Payer Perspectives on the Treatment Landscape of Spinal Muscular Atrophy (SMA) Findings from the AMCP Market Insights Program
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MARKET INSIGHTS Payer Perspectives on the Treatment Landscape of Spinal Muscular Atrophy (SMA) Findings from the AMCP Market Insights Program Groups and Employer Coalitions. Payers ranged in size Meeting Objective from less than 500,000 insured lives to nearly 10-million and, in total, covered over 40-million lives (Figure 1). • Understand how AMCP members identify and Discussions were led by two key opinion leaders who manage patients with SMA focused on best practices, the evolving SMA treatment • Identify how payers establish coverage criteria for landscape (including gene therapy) and the role of real new SMA therapies, including gene therapy world evidence on considerations for treatment decisions. • Assess the role of real-world data to better understand the impact of current and emerging Carrier and Newborn Screening SMA was added to the federal Recommended Uniform treatments for patients with SMA Screening Panel (RUSP) for newborn screening in 2018.4 The provision of carrier screening information is recommended by the American College of Obstetricians and Gynecologists for every pregnant woman.5 Prenatal Introduction carrier screening does not replace newborn screening. Spinal muscular atrophy (SMA) is a rare, autosomal Nor does newborn screening replace the potential value recessive genetic disease that causes progressive muscle weakness and atrophy. Approximately 6,000 children and of prenatal carrier screening. Genetic counseling about adults in the United States are affected, making SMA one potential reproductive outcomes should also be offered. of the most common rare diseases.1 SMA is divided into subtypes based on age of onset and maximum function “And it looks like earlier treatment achieved. SMA types are classified as 0, 1, 2, 3 and 4 and are all are associated with mutations in the Survival Motor is better — so support the prenatal Neuron 1 gene (SMN1) and SMN2 genes, which are located screening.” –Health Plan on chromosome 5q (Table 1).2 SMN1 creates SMN protein, a protein essential for motor neuron development. SMN2 also produces SMN protein but only a small percentage of Payer experts understood that timely newborn screening the protein produced by SMN2 can be used by the body. is a valuable tool to attain earlier treatment and stronger This deficiency causes the irreversible degeneration clinical benefit of preserved motor neuron function. Several of motor neurons, which leads to progressive muscle payers noted that intuitional policies are already in place to weakness and prevents patients from reaching motor support standard newborn screening for SMA. milestones or retaining motor functions. These muscles are necessary for crawling, walking, sitting up and head Care Management control. The more severe types of SMA can affect muscles The majority of people with SMA require constant support involved in feeding, swallowing and breathing. and attention by parents and caregivers. The condition Various medications are used to help those with is managed through multidisciplinary supportive care. SMA manage their symptoms but currently there is two However, supportive care strategies do not affect disease therapies. The SMA drug pipeline includes treatment progression, but aim to minimize the impact of disability, strategies to compensate fully or in part for the absence address complications and improve quality of life. These of SMN1 gene by increasing the level of functional SMN may involve respiratory, gastroenterology, and orthopedic protein via multiple approaches. The high upfront costs care, as well as nutritional support, physiotherapy, assistive and the cumulative budget impact of SMA treatments technologies, occupational therapy and social care.3 and the lack of long-term clinical durability data amplifies Payers acknowledged that SMA has a substantial effect treatment uncertainties and complicate payer coverage on the quality of life of patients, caregivers and their and reimbursement determinations. families. Some payers also noted their organizations were AMCP conducted a Market Insights program to identify mindful during policy development and decision making of perspectives of AMCP members on current and future the specific need to take into account the child population, therapies for SMA. The Market Insights program offered treatment timing, rarity and severity of the disease. by AMCP is a blinded research program sponsored by corporate member to address current conditions that are Current and Emerging Therapies a high priority among the AMCP membership. The payer Although recent progress has been made in experts in the SMA Market Insights Program represented a understanding molecular mechanisms underlying diverse mix of national and regional health plans, Pharmacy the pathogenesis of SMA, there is currently no cure. Benefit Managers, Integrated Delivery Networks, Physician Treatment involves prevention and management of August 2020 Market Insights 1 MARKET INSIGHTS Payer Perspectives on the Treatment Landscape of Spinal Muscular Atrophy (SMA) Table 1. Clinical Classification of SMA2 Highest Milestone Proportion of SMA Type Eponym Age at Onset Life Span Achieved Total SMA (%) 0 Prenatal < 6 months None < 1 1 Werdnig-Hoffman Prenatal - 6 months < 2 years without Never sits 60 respiratory support 2 Dubowitz 6-18 months 70% alive at 25 years Sits independently- 27 never stands 3 Kugelberg-Welander > 18 months Adulthood Stands and walks 12 4 Adult-onset Early adulthood Does not affect life Walk through < 1 (20s-30s) expectancy adulthood Adapted from Verhaart et al. 2017. the secondary effect of muscle weakness and loss. Zolgensma Clinically, even with early treatment, patients will The gene replacement therapy, onasemnogene continue to have symptoms of SMA. Developing abeparvovec (Zolgensma), is FDA approved to treat therapeutics for SMA have shown positive clinical patients less than two years of age with SMA.8 It results in various phases of drug development and uses the adeno-associated virus serotype 9 vector to routes of administration. replace a copy of SMN in order to supplement the defective SMN1 gene. Onasemnogene abeparvovec is given as a one-time intravenous administration. The “We’re talking about a therapy wholesale acquisition cost is $2.1 million per patient without which children generally die and falls within the upper bound of ICER’s value- .7,9 by two years of age.” –Physician based price benchmark range “This (gene therapy) is not a cure Nusinersen in the sense of it is a one time Nusinersen (Spinraza), an antisense oligonucleotide treatment... There is still going (ASO), was the first disease-modifying therapy to be impaired survival” to treat SMA when it was approved by the FDA –Physician in 2016.6 It targets the SMN2 gene to enhance its effectiveness. Nusinersen is administered via lumbar puncture with four loading doses and maintenance All but one of the payers stated that they have developed coverage criteria for onasemnongene doses every four months thereafter. An ICER review abeparvovec. It was common for payers to require found there is high-quality evidence that nusinersen genetic testing results and to limit coverage to one does benefits children with Type 1 SMA, and it appears per lifetime for a patient. to benefit presymptomatic SMA.7 Currently, the list price is $750,000 for the first year and $375,000 in Risdiplam subsequent years. Payer experts stated nusinersen is covered for Risdiplam is an investigational SMN2 splicing modifier and is an orally-administered liquid. It is designed patients with SMA, although some require evidence to durably increase and sustain SMN protein levels that the patient has a specific number of copies of the both throughout the central nervous system and in SMN2 gene (e.g., at least two copies). However, coverage peripheral tissues of the body. It is being evaluated is variable across plans and there may be benefit for its ability to help the SMN2 gene produce more limitations, such as nusinersen not being considered functional SMN protein throughout the body. medically necessary for individuals previously treated Risdiplam is expected to be submitted for FDA with gene therapy. In addition, some plans have approval in 2020. carved nusinersen out of the medical benefit and Payers are anticipating that risdiplam will have a broad are only covering it under pharmacy benefits due to label of SMA indications. They are anticipating managing better affordability and management tools. it under the pharmacy benefit and to distribute it through 2 Market Insights August 2020 MARKET INSIGHTS Payer Perspectives on the Treatment Landscape of Spinal Muscular Atrophy (SMA) Figure 1. Market Insights Participants the majority of payers stated that their organizations would likely evaluate a new therapy for SMA in less than six months of FDA approval. 8% Combination Therapies 17% and Sequencing 17% The availability of treatments for SMA that target independent genes (SNM1 and SMN2) and the potential for new neuroprotective options has led to discussions around combining therapies in the hopes 33% of better outcomes. Payers heard that the presence 25% of SMN2 can compensate for the SMN1 deletion to some degree and the number of SMN2 gene copies is inversely related to the severity of SMA, which can be used to predict the course of the disease. Currently there is no clinical trial evidence to support using Health Plan National Other multiple therapies concurrently or sequentially. How Health Plan Regional PBM effective treatments will be in the long