Bringing patients' views into medical approvals

2021-05-12

Patients want to have a say in decisions that affect their health. But decision-makers have not had the tools to listen. Patient preference studies offers just that: a tool for decision-makers to collect, and for patients to give, representative and well-informed input. Karin Schölin Bywall’s dissertation reveals when and how including patient preferences in regulatory decision-making.

Karin Schölin Bywall
Karin Schölin Bywall 

Decisions about what drugs should be developed, approved, to what extent they should be reimbursed are usually informed, if at all, by individual patients or small groups of patients. They are often helicoptered in for short periods of time and for, sometimes very, specific decisions. But one patient cannot represent all patients. This is where patient preferences can be useful.

Patient preference studies are tools to measure benefit-risk trade-offs patients make between different treatment options. For example, weighing one treatment option against another, one might choose the drug with a low risk of severe side effects over another drug, even if it is less effective to treat the disease. Knowing what patients prefer can offer valuable insight to decision-makers.

Karin Schölin Bywall, PhD student at Uppsala University's Centre for Research Ethics & Bioethics, has identified 15 decision-points where companies that develop medicines, the agencies that approve them, and in the health technology assessment bodies could benefit from patient preference information. But guidance on how to include patient preferences is needed. As part of her PhD project, Karin Schölin Bywall is conducting a case study within the PREFER project. Designed to offer just that, guidance on when and how to include patient preferences in decision-making.

Karin Schölin Bywall's dissertation
Karin Schölin Bywall's dissertation

“Patient preference information has the potential to reveal what patients prefer. What benefits they are looking for from a treatment, and what risks they want to avoid. By systematically examining patient preferences, patient input can be included next to clinical data in decision-making. Offering a long sought-after way of including the patient perspective in a way that can make a difference,” says Karin Schölin Bywall.

Making sure participants have the information they need to make well-informed trade-offs between different treatment options is key in any discrete choice experiment (or DCE, for short). The patients who participated in Karin Schölin Bywall’s DCE survey were informed about the different treatment attributes they were going to be asked to make trade-offs between through either plain text or a digital educational tool. Examining if preferences differ depending on how information is delivered can offer insight into best practice. And it turns out patients who were informed through the digital tool were more concerned about potential side-effects than those who were informed through plain-text.

By exploring the preferences of patients with Rheumatoid Arthritis (RA), Karin Schölin Bywall illustrates the many different preference patterns that can exist within the same patient population. What was most important to RA patients was the treatment’s likelihood to cause severe side effects, how effective it is, and how it is administered. Patients who valued efficiency were also more likely to accept severe side effects.

“Patient preference studies holds great potential. But it is important to examine how to perform them in the best way possible. That way, those who perform them can ensure they are valuable. And that the results can be used to inform decision-making along the medical product life cycle,” says Karin Schölin Bywall.

By Anna Holm

Schölin Bywall, K. (2021) Getting a Say: Bringing patients’ views on benefit-risk into medical approvals. [Dissertation]. Uppsala University. 

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