Pipeline Update: Anticipated Specialty Drug Approvals

Sep 25, 2014
With the FDA expected to approve a record 25 specialty drugs in 2014, payers must anticipate future plan costs and build proactive strategies for utilization management.
  • Cancer
  • Hepatitis C

The short-term pipeline for specialty drugs is robust, with several of the new products having potential to significantly increase drug spend by the end of next year. One of these classes is hepatitis C, which has been the focus of many payers throughout 2014 following the unprecedented launch of Sovaldi®. Never before has a drug been priced this high to treat a patient population so large.

Hepatitis C is not the only disease state with new drugs in development or recently approved. With the FDA expected to approve a record 25 specialty drugs in 2014, payers must anticipate future plan costs and build proactive management strategies for utilization management.

Several of the recent approvals are for drugs to treat orphan diseases including:

  • Hereditary Angioedema: A new C1 esterase inhibitor (recombinant) – Salix and Pharming’s Ruconest® – was approved in July to treat adult and adolescent patients with hereditary angioedema, the fifth drug to treat a rare, orphan condition that affects less than 10,000 Americans.
  • Gaucher Disease Type 1: Approximately 6,000 patients in the U.S. have Gaucher disease, an inherited lysosomal storage disorder and CerdelgaTM offers an oral alternative to enzyme replacement therapy.

Another condition that has seen the recent introduction of two new breakthrough therapies is cancer:

  • Gilead’s Zydelig® (idelalisib) is a novel oral phosphoinositide-3-kinase (PI3K) delta inhibitor for the treatment of three B-cell blood cancers.
  • Keytruda® (pembrolizumab) by Merck is an immunotherapy for patients with unresectable or metastatic melanoma that helps the body’s immune system attack cancer cells. It’s the first programmed death receptor-1(PD-1)-blocking antibody to gain FDA approval.

Other significant approvals still expected in the near future include drugs to treat:

  • Chronic Hepatitis C: New all-oral hepatitis C regimens aimed at eradicating genotype one disease could be positioned to treat up to 75% of U.S. hepatitis C patients and according to some projections, may cost more than Sovaldi.
  • Idiopathic Pulmonary Fibrosis: Two novel products that will treat the underlying cause of IPF are pending approval. They represent a significant medical advancement to present therapies but are also expected to cost approximately $100,000 per patient per year (PPPY).
  • Cystic Fibrosis: Lumacaftor is a novel molecular corrector of the cystic fibrosis transmembrane conductance regulator. In combination with Kalydeco® (ivacaftor – Vertex Pharmaceuticals) it could correct the underlying cause of disease for 50% of CF patients in the U.S., but possibly at a PPPY cost of more than $300,000.
  • Cancer: More than 1,000 targeted cancer treatments, many genetically guided, are under development, including immunotherapies targeting the cellular checkpoint PD-1. These therapies have the potential to improve outcomes for a number of cancer types when used in combination with other therapies. Most also have an average monthly per patient cost of about $10,000.
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