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Oral specialty drug options on the horizon


As more specialty drugs are approved, cost control strategies are key.

Specialty medications treat complicated, potentially life-threatening conditions such as cancer, hepatitis, nervous system or blood disorders, and autoimmune conditions, according to National Pharmaceutical Services. CMS defines a specialty medication as a drug which costs more than $670 per month.


“Treatment for complex or life-threatening health conditions includes the use of certain drugs broadly referred to as specialty drugs,” says Farrah Wong, PharmD, senior director, pipeline and drug surveillance at OptumRx. “These are typically made using advanced biotechnology methods and are referred to as ‘biologics’ or ‘large molecules.’”

While no standard definition exists, Wong explains, specialty drugs are defined as having one or more of the following characteristics:

• Complex to manufacture, requiring special handling and administration

• Injectable or oral, self-administered or administered by a healthcare provider

• Costly, both in total and on a per-patient basis

• Taken by a relatively small share of the population who have complex medical conditions

• Difficult for patients to take without ongoing clinical support; challenging for providers to manage


“Specialty medications delivered through injection often require the use of complex equipment and may have added risk for infection; several specialty conditions now have oral treatment options,” says Aimee Tharaldson, PharmD, senior clinical consultant in the Emerging Therapeutics department at Express Scripts.

For OptumRx in 2016, specialty drugs accounted for approximately 1% of all pharmacy prescriptions and 35% of cost, says Wong. The top two drug classes contributing to specialty trend were chronic inflammatory and oncology conditions. In the past year, the trends for these drug classes increased by 33.2% and 20.1% for chronic inflammatory and oncology products, respectively.

Pipeline and payer impact

According to Wong, oral specialty drugs coming up in the pipeline include Translarna (ataluren) and tezacaftor (VX-661) in combination with Kalydeco (ivacaftor).

Translarna is an orphan specialty drug in development for the treatment of nonsense mutation dystrophinopathy, including nonsense Duchenne muscular dystrophy. Translarna will likely exhibit weight-based dosing, so fixed pricing is not anticipated. Analysts speculate that the average annual cost of the drug may reach $500,000 or more.

Tezacaftor/ivacaftor is an oral orphan specialty drug in development for treating cystic fibrosis (CF) patients exhibiting a homozygous or heterozygous F508del mutation. Tezacaftor/ivacaftor can potentially treat up to 90% of the CF population. Due to the limited options available for CF, cost will likely remain expensive, upward from the mid $200,000s annually.

Tharaldson makes note of more oral specialty drug approvals:

• Idhifa (enasidenib) was approved in August for the treatment of adult patients with relapsed or refractory acute myeloid leukemia (AML) who have a specific genetic mutation. The drug is approved for use with a companion diagnostic, the RealTime IDH2 Assay, which is used to detect specific mutations in the IDH2 gene in patients with AML.

Idhifa is an oral protein restoration therapy for treating patients with relapsed or refractory acute myeloid leukemia with IDH2 mutations. Approximately 21,000 Americans are diagnosed with AML annually in the United States and up to 19% have IDH2 mutations. Pricing information is not yet available, however it may cost approximately $150,000 per year.

• Mavyret (glecaprevir and pibrentasvir) was approved to treat adults with chronic hepatitis C virus (HCV) genotypes 1-6 without cirrhosis or with mild cirrhosis, including patients with moderate to severe kidney disease and those who are on dialysis. Mavyret is also approved for adult patients with HCV genotype 1 infection who have been previously treated with a regimen either containing an NS5A inhibitor or an NS3/4A protease inhibitor but not both. According to the FDA, Mavyret is the first eight-week treatment approved for all HCV genotypes 1-6 in adult patients without cirrhosis who have not been previously treated. Standard treatment length was previously 12 weeks or more. It was approved in August 2017 and may cost around $75,000 per year.

• Vosevi (sofosbuvir and velpatasvir-and a new drug, voxilaprevir) was approved in July 2017 to treat adults with chronic HCV genotypes 1-6 without cirrhosis (liver disease) or with mild cirrhosis. Vosevi is the first treatment approved for patients who have been previously treated with the direct-acting antiviral drug sofosbuvir or other drugs for HCV that inhibit NS5A. The treatment may cost approximately $75,000 per year.

As more specialty drugs are approved, it is crucial that strategies to control spending are implemented in the managed care setting.

This article was updated 9/12/2017.


Erin Bastick, PharmD, RPh, is a staff pharmacist at Southwest General Health Center, Middleburg Heights, Ohio.


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