in studies. And their cost can limit the number of trials that companies can fund, drugmakers say.
A 2016 law required the FDA to explore greater use of real-world data, and the agency is developing standards to assess the reliability of different data sources and which kinds of decisions the data support.
“Real-world evidence should not be a means toward dropping standards, but rather a mechanism to have more efficiency in evidence generation while maintaining standards,” said FDA Principal Deputy Commissioner Amy Abernethy, a former executive at health-data firm Flatiron Health.
A market has emerged in recent years for digital drug-use information. Iqvia Inc., which tracks prescription and health data, has about a dozen projects under way, said Nancy Dreyer, the company’s chief scientific officer of real-world evidence.
Swiss drugmaker Roche Holding AG RHHBY 1.27% has spent about $5 billion in recent years acquiring two health-data companies: Flatiron provides electronic health-record software to cancer clinics and analyzes data from those records for drug companies, while Foundation Medicine tests patients’ tumor samples for genetic mutations and aggregates the information for drug research.
Roche included a Flatiron analysis of patients whose cancer has a certain genetic defect in the company’s application to market a new drug targeting that defect, Rozlytrek, which FDA approved in August.
Amgen used real-world data from leukemia patients to serve as a comparison for a small clinical trial in which all patients in the trial received its leukemia drug Blincyto. The FDA last year used the analysis in its decision to approve a new use for Blincyto treating patients who are in remission but have some cancerous cells that put them at risk for relapse.
Amgen would have had to enroll at least 50% more patients in the clinical trial to have a standard control arm, said Elliott Levy, Amgen’s senior vice president of global development.
Such trials “typically involve the potential to be randomized to existing standard of care or even no therapy, when what patients want is the opportunity to be treated with a promising experimental” drug, he said.
More
- Fast-Track Drug Approval, Designed for Emergencies, Is Now Routine (June 5, 2019)
- FDA to Ease Some Cancer Patients’ Access to Experimental Drugs (June 3, 2019)
- Startups Aim to Fix ‘Broken’ Clinical Trials System (Oct. 12, 2018)
- Are Big Clinical Trials Relevant? Researchers Disagree (May 29, 2018)
- Clinical Trials Need More Subjects (April 13, 2016)
J&J used Flatiron and Foundation data to find that bladder-cancer patients with a certain genetic trait didn’t benefit significantly from certain immune-boosting drugs. This analysis augmented a separate clinical trial in which patients with the same genetic trait did benefit from J&J’s experimental drug Balversa, said Craig Tendler, vice president of oncology clinical development and global medical affairs at J&J.
J&J included the Flatiron/Foundation results in its application for Balversa’s approval, which the FDA granted in April 2019.
Pfizer’s breast-cancer drug Ibrance was originally approved in 2015 for women based on a clinical trial. After some doctors began prescribing the drug to men off label, Pfizer contracted with Flatiron to mine electronic health records to see how men who got the drug fared compared with those who hadn’t.
Separately, Iqvia reviewed prescription claims and found male breast-cancer patients stayed on Ibrance longer than those taking other drugs.
Chris Boshoff, Pfizer’s chief development officer for oncology, said the analyses took less than a year and cost a fraction of a clinical trial, which he estimated would have taken four or five years and cost tens of millions of dollars.
But an FDA reviewer of Pfizer’s application said the Flatiron data provided limited evidence of Ibrance’s effectiveness in men because of small sample sizes, according to an FDA document obtained by The Wall Street Journal via a public-records request. And the two patient groups weren’t well balanced on factors like age, the FDA reviewer said.
FDA reviewers also said it was difficult to conclude from the Iqvia analysis whether an increase in prescription duration translated into improved survival or a delay in disease progression.
Yet the FDA approved the use of Ibrance in men, noting that the prior clinical trials in women gave the agency the confidence the drug could help men, supported by the real-world evidence and Pfizer’s safety-tracking databases.
Write to Peter Loftus at peter.loftus@wsj.com
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