An Update on the Specialty Pharmaceuticals Pipeline

Tori Socha

Minneapolis—In a contemporary issues session at the AMCP meeting titled Specialty Pharmaceuticals Pipeline Update, attendees heard from Aimee Tharaldson, PharmD, senior clinical consultant, emerging therapeutics, at Express Scripts. Dr. Tharaldson began the session by listing the objectives of her presentation: describe key specialty market trends, discuss the current specialty market, and highlight the near-term specialty pipeline.

The key specialty trends identified by Dr. Tharaldson included treatments for many conditions shifting from traditional therapies to specialty medications; manufacturers focusing on the specialty market; the introduction of new oral drugs for the treatment of rheumatoid arthritis (RA), multiple sclerosis (MS), and hepatitis C; and the development of specialty drugs for the treatment of rare diseases and cancer.

Dr. Tharaldson then turned to the state of the current specialty market, noting that the trend in spending and utilization of specialty medications continues to dwarf that of traditional drugs. In 2010, 74% of medical and drug spending was in the traditional market, with 14% in the specialty medical market and 12% in the specialty drug market; in 2013 the projections are for 60% of total spending to be in the traditional market, with 22% of spending in the specialty medical market and 18% in the specialty drug market.

The trend toward the specialty market is also discernible in the number of new drug approvals from the US Food and Drug Administration. In 2008, there were 16 traditional and 8 specialty drugs approved, in 2009 there 15 traditional drugs and 10 specialty drugs approved, and in 2010, there were 8 traditional drugs and 17 specialty drugs approved.

Recent specialty drug approvals mentioned by Dr. Tharaldson were Egrifta (tesamorelin injection; indication: lipodystrophy; approval: 11/10/10), Halaven (eribulin mesylate; indication: breast cancer; approval 11/15/10), Xgeva (denosumab; indication: bone metastases; approval 11/18/10), Makena (hydroxyprogesterone caproate; indication: prevent preterm birth; approval 2/3/11), Corifact (factor XIII concentrate [human]; indication: factor XIII deficiency; approval 2/17/11), Benlysta (belimumab; indication: lupus; approval 3/9/11), and Yervoy (ipilimumab injection; indication: malignant melanoma; approval 3/25/11).

The final section of the presentation was an overview of the near-term specialty pipeline. Therapy classes with specialty drugs in development include inflammatory conditions, MS, cancer, hepatitis C, lysosomal storage disorders, and cystic fibrosis. Trends in the drugs in the pipeline for inflammatory conditions indicate that oral biologics will compete with injectables, with new therapies for the treatment of gout and lupus and what Dr. Tharaldson called “biobetters” on the horizon. Approvals for biologics for the treatment of inflammatory conditions (RA, gout, psoriasis, and lupus) are expected beginning in August 2011 through 2013.

Trends in the MS pipeline also indicate an emphasis on oral disease-modifying drugs, with possible use of combination therapies for MS and expanded use of currently marketed drugs for the treatment of MS. Drugs in the MS pipeline are expected to receive FDA approval from 2012 through 2014.

Cancer pipeline trends indicate that cancer will be treated as a chronic condition with oral drugs and targeted therapies. Drugs are being developed to treat niche or orphan cancer types, and the role of pharmacogenomics in cancer treatment is steadily increasing. Drugs pending approval in the cancer pipeline are expected to be approved between now and November 2011. Other cancer treatment drugs are in the pipeline for approval through 2012.

Hepatitis C pipeline trends indicate that oral protease inhibitors will be “blockbusters,” Dr. Tharaldson said. She also said within 2 to 3 years novel interferons will be available for the treatment of hepatitis C. Drugs in the pipeline are slated for approval in 2013 and 2014.

Lysosomal storage disorder pipeline trends show development of treatments for Gaucher disease and Fabry disease, with new enzyme replacement therapies in the pipeline. Possible approval dates for drugs in this class are late 2011 through 2013.

Trends in the cystic fibrosis pipeline include new drugs intended to increase mucus clearance, treat the underlying disease, and fight lung infections. Projected approval dates are 2011 through 2012, Dr. Tharaldson said.