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Patient centricity requires clinical trials to be based on life outside the lab, writes Mads Holme.
Patient centricity requires clinical trials to be based on life outside the lab, writes Mikkel Brok-Kristensen.
Have you ever given up on a jar of preserves because you couldn’t get the lid off? Imagine if that was your medication for rheumatoid arthritis (RA), prescribed to you so you could better use your hands. Such a combination might seem absurd, but it is what often happens when the clinical trial is the only assessment of whether or not a product should go to market.
Despite industry-wide ambitions of patient centricity, new innovation in pharma is still almost exclusively evaluated on safety and medical efficacy in the clinical trial setting. A number of the rheumatoid arthritis patients we met in a study struggled with administering the biologic prescribed to them. One, let us call her Rachel, couldn’t self-inject because her hands were in such poor shape. In the clinical trial setting, she would have had help - nurses and doctors on staff, making sure she is comfortable. In real life, she’s on her own.
When we test a drug’s potential to benefit patients like Rachel, we base our goals on a strict numeric ideal – one that is derived from a healthy body. And a successful treatment is one that can bring any given patient as close to that ideal as possible with as few risks as possible. This is then done in a setting where patients are supported by a bevy of medical practitioners in way never found in the real world. In some cases, the product is even tested in a room without a context-usually a hospital room or a doctor’s office-with no reminder or call of real life activities. It’s much easier to create a sophisticated medical solution for a patient lying on a hospital bed watching TV.
If you really want patient-centricity to permeate all areas of drug development, we suggest you take a look at the invisible costs involved, and the needs beyond efficacy often ignored. In the case of RA, what is the actual cost of treatment? Cost comes in the months patients spend waiting to see if their biologic will be effective. Or in becoming dependent on others for treatment administration - requiring either a second-party administrator in home, or a half-day hospital infusion. Cost is in the form of the hours physicians spend researching on the best treatment, trying to minimize their risk, when prescribing new drugs to patients.
And what are the needs to which we often are blinded? For some patients and doctors, it is easy to trade away the most efficacious treatment with fluctuations for a ‘lesser’ but predictable one. For others, discretion of treatment trumps the ideal medication - often leading to decreased compliance. Prescribers and patients both seek predictability in this disease riddled with uncertainty - trying to find not the best treatment for the average patient, but the best treatment for each individual. They seek treatments that target the most effected limbs, not necessarily all.
This is the case for Rachel, who’s had RA for 10 years, and is still searching for the right biologic. Because surprisingly, chronic pain is not her biggest concern. Rather she needs a treatment that that will allow her to be certain that she can go to her son’s graduation, she wants a drug that will give her sufficiently dexterity to continue working as jewelry designer.
Patient-centric thinking will help to address these costs and deliver on these needs, preventing the common mistake of developing drugs that have no practical application for patients in the real world. And we need more forward thinking companies to develop drugs that succeed-not just with the two metrics of medical efficacy and safety-but with real individuals in the realities of their everyday lives.
As industry experts know, the clinical trial is much more than the test itself, and poses an opportunity to meaningfully innovate both at the molecular but also design level. In the example of RA, where uncertainty plagues both patients and prescribers, an entire market has emerged in developing biomarkers; but others are simply thinking about fast-onset in new ways. Rather than seeing that the drug works, and that it works quickly, they are also looking for signs of transparency: an early indicator that the drug will work in the future. In so doing, the burden of uncertainty for prescribers is lessened, as is the opportunity cost of patients in trying a new treatment regimen.
Adding in further parameters such as transparency is no easy task and will increase the likelihood of failure in clinical trials. But it will also decrease the risks of only developing a me-to drug and with it increase the chances of commercial success. What forward-thinking companies have done is take steps towards redefining their parameters for drug development. For too many companies, the FDA is the final determinant of success. But, patient-centric thinking means success is-and should always be-in the eyes of the patients and physicians.
Mikkel Brok-Kristensen is a partner at ReD Associates, a strategy consulting firm based in the human sciences.