Multiple Sclerosis: Understanding the Value of Novel Treatments

Multiple sclerosis (MS) is chronic, inflammatory immune-mediated disease in which the immune system mistakenly attacks myelin in the central nervous system. Worldwide, this debilitating neurologic disorder impacts 2.3 million people, and about 400,000 persons in the United States. The majority diagnosed with the disease are between 20 and 40 years of age. The most common pattern of disease is relapsing-remitting MS affecting approximately 85% of individuals, according to the National Multiple Sclerosis Society.

Treatment Costs & Options

MS imposes a significant economic burden on patients and the US health care system. The biggest cost driver for patients with MS are prescription drugs, specifically disease-modifying therapies (DMTs) which account for more than half of all direct medical costs. Recent data estimate that the total disease burden is approximately $2.5 billion and lifetime costs for individuals battling this disease exceed $4 million. In a recent study, published in Journal of Clinical Neuroscience, researchers investigated the recent trend of medical cost and financial burden of MS management in the United States using available data from the National Inpatient Sample database (from 1994-2013). The analysis showed an estimated half million increase in MS admissions, annually, which was projected to exceed 43.5 million by end of 2017.

A separate study that looked at the economics and cost-effectiveness of MS therapies in the United States noted that current costs for most DMTs exceed $70,000 a year, and are generally not cost-effective. 

No cure yet exists for MS, and treating MS is a balancing act between fighting the immune response and allowing the immune system to continue to defend the body. Patients with MS will require DMTs to alleviate symptoms and manage exacerbations; DMTs include injectable, oral, and infused therapies (Table).

The newest drug to enter the market is Genentech’s Ocrevus (ocrelizumab) in March 2017. This monoclonal antibody was FDA approved for the treatment of primary-progressive MS (PPMS) or relapsing MS. Prior to its approval, no medication was available to treat PPMS, which affects 15% of MS patients, explained Amiee Tharaldson, PharmD, senior clinical consultant of emerging therapeutics at Express Scripts, during a session on specialty pharmacy pipeline drugs at AMCP Nexus 2017.

This first-line treatment, which targets CD20-positive B cells, is administered by intravenous infusion every 6 months. The first dose is given in 2 infusions, 2 weeks apart. Subsequent doses are given as a single infusion. The efficacy and safety of Ocrevus was assessed in the ORATRIO and OPERA I and OPERA II studies. In the ORATORIO study, Ocrevus compared with placebo significantly reduced the risk of progression of clinical disability by 24% in patients with PPMS. In the OPERA I and OPERA II studies involving patients with relapsing MS, Ocrevus significantly reduced the annualized relapse rate by up to 47% compared with Rebif over 2 years. 

MS Drug Pipeline

Dr Tharaldson noted that there is not a lot going on in the “near-term MS market. It is going to be a few more years before we see more drugs approved for progressive MS—so it is going to be Ocrevus for at least the next 3 years.”

Perhaps, the big story in the MS pipeline was in October 2017 when Mylan announced the launch of its generic of Copaxone—both as a 40mg/mL3 times a week treatment and as a 20-mg/mL once-daily injection for patients with relapsing forms of MS. “Over 80% of the market share has shifted to Copaxone 40 mg. Our annual sales of Copaxone is $3.6 billion. Mylan’s generic was launched at about a 10% discount. Once we see additional generic manufacturers launch their generics to Copaxone 40 mg, we can see that price drop in the future,” said Dr Tharaldson.

Additionally, competitors to Gilenya are nearing FDA approval for relapsing MS, she said. Celegene’s ozanimod could see FDA approval mid-2018 and Novartis’ siponimod could be approved in 2019. Both are oral therapies taken once daily.

Implications for Stakeholders

The availability of increasing DMTs has provided patients and clinicians with a multiplicity of therapeutic options for MS. However, payers must balance cost, outcomes, and access in decision-making for MS formularies. Additionally, the effect of high drug prices on patient care needs to be considered.

According to Dr Tharaldson, Genentech set the price of Ocrevus at $65,000 a year, nearly 20% below the current market average for an MS treatment. Daniel Hartung, PharmD, MPH, noted in a study in Neurotherapeutics that the single largest payer of MS-related costs in the United States is the Medicare program. His research found that Medicare Part D expenditures for 12 DMTs from 2011 to 2015 more than doubled from $1.5 billion to $4.0 billion. The study also showed that in annual cost of DMTs ranged from $65,000 to $86,966.