Patients Rising Now advocates on behalf of patients with life-threatening conditions and chronic diseases for them to have access to vital therapies and services. Access is a matter of survival and quality of life for those patients, and it spans affordability, insurance coverage, and physical access. To support improved access, we are committed to engaging all stakeholders to foster realistic, patient-centered, solution-oriented discussions for particular conditions and the entire U.S. health care system. That is, our goal is a balanced dialogue that illuminates the truth about health care in a just and equitable way.

We appreciate the opportunity to provide our comments on ICER’s March 14thDraft Evidence Report about “Siponimod for the Treatment of Secondary Progressive Multiple Sclerosis.”

Patient-Center Perspectives

We want to congratulate ICER for working prospectively with the MS Coalition on the survey of MS patients to help illuminate patient specific perspectives and concerns. Doing this is a great step forward for ICER, and is particularly important because of the lack of patient-reported information in the single clinical trial for the compound of interest for this draft report, which specifically noted “The EXPAND trial did not evaluate many patient-reported outcomes, and quality of life measurements were conspicuously absent from the results.”[i]

However, we do want to note that the survey respondents were overwhelmingly white,[ii]and recent data has shown that the incidence of MS in blacks is not lower than whites, as had previously been believed, but may actually be greater, and further, they may have different patterns of manifestation and progression.[iii]We want to raise this issue because demographic differences may lead to different patient-centered concerns and perspectives about insurance coverage, access, and affordability, as well as the quality of life parameters. For example, for U.S. adults 19 to 64 years old, blacks are much more likely to be uninsured compared to whites (14% v. 8%).[iv]The importance of insurance coverage for patients receiving appropriate treatments is well known, and the draft report also notes that even people insurance can face barriers to accessing treatments: “Clinicians are sometimes hesitant to label a patient as ‘progressive’ given that doing so may eliminate insurance coverage for certain medications.”[v]

As you know, MS is now a long-term progressive condition. That is, with currently available treatments, people with MS can expect a relatively long life compared to people with other neurodegenerative diseases such as ALS or Alzheimer’s. This means that people with MS have a greater opportunity to benefit from newer treatments that may be developed in their life after they have been diagnosed. This value of hope is an important consideration for evaluating new treatments that may have incremental benefits in slowing progression of diseases such as MS where the expectation for future treatments may be categorized as slowing disease progression, stopping disease progression, and reversing disease progression.

Another patient perspective issue is how oral treatment affects access, particularly when the other treatment options are infusions or injections that require going to a doctor’s office or clinic. Specifically, for people with MS who have mobility problems or problems getting assistance with transport, oral forms may be a more feasible and realistic treatment option. And for people with MS who are working, not having to go to get infusion twice a year also would likely mean not having to miss two days of work. And oral treatment options also reduce disutility for caregivers by reducing transportation support and time obligations. In addition, different coverage rules (such as step-therapy requirements), and cost-sharing structures between pharmacy and medical benefits (i.e., between treatment with a pill versus an infusion), can create an uneven decision playing field for patients and clinicians as they try to choose between different treatment options. Those economic and coverage rule barriers can interfere with pure clinically based shared patient-clinician decision making. We recognize that the draft report notes some of these differences in its discussion of Coverage Policies[vi]but it would be better if ICER also explored the variability for coverage differences – particularly between private insurance plans and Medicare.

We would hope that these patient perspectives and factors would be extensively discussed at ICER’s May 23rdmeeting about this topic, and presented in depth in the final report.

Read the complete comment letter here.

[i]Draft report, “Siponimod for the Treatment of Secondary Progressive Multiple Sclerosis,” p. 37

[ii]Draft report, “Siponimod for the Treatment of Secondary Progressive Multiple Sclerosis,” p. 11

[iii]“Incidence of multiple sclerosis in multiple racial and ethnic groups,” Neurology. 2013 May 7;80(19):1734-9, and “African Americans: National Multiple Sclerosis Society,” 3/22/19)

[iv]Kaiser Family Foundation, “Changes in Health Coverage by Race and Ethnicity since Implementation of the ACA, 2013-2017” Figure 5.

[v]Draft report, “Siponimod for the Treatment of Secondary Progressive Multiple Sclerosis,” p. 2

[vi]Draft report, “Siponimod for the Treatment of Secondary Progressive Multiple Sclerosis,” p. 16