Hunt for real-world data

June 29, 2018 0 By CH Unnikrishnan

Drug makers are in race to gather data outside clinical trials as end-users demand evidence for efficacy and value of drugs in day to day clinical practice

In February, the Swiss drug giant Roche acquired Flatiron Health, a start-up analyzing real-time oncology data to help cancer patients and doctors, in a $1.9 billion deal.

Roche is one of the drug makers racing to strike deals with big data analytics companies hunting for information on how drugs work in the real world.
Traditionally, medicines are evaluated through randomised clinical trials (RCT). Many drugs shine in clinical trials, but under-deliver in day-to-day clinical practice.

This is where gathering real-world evidence (RWE) is gaining importance. Companies are gathering and generating data outside RCT to produce evidence for efficacy and value of drugs in the real world.

The rise of electronic medical records, insurance claims databases, fitness wearables and even social media aids this trend by offering a trove of new information.

A Deloitte poll of 2017 found more than half of the life science companies to be engaged in the process of developing real world data capabilities.
Major drug companies now have departments dedicated for use of real-world data across multiple diseases. Quite a few companies have already completed scientific studies using the data into areas addressed by their drugs. AstraZeneca and Sanofi, for instance, are conducting research on diabetes. Joint research in stroke prevention by Pfizer and Bristol-Myers Squibb, and a bowel disease project by Takeda Pharmaceuticals are other examples.

The trend is fast catching up in the recent years. Half of the world’s 1,800 clinical studies involving real-world or real-life data since 2006 have been started in the last three years. Last year, the number of such studies hit an all time high of 300, mostly from therapeutic areas of cancer, heart disease and respiratory disorders, according to the clinicaltrials.gov website run by the U.S. National Institutes of Health.

RWE will create evidence of how a typical patient responds to a drug.
”Efficacy studies show us in very basic terms how effective the treatment is from a biomedical perspective. But then, when the treatment is out there in the real world, we know it is affected by many things,’’ said John Weinman, professor of psychology at King’s College, London.

Hence, there is a disconnect between the data companies need to generate to win approval and the data they need to persuade people to pay and use their drugs. Responding to the concerns of the drug prices, governments and insurance payers are tying payments to outcomes. Novartis, for example, secured an outcome based model for Kymriah, its CAR T-cell therapy

In such a situation, real world efficacy data will become a requirement for full payment for a drug. If the drug fails to deliver as expected, the manufacturer cannot insist on the sticker price.

Considering the rising demand from providers and payers, real-world evidence is likely to become a vital aspect for pre- and post-drug approval processes. U.S. Food and Drug Administration, the regulator of the world’s largest drug market, is currently evaluating the expanded use of RWE under the 21st Century Cures Act.

“As the breadth and reliability of RWE increases, so do opportunities for FDA to also make use of this information,” US FDA Commissioner Scott Gottlieb said in a speech in September.

Experts in the field believe more widespread use of RWE could cut drug development costs and help doctors make better medical choices.
Some academics even predict that real world studies could replace phase III trials in which typical patients are randomised to receive the experimental therapy.

In the meantime, growth of RWE raises also raises questions about data access and patient privacy.

In 2016, Britain’s National Health Service (NHS) had to scrap a highly ambitious project to pool anonymised NHS patient data for both academic and commercial use, as both patients and doctors protested. Drug firms too admit that ensuring privacy and scientific rigour is a challenge.

Real world efficacy data could soon become a requirement for full payment for a drug. If the drug fails to deliver as expected, the manufacturer cannot insist on the sticker price