Although the FDA approved fewer novel new therapies last year than it did in 2015, the specialty drug pipeline remains strong. This new year should see approvals of products in areas that already have multiple competitors, such as anti-inflammatory conditions and multiple sclerosis, as well as treatments for conditions sorely in need of ones, such as tardive dyskinesia (TD) and non-alcoholic steatohepatitis (NASH).

“Due to increased trend and spend for specialty drugs year over year, we believe that payers should pay particular attention to the specialty pipeline in general,” says Eileen Pincay, senior pharmacy consultant in the national pharmacy benefits practice for The Segal Group. “At Segal we typically look at our clients’ top therapeutic categories and top drug spend and trend to identify where the most need of attention would be for our clients. Typically, we see that about a majority of pharmacy drug spend is represented in five key classes: multiple sclerosis, hepatitis C, cancer, inflammatory conditions (namely, rheumatoid arthritis) and HIV. Although the projected specialty drug/biotech trend rate for 2016 will decrease slightly from 2015 to 18.9% from 19.4%, specialty drug costs are predicted to represent 50% of overall drug costs by 2018.”

“Specialty drugs will continue as a large share of pharma’s pipeline,” agrees Dan Mendelson, president of Avalere Health. He points to oncology, particularly oral products; orphan and rare disease drugs; biosimilars; and medications for infectious diseases such as HIV that consist of single pill products. “Payers should also pay attention to drugs” for asthma, Duchenne muscular dystrophy (SPN 10/16, p. 1) and NASH, which has nine products in the pipeline, he tells AIS Health. “These high-cost/innovative products will require payers to continue to increase and enhance their utilization and clinical management strategies.”

Rare Conditions Could Finally Get Therapies

“This year there are exciting developments for several new drugs…on the horizon for rare conditions where there are no or very limited treatments today,” says Thom Stambaugh, vice president of specialty pharmacy at Cigna Corp. “These conditions include muscular dystrophy, Huntington’s disease, ALS and other degenerative neuromuscular diseases. This is tremendous news for our customers and their families. However, for the best clinical and financial results related to these health conditions, the focus needs to be broader than managing the new prescription. An integrated, connected care approach is necessary to help customers manage their total health across pharmacy, medical, behavioral and disability benefits.”

Benralizumab, AstraZeneca’s “subcutaneous interleukine-5 (IL-5) inhibitor for uncontrolled eosinophilic asthma [given] every four weeks, is expected to be approved later this year,” says Aimee Tharaldson, Pharm.D., senior clinical pharmacist advisor at Express Scripts Holding Co. “Benralizumab will be available in a prefilled syringe that is self-administered.”

She also points to the potential March approval of Regeneron and Sanofi’s Dupixent (dupilumab), “a subcutaneous injection IL-4 and IL-13 inhibitor, which will be given every two weeks for moderate to severe atopic dermatitis.…Data for dupilumab show very high efficacy rates given alone or in combination with topical glucocorticoids.” According to David Lassen, Pharm.D., chief clinical officer for Prime Therapeutics LLC, “This injectable drug is believed by its manufacturer to be a ‘blockbuster’ drug with an annual cost of $40,000-$50,000 on the pharmacy benefit.” Lynn Nishida, area vice president, pharmacy for Solid Benefit Guidance, tells AIS Health if Dupixent is approved, it “will be the first systematic therapy for atopic dermatitis. With a global prevalence of the disease and limited options to treat moderate to severe conditions, the drug’s commercial potential is significant.…Dupixent is anticipated to make a splash in a market that…to date has been limited to topical options (Elidel, Protopic, and now Eucrisa) and oral corticosteroids or immunosuppressant treatment.”

MS Drug Would Treat Rarest Form

Stephen Cichy, founder and managing director, Monarch Specialty Group, LLC, cites Roche’s Ocrevus (ocrelizumab), where interest “centers around this drug’s potential to treat both relapsing and primary progressive forms of multiple sclerosis, which has not yet been seen with current approved therapies. Primary progressive MS is the rarest form of the disease and is characterized as being progressive from the start. There are no acute relapses or remissions in PPMS, which affects about 10% of people diagnosed with the disease.”

The FDA’s action date for a decision on Ocrevus “was recently delayed three months until March,” notes Lassen. “Because there are currently no treatments available for PPMS, there could be significant uptake of Ocrevus for that population. There is a potential cost to this drug of $100,000 per year, so payers could see net new spend in this category.”

“Interest with Sanofi and Regeneron’s biologic drug sarilumab [brand name Saracta] centers around its Phase III clinical data highlighting, among other things, its ability to beat AbbVie’s Humira (adalimumab)…head-to-head,” Lassen tells AIS Health. That drug and Janssen and GlaxoSmithKline’s sirukumab may be approved later this year for rheumatoid arthritis, competing with Actemra. Baricitinib is a potential competitor to Xeljanz and Xeljanz extended release, and Siliq, if approved, would compete with Cosentyx and Taltz for psoriasis.

Tharaldson says three hepatitis C drugs “are expecting approval in the second half of 2017: glecaprevir-pibrentasvir (AbbVie) a next-generation protease inhibitor (PI) and NS5A inhibitor combination for genotypes 1 through 6; voxilaprevir-velpatasvir-sofosbuvir (Gilead), a PI, NS5A inhibitor and Nuc against genotypes 1-6; and MK-3682-elbasvir-grazoprevir (Merck), a Nuc NS5A inhibitor and a PI for genotypes 1-6.”

“The hemophilia segment is primed for potential new disruptive therapies leading into 2017, with drugs like Genentech’s once-weekly subcutaneous injection for prophylaxis (emicizumab) leading the way,” says Cichy. “Also, Alnylam’s early phase ALN-AT3 (fitusiran) is being explored as a once quarterly (or monthly) subcutaneous injection for prophylaxis. In each of these cases, focus is on delivering a more convenient and less burdensome solution for patients with hemophilia A.” Tharaldson adds that “Novo Nordisk is developing the long-acting factor IX, nonacog beta pegol, for hemophilia B, which will be given as a once-weekly IV infusion and approval is expected in May.”

Tharaldson says two potential treatments for TD, an involuntary, repetitive movement disorder that has no current treatments and affects around 500,000 Americans, are expected this year: Ingrezza (valbenazine) from Neurocrine Biosciences, Inc. and Austedo (deutetrabenazine) from Teva Pharmaceutical Industries, Ltd., for both TD and Huntingdon’s disease.

Lassen says another drug to watch for is “voretigene neparvovec, for the treatment of inherited retinal disease caused by mutations in the RPE65 gene. This is an emerging therapy that may have a high price and will be managed on the medical benefit.”

Pincay notes that the drug Ocaliva (obeticholic acid), which the FDA approved last May to treat primary biliary cholangitis (SPN 6/16, p. 6), “is being studied in NASH, and if approved…will be the first drug to treat NASH.” This condition “affects approximately 6 million to 16 million Americans,” and approval for this indication for the medication — which costs around $70,000 annually — has “the real blockbuster potential,” she tells AIS Health, as NASH is “a higher incidence condition.…Due to high costs…and potential for off-label use at the present time, there are some PBMs offering management to ensure appropriate use.”