Almost no medications are without risks of side effects. When new drugs are approved, decision makers must balance risks and benefits. To make the balancing, they use results from clinical trials where the drugs are tested on patients to determine (among other things) efficacy and side effects.
But how do you balance risks and benefits? Is the balancing completely objective, so that all that is needed is results from clinical trials? Or can risks and benefits be valued differently?
It has been noted that decision makers can value risks and benefits differently from patients. Therefore, results merely from clinical trials do not suffice. Decision makers also need to understand how the patients themselves value the risks and the benefits associated with treatments of their disease. The patients need to be asked about their preferences.
Karin Schölin Bywall is a PhD student at CRB. She plans to carry out preference studies with patients suffering from rheumatoid arthritis. The task is complex, since risks and benefits are multidimensional. Rheumatoid arthritis is a chronic disease with several symptoms, such as pain, stiffness, fatigue, fever, weakness, deformity, malaise, weight loss and depression. Medications can be variously effective on different symptoms, while they can have a range of side effects. Which positive effect on which symptom is sufficiently important for the patients to outweigh a certain level of one of the side effects?
Many patients naturally want the drug to enable them to work, despite the disease. However, if the pain is relieved enough to enable carrying out the work, while the medicine has as a side effect such fatigue that the patient cannot get out of bed, then the desired benefit is not provided.
To prepare her preference study, Karin Schölin Bywall decided to approach the patient group immediately. From the very beginning, she wanted to engage the patients in her research, by interviewing them about how they perceive participating in preference studies on new drugs against rheumatoid arthritis.
The patients stated that they saw it as important to be involved in regulatory decisions about new treatments of their disease. So that decision makers understand the patients’ own experiences of the benefits and risks that such drugs may have, and what the benefits and risks mean in practice, in the daily life of a rheumatic.
Results from the interviews are reported in the journal, The Patient – Patient-Centered Outcomes Research. The article emphasizes that preference studies can lead to drugs that the patient group is more motivated to take according to the physician’s instructions, which can improve clinical outcomes in the patients. The patients further stated that as participants in preference studies they want good information about how the drug functions, about how the study will be used by decision makers, and about where in the decision-making process the study will be used.
Feedback from patients is likely to become increasingly important in future decisions on medical products.
Schölin Bywall, K.; Veldwijk, J.; Hansson, M. G.; Kihlbom, U. “Patient Perspectives on the Value of Patient Preference Information in Regulatory Decision Making: A Qualitative Study in Swedish Patients with Rheumatoid Arthritis.” The Patient – Patient-Centered Outcomes Research, 2018. DOI: 10.1007/s40271-018-0344-2
Recent Comments