The development and approval of specialty pharmaceuticals is on the rise. In fact, between the Orphan Drug Act of 1983, which incentivized pharmaceutical manufacturers of orphan drugs, and 2012’s Safety and Innovation Act, which fast-tracked approvals for breakthrough therapies, the specialty drug pipeline has overtaken more conventional medications over the past few years.
In recent years, therapy classes showing the highest specialty development including medical conditions including inflammatory disease, oncology, multiple sclerosis, and the human immunodeficiency virus (HIV). Aimee Tharaldson, PharmD, senior clinical consultant at Express Scripts, says that drug manufacturers continue to look for ways to innovate and improve treatments for such conditions.
“The traditional market is relatively saturated and highly genericized,” says Tharaldson. “It’s more lucrative for manufacturers to develop specialty medications as shorter, less expensive trials are typically required, allowing manufacturers to set higher prices.”
Experts, including Tharaldson, expect that trend to continue. In the future, the specialty market will have more options available for patients—and will be in a better position to compete with generic and biosimilar drugs. And that is why, Tharaldson says, it is vital that managed care organizations are following the specialty pharmacy pipeline. She discussed the emerging specialty medication market in the session, “Specialty Pharmaceuticals in Development,” at the Academy of Managed Care Pharmacy (AMCP) Nexus event, to be held October 29-November 1, 2019, in National Harbor, Maryland.
When asked about the types of emerging specialty medications expected within the next 12 months, Tharaldson highlights impending drugs that can effectively treat moderate-to-severe atopic dermatitis, a condition that affects more than 5 million adults and children in the United States alone.
“Pfizer’s abrocitinib is a JAK inhibitor in development for moderate-to-severe atopic dermatitis. It is an oral, once-daily therapy that will compete with Sanofi and Regeneron’s Dupixent, which is administered every other week by subcutaneous injection,” she explains. “Abrocitinib has demonstrated efficacy and is well tolerated. In addition, Lilly and Incyte’s baricitinib (Olumiant) is expected to gain an atopic dermatitis indication in 2020 and AbbVie’s Rinvoq (upadacitinib) is expected to be approved for atopic dermatitis in 2021.”
She also notes that Vertex’s elexacaftor/tezacaftor/ivacaftor is expected to be approved by March 19, 2020, for the treatment of cystic fibrosis (CF) in patients aged 12 years of age and older with specific genetic indicators.
“Elexacaftor is a next-generation cystic fibrosis transmembrane conductance regulator (CFTR) gene corrector, tezacaftor is another CFTR corrector and ivacaftor is a CFTR potentiator,” she says. “This oral, fixed-dose combination tablet is taken in the morning and one ivacaftor tablet is taken in the evening. This new combination is estimated to be effective for 90% or more of patients who have cystic fibrosis. Current therapies are only effective for up to 70% of patients with this condition. Additional triple-drug therapies for cystic fibrosis from Galapagos and Proteostasis may reach the market in 2021.”
Taken together, these new drugs could significantly increase the number of patients being treated with potentially high-cost specialty medications—which, in turn, affect the budgets of employers and insurers.
“Ultimately, with these trends, drug prices could become unsustainable for employers and health plans without preparation and solutions that address cost drivers, including non-adherence, lack of quantifiable outcomes data, and fragmentation of care delivery,” she says. “Currently, 45% of per member per year drug spend under the pharmacy benefit is for specialty medications—and we’re expecting the spend on these drugs to account for over half of pharmacy drug spend in 2020. Knowledge of specialty pipeline medications can help prepare AMCP Nexus attendees for new drugs that are poised to impact the market.”
Kayt Sukel is a science and health writer based outside Houston.