Cost issues aside, outlook solid for continued rise of specialty Rx

Specialty and biotech medicines aimed at treating cancer, multiple sclerosis and rare diseases will dominate much of drug development in 2017 and beyond, one of the nation’s top specialty pharmacy providers concluded.

(To view the full Category Review, click here.)

In 2016, the number of novel new medicines approved by the Food and Drug Administration “declined substantially compared with each of the previous two years,” noted a new report on specialty drug approvals from Diplomat, the nation’s largest independent specialty pharmacy. But roughly half of the 22 newly developed drugs approved in 2016 are considered specialty pharmaceuticals. The FDA also cleared seven new biologically engineered medicines.

Despite the “perception that fewer novel new drug approvals signifies slowed growth in the specialty market, this number does not speak to the impact of the drugs that were approved … in each of the broad disease states covered by specialty pharmacy,” the report noted.

Specialty drug makers continue to train much of their research and development on four broad disease categories — cancer, autoimmune diseases including multiple sclerosis, hepatitis C and rare diseases.

Only two orally dosed oncology products, AbbVie’s Venclexta (venetoclax) and Rubraca (rucaparib) from Clovis Oncology, won FDA approval in 2016. But that relatively paltry approval rate belies the “overall high level of activity in the oncology pipeline,” Diplomat noted.

“While the number of new approvals was low in oncology for 2016, … there were 15 instances of an oncology agent gaining approval for an expanded indication, new dose formulation, new combination or coverage of an expanded age group,” noted the report.

Diplomat predicts that the pace of approvals for new cancer drugs will accelerate this year, with “multiple approvals … forecast in both breast and blood cancers.”

Oncology meds newly approved or up for expected approval in 2017 or early 2018 include two new treatments for acute myeloid leukemia, Midostaurin from Novartis and Enasidenib from Agios Pharmaceuticals and Celgene. Newly approved for the treatment of marginal zone lymphoma is Imbruvica from Abbvie and Janssen Biotech. Also in the approval pipeline are at least two new breast cancer treatments, including Puma Biotechnology’s Neratanib and Ribociclib from Novartis.

“Oncology will remain a prime focus for drug research and development in future years,” said Murray Aitken, SVP and executive director of QuintilesIMS Health. “Cancer treatments represent the largest category of the 225 new medicines expected to be introduced within the next five years,” he said.

On the immunology front, the FDA cleared two new treatments for autoimmune diseases in 2016, Zinbryta (daclizumab) from Biogen and AbbVie, and Eli Lilly’s Taltz (ixekizumab) to treat moderate to severe plaque psoriasis. Genentech’s Ocrelizumab, granted breakthrough therapy status by the FDA for treatment of primary-progressive MS, is coming in 2017. Also projected for this year are products aimed at atopic dermatitis/eczema — topical Eucrisa from Pfizer and Dupilumab from Regeneron Pharmaceuticals.

So-called orphan drugs targeting rare diseases also remain a prime focus for research and development. Of the 29 new drugs and biologics gaining FDA approval in 2016, nine earned orphan designation.