With such a focus these days on buzzwords such as "patient-centric," "patient-centered," and "patient-centricity," some people are predicting “patient engagement” to be the next big movement in our industry, much as blockbuster drugs were a decade or more ago. But while improving patient engagement sounds fairly straightforward for those who directly interact with patients (e.g., doctors, nurses, nurse practitioners, and physicians assistants), for the biopharmaceutical industry, successfully executing this concept has been — and continues to be — much more challenging.
If achieving patient-centricity is biopharma’s Holy Grail, I thought I would seek some insight on this topic from the leadership team of a business built on serving patients. Founded in 2004, PatientsLikeMe, a forprofit company built on the principle of patient engagement, has grown from a single online community for ALS patients to a business covering 2,300 health conditions gathering real-time data from 300,000 members.
In the company’s bustling, brownstone headquarters in Cambridge, MA, I was joined by Jamie Heywood, cofounder and chairman; Ben Heywood, cofounder and president; and Martin Coulter, CEO. Together, they shared with me their perspectives on why biopharma companies struggle when it comes to engaging with patients, as well as what companies can do to become more patient-centered.
WHY DOES PHARMA STRUGGLE WHEN SEEKING TO ENGAGE PATIENTS?
While being highly regulated or culturally conservative are most likely contributing factors to biopharma’s patient-engagement struggles, to understand being patient-centered as a strategic solution, the PatientsLikeMe execs suggest you go to the root of the problem — the deeply infused cultural rules of clinical research.
“As you're talking to people about how to do clinical research,” Jamie Heywood analogizes, “it is as if Moses came down from Mount Sinai with a third tablet that said, 'Thou shalt only do a double-blind placebo-controlled trial and believe only the evidence from that.'” This mentality creates a number of problems. “The purpose of blinding a study is to eliminate biases,” he shares. “Because blinding is the only approach many researchers have ever known, and, therefore, the only evidence which they will ever believe, they are less open to considering new ways of conducting clinical research.” For example, PatientsLikeMe has developed an ALS predictive model that Jamie says is so accurate it is conceivable to conduct a clinical trial without a placebo. “It’s now been validated in multiple publications,” he states. “If you can determine whether a drug makes a difference against prediction instead of placebo, this is really neat because you can do what might be an 80- or 100-person trial with just 25 or 30 people. That's dramatic cost savings and you can do more dose ranges for the same money.” Jamie believes that being able to conduct a clinical trial against prediction versus placebo could not only be cheaper but also better and faster to market. Though the rules of clinical drug trials are constantly being revised and rewritten, testing against prediction is not considered a viable option. Patients desperate for solutions are willing to try just about anything. As such, they want researchers open to trying new solutions, not ones blinded by their own biases.
Although the PatientsLikeMe team admits the present way in which clinical trials are conducted is adequate for the purpose of getting a drug approved, they contend it is not at all patient-centered. “Medicine and clinical research are very paternalistic fields that have largely regarded patients as subjects which perform best when they are adherent, compliant, unquestioning followers of the rules,” says Jamie. This attitude is not conducive to creating a relationship between patient and researcher, and subsequently, patient and biopharma. To achieve patient-centricity requires creating a relationship where the patient is an equal partner similar to your other business partnerships. “In a partnership with a biotech or a clinical research company, you're not sitting there at the end, saying, ‘Hey, you patient, help me out here so I can make this medicine that makes your disease better that you may or may not be able to buy from me in the future,’” says Jamie. “That's not a partnership. That's a subject relationship.”
Another reason why biopharma has struggled with patient engagement is that the industry has relied on one-way communication that often includes only mass media and the healthcare provider. “As that world changes and the physician channel is going away, there’s this realization of needing to develop a different model for establishing a relationship with patients,” says Coulter. “But there's a lack of infrastructure, experience, and understanding as to how to do this effectively.” Ben Heywood adds, “The first instinct of the patient-centered tactic is to invite a patient to ‘the table’ — to a conference or to an ad board.” While this is a big step, the PatientsLikeMe team cautions to avoid the temptation of racing to create a solution to your patient-engagement problem before thoroughly considering the various challenges and how best to overcome them.
DON’T LEAP BEFORE YOU LOOK
One of the most basic challenges when engaging with patients is, “How does a human being know what to do in an information age?” asks Jamie. “The question can be as simple as for the headache I have right now, 'What's the best way to treat it?' Even though these drugs have been used by hundreds of billions of people, we have no idea what the answer to that question is.” According to Jamie, there is a limit to the number of variables a human, as a heuristic engine, can process to reach an effective conclusion. In other words, before you invite patients to the table, understand the questions you need to ask them so you are collecting the right data. For example, consider the proxy measures of healthcare, which look at readmission rates or hospital acquired infections. “The core measures are falls or bedsores,” says Coulter. “These don’t really get to the nature of the underlying condition.” If you want to better understand the outcome, start by measuring the experience of the process that led the patient to the outcome. Regarding clinical trials, Ben adds, “Patient reported outcome [PRO] measures are typically designed in the context of doing research and understanding an end point, as opposed to having a patient-centered view of an enabling self-measurement and self-disease management.” If you want to develop better end points, you need to meet the challenge of understanding what matters to the patient during their disease journey.
Another challenge to consider is that consumers are increasingly looking for, and have been given, control in their lives. “From how we order a taxi to how we reserve our next hotel stay, consumers are increasingly looking for data, referrals, and input from the crowd,” says Coulter. But during or after the clinical trial, participants aren’t usually given much access to the data. According to Jamie, “In the highly regimented and rigorous field of clinical research, your currency is building a population of patients, separating them into two different groups, conducting research, and collecting data from which you can then publish articles.” As data and publication are what clinical researchers perceive as valuable, Jamie believes this encourages researchers to only want to share conclusions and not the data from which they were derived. The challenge is not only creating a willingness to exchange data but also making that data digestible for patients. “This requires organizations to be able to figure out not only what's important to patients, but also how to present the findings in terms that resonate with them,” says Coulter. “That's a skill the life sciences industry needs to develop.”
An additional challenge is the traditional lengthy cycle times of research projects. “Patient-engagement research projects with six-month answer cycles are often overdesigned to produce precise answers,” says Jamie. He suggests if you want to start operating your business where you’re making decisions in days and weeks instead of bi-annually, retrain your operational directors and vice presidents to seek outside resources which can help operationalize a real-time engagement model and stop overdesigning questions. While adopting a patient-centered approach may make sense, expect resistance at various levels. For example, implementing a patient-engagement approach across a company’s therapeutic portfolio (e.g., oncology) might meet resistance from individuals responsible for a single asset (i.e., experimental treatment) within the therapeutic category, especially if input adds new information that they fear could add risk to the asset’s success. A further challenge is operating in the clinical research world as it exists while trying to think about, and invest in, how you want it to look in the future. “Essentially, there is no funding, no model, and no mechanism for continually improving the measurement of disease via patient engagement,” Jamie says. “At some point in the future, devices around us will monitor enough of our lives that the idea of surveys will be gone. While Jawbone [wrist band fitness activity tracker] as a tool for measuring mobility in multiple sclerosis patients as a changed measurement methodology in a trial is not validated and ready today, at some point it will be.” Jamie refers to the time period between now and eventual validation of personal activity tracking devices for use in clinical trials as an inflection point. Though he believes these devices will save therapeutic development companies hundreds of millions of dollars in the future, the benefits and savings will go to only those leaders willing to invest in understanding which of these technologies, and in which contexts, matter. “Leaders need to be willing to invest in these and other inflection points.” Ben says transitioning a new concept, such as patient engagement, from the innovation group to operations is challenging. “Demonstrating the value and getting operational buy-in is possible as long as the budget to pay for it is coming from innovation,” he states. “What we have seen is the traditional approach of easing it into the operating budget with the initiative being partially funded by both groups.” While this sometimes works during the transition, it often fails when operations has full financial responsibility. He suggests if adopting a patient-centered strategy, be sure to consider how to fund it, including creating transition budgets and teams to help the initiative move successfully from an innovative experiment to an operational best practice.
TRANSFORMING TO PATIENT-CENTERED REQUIRES TRANSFORMATIVE LEADERSHIP
Jamie Heywood believes truly transformative businesses were not necessarily built on the idea of being a business. “They were built on a mission that became a business,” he attests. “Google did not have a revenue model when it was invented and began to understand how to measure the Web. There were other search engines operating on more traditional heuristics about how to understand things. The parallel to adopting a patient-centered approach at your company is looking at the actual patient experience as essentially the deterministic value of healthcare delivery or drug development.” While he believes the Framingham Heart Study and the Nurse’s Health Study to be excellent examples of this concept in action, it’s not broadly accepted. “We've seen, depressingly, examples where we’ve delivered transformative levels of value to one franchise team, and that team has tried to bring that same value to other parts of the organization. But the organization goes through the same resistance curve because it just replicates the problem in a different silo. Convincing the pharmaceutical Business to think about developing a relationship with the customer/patient requires transformative leadership. “Even with a biopharma company that has 25,000 to 50,000 employees, there are really only about 100 people in those enterprises capable of changing the way a decision is made.” To assist you in becoming patient-centered, he advises bringing in change management experts to help.
Transformative leaders need to approach partnering with patients as, “I'm going to solve some problems for you the patient that may or may not directly address our company’s needs right now,” says Jamie. “And, I'm going to make sure that I enable an infrastructure so that you the patient are better at solving your own problems through this partnership.” He analogizes it as being the opposite approach to the bygone days of coal mining. “When a coal company came into a town, tore off the top of a mountain, and left a bunch of chemicals in the streams, even though they'd given everyone a job for a little while, they've left the town worse off.” Instead, strive to create a system that leaves the patients better off. “I was at a discussion yesterday with a major pharma about a research project,” he shares as an example of what patient-centered is not. “They were really excited about how they did this and that, and said, ‘We're being so patient-centric.’ I said, ‘Really? What did the patients get out of it?’ The answer was, ‘Oh, they're excited about participating in research, so we make them feel better, because they're participating in research.’” If you want to be patient-centered, ask yourself, "What's in it for the patient? How will you leave them better off? What can you give them to help them organize on their own? Does that data need to be competitive or can it be collaborative? How can you make it available? These are things conspicuously absent in many industry approaches to developing a relationship with patients."
The Disappointment Of Clinical Outsourcing
Jamie Heywood, the cofounder and chairman of PatientsLikeMe, is disappointed with how clinical trials are outsourced today. He says that although CROs have done a great job optimizing the processes surrounding trials, the sponsors have done little more than tweak some of the parameters that have always been in place. “We've basically committed to a trial model that was designed 20 years ago, outsourced everything according to that model’s specifications, and then built an entire optimization system to drive down costs. But during all of this, we’ve stopped thinking about what a trial is for.”
Heywood believes patients and diseases need “continuous measurement improvement.” “Pharma needs to enable continual collection of increasingly meaningful information about patient experiences. Doing so will help improve measurement and drive better decision making,” he says. He also believes pharma should invest accordingly. “If you're not spending significantly to optimize how you measure a disease, then don’t complain about the billion-dollar price tag of drug development,” he comments. Instead, broadly engage as partners with the patient community so you can collaboratively improve measures, target patients more effectively, and ultimately reduce clinical trial costs.
Patient-Centered Requires Big-Picture Thinking
Idiopathic pulmonary fibrosis, IPF, is about to receive a big cash infusion. “There are now six companies specializing in IPF that are either in or going toward a phase 3 program,” says PatientsLikeMe chairman and cofounder Jamie Heywood. A rough number of $200 million each equates to $1.2 billion in spending. One of the problems Heywood envisions is that these six companies are spending more money than they should. Unlike diseases such as ALS, which has an extremely strong clinical trial network, IPF does not. “It doesn't have a network of clinics,” he says. “It doesn't have an outcome measure that is accepted or used by the FDA to approve a drug. The current measure, forced vital capacity, doesn't really match the decline of the disease — and the vast majority of IPF patients do not participate in clinical research.” Heywood believes these are all fixable problems. “You can build an open clinical research network that is patient-centered and recruit most of the people with the disease. You can educate patients and develop the measures, deploy them clinically, and validate.” Instead of operating in isolation, these companies should do some big-picture thinking about how to best help the patient, and he believes all will benefit. “Imagine if the companies pooled $50 million toward solving the various problems around conducting IPF research,” he ponders. “Suddenly, you could take a $1.2-billion cost and halve it.”
Heywood feels that becoming truly patientcentered requires biopharma to take on bigpicture collaborative thinking. “This type of approach would result in getting faster and better signal detection, a better regulatory response, and a far better patient experience,” he attests. “Further, because you're integrating trial level measurement into the care process, you can eliminate most phase 4s; risk management is automatic, and as an added benefit you get real-world comparative effectiveness.”