VP and Global Head Epidemiology & Benefit - Risk Evaluation, Sanofi Bridgewater, United States
Background: Patients with multiple sclerosis (M)S face a variety of everyday risks from their disease and treatment options due to the often heavily impactful nature of the disease and the serious risks of the treatments. Various studies have been done to evaluate different attributes among varying populations, but there remains a lack of understanding around what aspects of therapeutic interventions are valued most by patient groups.
Objectives: To better understand the perspectives of patients with MS deciding on treatment options and how they weigh and trade-off multiple benefits and risks.
Methods: A literature review was performed on MS patient preference studies focused on all disease stages. A third of the studies were among relapsing remitting (RR) patients, and most of them were conducted in Western countries. Of the 70 articles reviewed, 40 were included in the final evaluation: 36 quantitative, 3 qualitative, and 1 study with mixed methods. In over 80% of the studies, the majority of patients were women, consistent with the fact that women had a higher risk of developing MS than men.
Results: Among the most frequently reported preferences, efficacy in delaying disease progression, the clinical outcome of disease-modifying therapies (DMTs) most prescribed for MS, was of the highest importance. Other attributes that played an important role were mode and frequency of administration, with the strongest preference on oral administration at a low frequency, and risks of relapses and side effects Patients were willing to make various tradeoffs between risks of side effects and delayed progression and relapse. Patients would be willing to accept an increase in the frequency of serious risks of up to 3.8% as a tradeoff for a 1 year delay in the disease progression. Studies found that patients with less severe MS were more concerned with the frequency of mild side effects and less about preventing disease progression than those with more severe MS.
Conclusions: Overall, delaying disease progression was of the highest importance to patients. Patients were willing to make various tradeoffs between risks of side effects, different modes and frequencies of treatment administrations and delayed progression and relapse. Further research needs to be done to improve the understanding of patient preferences in treatment options to ensure that future medications meet patients’ desires and expectations better.