Improving quality, affordability, and equity of multiple sclerosis care

Abstract

Objective The prevailing approaches to selecting multiple sclerosis (MS) disease modifying therapies (DMTs) have contributed to exponential increases in societal expenditures and out‐of‐pocket expenses, without compelling evidence of improved outcomes. Guidance is lacking regarding when and in whom the benefits of preventing MS‐related disability likely outweighs the risks of highly effective DMTs (HET) and when it is appropriate to consider DMT costs. Our objective was to develop a standardized approach to improve the quality, affordability and equity of MS care. Methods MS experts partnered with health plan pharmacists to develop an ethical, risk‐stratified, cost‐sensitive treatment algorithm. We developed a risk‐stratification schema to classify patients with relapsing forms of MS as high, intermediate or low risk of disability based on the best available evidence and, when the evidence was poor or lacking, by consensus. DMTs are grouped as highly, modestly or low/uncertain effectiveness and preferentially ranked within groups by safety based on pre‐specified criteria. We reviewed FDA documents and the published literature. When efficacy and safety are equivalent, the lower cost DMT is preferred. Results Assignment to the high‐risk group prompts treatment with preferred HETs early in the disease course. For persons in the intermediate‐ or low‐risk groups with cost or health care access barriers, we incorporated induction therapy with an affordable B‐cell depleting agent. Based on more favorable safety profiles, our preferred approach prioritizes use of rituximab and natalizumab among HETs and interferon‐betas or glatiramer acetate among modestly effective agents. Interpretation The risk‐stratified treatment approach we recommend provides clear, measurable guidance in whom and when to prescribe HETs, when to prioritize lower cost DMTs and how to accommodate persons with MS with cost or other barriers to DMT use. It can be adapted to other cost structures and updated quickly as new information emerges. We recommend that physician groups partner with health insurance plans to adapt our approach to their settings, particularly in the United States. Future studies are needed to resolve the considerable uncertainty about how much variability in prognosis specific risk factors explain.

The Multiple Sclerosis Treatment Optimization Program

“…The primary strategy to increase the use of HET was the creation of a risk‐stratified MS treatment algorithm described in detail elsewhere. 10 Briefly, the algorithm helps clinicians judge when the risk of intermediate or long‐term disability from relapsing forms of MS outweighs the rare, but serious side effects of the preferred HETs, natalizumab or rituximab. The algorithm is based on the best available evidence, and when the evidence is weak or absent, consensus.…”

“…This preferred formulary prioritizes the safest DMTs within the HET or meDMT groups, and the lowest cost within group DMT when efficacy and safety profiles are similar. 10 Preferred HETs were natalizumab, rituximab, fingolimod and preferred meDMTs, the lowest‐priced Interferon‐beta and/or glatiramer acetate product when it was launched in 2013. Dimethyl fumarate and teriflunomide have inferior safety profiles, yet similar efficacy to glatiramer acetate or interferon‐betas, and therefore were classified as non‐preferred meDMTs.…”

“…Dimethyl fumarate and teriflunomide have inferior safety profiles, yet similar efficacy to glatiramer acetate or interferon‐betas, and therefore were classified as non‐preferred meDMTs. 10 If a patient cannot tolerate the preferred formulary DMT or take it as prescribed for other reasons, switching to another DMT, including non‐preferred DMTs as appropriate, is strongly recommended. This recommended formulary contrasts with the health plan's unpopular and unsuccessful approach of requiring that the lowest cost DMT be used preferentially, regardless of its efficacy or safety or a pwMS underlying risk of disability.…”

“…This MS specialist group meets monthly for 1 h during their regularly scheduled clinic time, in lieu of seeing patients, to review data, develop, and update best practice recommendations. 10 …”

“…We developed evidence‐based criteria for classifying DMTs by efficacy on clinical outcomes (e.g., relapses and disability progression) in relapsing forms of MS described in detail elsewhere. 10 Briefly, HETs are those that have demonstrated evidence of superiority to an active comparator in at least one head‐to‐head RCT and/or evidence of potency defined as a large magnitude of effect in an RCT conducted in a population with highly active relapsing MS or a positive RCT conducted in pwMS who relapsed on modestly effective DMTs. In 2010, three HET products (natalizumab, rituximab, and fingolimod) and five meDMT products (four interferon‐betas and one glatiramer acetate) were available for use.…”