Siponimod for the treatment of secondary progressive multiple sclerosis: effectiveness and value : final evidence report
- Collection:
- Health Policy and Services Research
- Author(s):
- Sharaf, Ravi, author Synnott, Patricia, author Samur, Sumeyye, author Chapman, Rick M., author Adair, Ellie, author Rind, David, author Pearson, Steve, author O'Grady, Madeline, author Bloudek, Lisa, author Carlson, Josh J., author
- Contributor(s):
- Institute for Clinical and Economic Review, issuing body.
- Publication:
- [Boston, Massachusetts] : Institute for Clinical and Economic Review, June 20, 2019
- Language(s):
- English
- Format:
- Text
- Subject(s):
- Multiple Sclerosis, Chronic Progressive -- drug therapy Sphingosine 1 Phosphate Receptor Modulators United States
- Genre(s):
- Technical Report
- Abstract:
- Multiple sclerosis (MS) is a chronic, immune-mediated inflammatory, demyelinating, and neurodegenerative disease of the central nervous system (CNS). Commonly-cited analyses estimate the prevalence of MS in the United States to approximate 400,000 Americans, although a recent analysis suggests the prevalence may be closer to one million. MS disproportionately affects women and is typically diagnosed between the ages of 25-45. The onset of symptoms often coincides with an individual’s most productive years at home, work, and in the community. Direct medical costs associated with MS management, coupled with indirect costs from lost productivity, have been estimated to total $24.2 billion per year in the United States. As price increases for MS disease-modifying therapies (DMTs) outpace prescription drug inflation, disease-related costs are expected to rise. The diagnostic criteria used to define MS have evolved over time. The clinical course has commonly been characterized as relapsing-remitting or progressive. Relapsing-remitting MS (RRMS) is a relapsing phenotype that is the initial presentation of 85% to 90% of MS patients. It is characterized by “relapses” which are discrete clinical episodes of neurologic deficits that usually reflect an inflammatory demyelinating event in the CNS, with or without recovery. Progressive MS comprises primary progressive MS (PPMS) and secondary progressive MS (SPMS). The clinical course in PPMS and SPMS is characterized and distinguished from RRMS by increasing neurologic disability that occurs independent of, or in the absence of, relapses. PPMS involves a progressive course from disease onset. SPMS is a progressive course that develops following an initial relapsing-remitting course. RRMS and progressive MS are categorized as “active” or “not active” based on the presence or absence of clinical relapse or inflammatory activity on magnetic resonance imaging (MRI). Studies conducted prior to the advent of MS DMTs showed that most patients with RRMS transitioned to SPMS within 25 years, though the risk of conversion may be lower, given early treatment with highly effective DMTs as well as changes in classification with newer imaging modalities that have greater sensitivity to detect CNS inflammation. Distinguishing between relapsing-remitting and progressive phenotypes can be challenging and the phenotypes can overlap. It can be difficult to determine whether a patient has truly transitioned to SPMS (i.e., accumulating disability independent of relapses) versus when they are having incomplete recovery from frequent relapses. The therapeutic goal in MS is to decrease disease activity and disability progression. The Food and Drug Administration (FDA) has approved more than 10 DMTs for “relapsing forms” of MS, although prior to March 2019, the FDA did not explicitly define what constituted “relapsing forms of MS” and whether active SPMS was included in this group. The only FDA-approved therapies with explicit indications for non-active progressive MS include ocrelizumab for PPMS and mitoxantrone for SPMS, although use of the latter had been limited by significant short and long-term risks
- Copyright:
- Reproduced with permission of the copyright holder. Further use of the material is subject to CC BY license. (More information)
- Extent:
- 1 online resource (1 PDF file (various pagings))
- Illustrations:
- Illustrations
- NLM Unique ID:
- 9918401086506676 (See catalog record)
- Permanent Link:
- http://resource.nlm.nih.gov/9918401086506676