Perceptions Regarding the FDA Approval of MS Therapies

Background: The process for MS therapy approval by the United States Food and Drug Administration (FDA) may not be well-understood by all persons living with MS.  Little research has been conducted on the understanding of what approval by the FDA means in terms of risks and benefits, and whether the level of understanding differs among those who have taken disease modifying therapy (DMT).

Objectives: Assess the perceptions of and opinions on the FDA approval process by persons diagnosed with MS.

Methods: Focus groups and field testing involving people with MS resulted in a survey that was made available to participants of the North American Research Committee on MS (NARCOMS) Registry and through the National MS Society (NMSS) website in the spring of 2016.  Inclusion criteria included responses to at least one of the three FDA sections and completion of DMT history.

Results: Of the 3533 respondents, 78.1% were female and 92.3% Caucasian. Mean (SD) age at time of survey was 55.2(11.1) and disease duration 16.7(9.9) years. The current disability level as measured by Patient Determine Disease Steps (PDDS) median (IQR) score was 3(1, 5) or Early Gait (Mild Disability, Late Cane Use), with 31.8% reporting progression in prior 6 months. 88.9% indicated they have taken at least one DMT in the past, with 47.5% currently taking a DMT. When asked what does it mean when a drug has been “approved by the FDA” for use by people with MS, a higher proportion of those with a history of DMT use said that “the drug is now available for use by people with MS” (79.2 DMT Ever vs 72.6% DMT Never, p=0.0034), “the drug has been tested in people with MS” (78.2 vs 70.1%, p=0.0004), “the drug provides benefit for people with MS” (74.9 vs 58.1%, p<0.0001), “the benefits of the drug outweigh the risks of the drug” (55.9 vs 42.9%, p<0.0001).  A lower proportion of those that have taken a DMT thought approval means “the drug has no harmful side effects” (3.6 vs 8.6, p<0.0001) and that “FDA approval doesn’t mean anything about the drug” (5.5 vs 14.1%, p<0.0001).  There was no difference in the proportion thinking FDA approval meant that “The risks of the drug for people with MS are fully understood” (18.1 vs 20.8%, p=0.20) or “The drug is better than the drugs approved before for use by people with MS” (6.5 vs 8.6%, p=0.12).

Conclusions: Those with a personal history of DMT use have an   understanding of what FDA approval means for MS DMTs that is consistent with the official FDA mandate. However, regardless of their DMT history some persons with MS indicated that FDA approval has no meaning, that there are no harmful side effects, or that the effects are fully understood. These beliefs suggest that the approval process is either not fully understood by some people with MS or that there are competing beliefs about the roles and functioning of the FDA.