Patient Centricity: Joining the Qualitative with the Quantitative
By Kathleen Hoffman, PhD, MSPH
Reams of market research and data can provide insight, but so can one patient in the room, telling you what really matters to them.1~Paul Tunnah, PharmaPhorum founder
The FDA’s drug approval process requires developers to submit quantitative data demonstrating that the treatment is safe and effective after testing in a representative target population. But if you’re accustomed to using quantitative studies to attain approval, you might be feeling jaded about patient centricity guidances. As Tunna’s article says, “…everyone seems keener to shout about big data, digital health, artificial intelligence and other exciting technologies” these days.1 Aren’t the FDA Patient Focused Drug Development guidances more of a legislated nicety that’s been added to the burden of scientific proof?
Perhaps it is time to revisit the value of adding the patient voice to clinical science R&D. We need examples where patient input yielded data that advanced drug development and increased the likelihood of patient adherence. There are several studies that provide encouraging results.
Increasing Clinical Trial Participation
Researchers still struggle with meeting numbers for clinical trial subject enrollment. A 2018 review of the literature titled Factors associated with clinical trials that fail and opportunities for improving the likelihood of success published by Contemporary Clinical Trials Communications quoted a study finding that only 25% of cancer trials met their enrollment requirements, and then 18% of the actual trials closed with less than half of the target number of participants.2 Rather than assuming that dropouts are inevitable, why not ask patients who have the specific condition what would help keep them enrolled in a trial?
In 2018, Inspire performed a study eliciting patient preferences and insights on clinical trials. Over 1600 Inspire members living with sarcoidosis, scleroderma, ovarian cancer, prostate cancer, colorectal cancer, arthritis, and psoriasis completed it. Among other results, the survey found that the barriers to clinical trial participation varied depending on the disease group. For example, transportation to clinical trials was significantly more influential in participating for people with scleroderma than the other conditions. Women with ovarian cancer were more likely to participate in a clinical trial if their current doctor was able to do the test. (Significance p<=.05.) The implication is that knowing participant needs and accommodating them in trial design supports the continued statistical validity of the findings.
Identifying factors influencing long-term adherence
Patients are the end users of any treatment, and patients with chronic conditions may be prescribed maintenance medications. What if a drug is clinically effective, but patients stop taking it because of the side-effects? What percentage of patients might do this? As the VP of U.S. Medical Affairs of Renal Cardio at AstraZeneca, Dr. Kerry Cooper, indicated in a 2019 interview,
“We must understand [a drug’s] overall value for [the patients], including right down to how they respond to taking it. If [an oral] therapy has a bad texture, bad taste, or is not palatable, they are not going to take it long-term.”3
What side effects are patients willing to accept? Determining what patients will tolerate during treatment design helps companies pursue treatments that patients decide are worth taking instead of abandoning. For example, for some patients taking statins, adherence falls off in real use. To some degree this is because the side-effects become more obvious to the user than the benefits of adherence.4Using NLP and qualitative hands-on sociolinguistic analysis
a recent study, conducted at Inspire with Dr. Farris Timimi, cardiologist at the Mayo Clinic, identified the side effect of memory loss or “instant Alzheimers” in patients’ communications about taking statins.5
Acceptable Risks and Benefits from Patient Perspective
Developing drugs that demonstrate a clinical effect but that patients won’t take is a waste of everyone’s resources. The FDA is investigating methods for quantifying patient preferences in the risks and benefits of a treatment — in addition to the scientifically determined risks and benefits — as guidance for regulatory decisions.6
The risks and benefits patients consider depend on the nature of the condition. The proposed benefit of adding patient perspectives on risk to regulatory approval is that the FDA can focus its resources on drugs and treatments that are most likely to succeed with patients beyond the clinical trial setting. In one initiative, CDRH sponsored a discrete-choice experiment case study to quantify obese respondents’ perspectives on “meaningful benefits.” The eventual decision to approve a device was based in part on the resulting data showing that a substantial portion of obese patients would accept the risks associated with a surgically implanted device if they lost a sufficient amount of weight. 6
Patient-centricity is not just a nicety; patient-centricity is being used to solve deficiencies in implementation. . These examples show ways in which private industry and regulatory agencies are using patient experience and patient-generated data to address the chronic problems of under enrollment in trials and medications that succeed in clinical testing but are less successful in patient adherence.
It feels effortful because the industry is in the experimental phase: stakeholders are testing various methods for statistically validating patient experience information and figuring out how to incorporate what it reveals into product development. There is a growing body of evidence, but no agreement on standards of proof. As the authors of one study put it:
…only patients live with their medical conditions and need to make the choices required for their care. To properly take these views into account, investigators must have reliable and accurate methods, tools, and approaches.6 ~ F. Reed Johnson PhD, Mo Zhou MA
Below we share a commercial marketing case study using both qualitative and quantitative strategies to understand the patient experience and understanding of diagnostic tests as well as their perspectives on marketing content and design.
Inspire offers a trusted community to patients and caregivers. Our goal with this blog, this website and our content is to provide the life science industry access to the true, authentic patient voice. In so doing, we support faithful operationalization of patient-centricity. Take a look at our case studies, eBooks and news outlet coverage.
1 https://pharmaphorum.com/views-analysis-patients/where-next-for-patient-centricity-in-pharma/ 11/08/2019
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