Real world evidence has value throughout the healthcare system in helping drug developers, payers and patients make better, more informed, decisions. For pharmaceutical companies to extract the most value, however, they must put the patient first and piggyback on that experience.
Speaking at the lunch plenary at Biotech Showcase™ 2017, Jamie Heywood, co-founder and chairman of PatientsLikeMe, asks the critical question, “Who’s the customer?” That answer determines how the information is used.
From the patients’ perspectives, Heywood says, “there’s immense need for insight to live well with a disease, to make choices in their lives, environments and medical care that make their lives better.” Real world evidence measures those choices, including complex medical interventions and what happens to others who make those same decisions.
When the FDA is the customer, “using real world evidence can inform decisions around, for instance, adverse event monitoring and Phase IV confirmatory trials for a drug under accelerated approval. Supplemental indications also can be approved, and companies can generate data with collective confidence,” Wade Ackerman, partner, Covington & Burling LLP says.
Ackerman, who was instrumental in passage of the 21st Century Cures Act, says that Act has a provision for real-world evidence that is aligned with PDUFA 6. “The law pushes, but not too fast,” he says. When PDUFA reauthorization hearings take place this summer, the FDA is expected to set up public meetings regarding guidances for data collection, standardization and analysis.
For drug developers, evaluating real-world evidence is instrumental in transforming a hurdle into an opportunity. “What’s revolutionary is the use of real-world evidence to get to market faster and find niche markets through interactions with patients,” says Philippe Lopes-Fernandes, senior VP, global head of business development and alliance management at EMD Serono. “Having the right data is critical to assess the potential of the product and ensure the right price.” Interaction with patients at earlier and earlier stages of drug development, therefore, helps companies design studies that produce that data.
One pharma option is to use real-world evidence in early Phase II expanded access programs. This has particular potential for areas of unmet medical need. While the FDA remains cautious, Ackerman reports great excitement behind closed doors for sponsors to move this forward. Another option is to identify opportunities in personalized medicine that increase effectiveness without marking any therapy overly personalized.
Payers’ acceptance of real-world data is a relatively recent phenomenon. Ted Haack, owner and managing partner of Haack & Associates, recalls discussions with Germany’s healthcare system several years ago. Despite have “tons of information” for Lipitor, the data wasn’t accepted. A few years later in 2012, he had similar discussion with Germany about Lyrica, based on post-launch data. This time, he was successful.
Patient privacy and social media
Balancing patient privacy with data collection can, at times, be challenging. PatientsLikeMe views privacy as a “complicated legal and ethical component. There is a social contract,” Heywood says. He maintains that social contract can be changed in ways that benefit individuals and the healthcare system. By sharing data about their conditions, patients can regain some of the power their diseases took away by helping someone else. “There’s also a social aspect. Most patients want to interact with people who have the same challenges they have,” Heywood says.
PatientsLikeMe sites go beyond conversations. “We’re building a framework for research that lets scientists get the depth of the meaning behind the conversation, to determine what’s meaningful to individuals. This is different from generating ‘clinically significant’ data.”
Notions of patient privacy vary geographically, too, which affects opt-in to any patient database. “The French have a higher concept of privacy than Americans, and Germans have a higher concept than the French, Heywood acknowledges. “Nonetheless, enough people will participate to get meaningful data.”
Payers care about real-world data
“Payers are interested in defining the disease and discussing its progress,” Steve Wooding, head of global commercial strategy organization for the Janssen Pharmaceutical Companies of Johnson & Johnson says. It remains a gray area, though. They are interested more in short-term solutions based on real-world evidence, like the effectiveness of an antiviral therapy, than on the long-term results of medications for chronic conditions. That’s because patients in the US generally change healthcare plans every three to five years. Therefore, payers have little incentive to improve care or cut costs for clients who probably won’t be with them in 20 years’ time. To use real-world evidence effectively with these payers, then, drug developers need to understand their interests.
All of these steps, eventually, may lead to Heywood’s vision of a deeply digitized patient. In that vision, patient information is digitized to the extent that individual patients can request a bid —a request for proposal—for a company to improve his or her health in a given direction, and a healthcare system that reimburses that company for actually improving the patient’s health. That may sound like science fiction but, 10 years ago, so did the iPhone, Heywood points out.
And, like the iPhone, discussing real-world evidence is fashionable. To make that evidence useful, Wooding says, “start with a question. If it doesn’t answer a question about your drug and where it’s going, it’s just noise.”