The Specialty Pharmacy Pipeline: What's Next?

In a session at the 2012 Academy of Managed Care Pharmacy Educational Conference, Aimee Tharaldson, PharmD, identified the top specialty drugs in the pipeline and highlighted the most recent therapeutic trends within each medication class.

Specialty Pharmacy Times author Aimee Tharaldson, PharmD, senior clinical consultant, Emerging Therapeutics, Express Scripts, recently conducted a session at the Academy of Managed Care Pharmacy Educational Conference 2012 on the specialty pharmacy pipeline. In her session, she identified the top specialty drugs on the horizon and discussed the role of these therapies in the treatment of specialty disease states. She also summarized the ways in which new therapies will impact the market.

Specialty Market Trends

Dr. Tharaldson provided a bit of background on the existing trends in the dynamic specialty landscape before moving on to describe the future implications of new mechanisms in the field. She noted that draft guidance on biosimilars was released in February 2012 and additional guidance is expected to be released in 2013. Until then, most of these types of products will not be approved through the 351K pathway; rather, these products will most likely be submitted through biologic license applications (BLAs). As a result, initially there will be more BLAs than 351Ks, and biosimilars will be treated more as competing brands. Although legal hurdles are expected, the interchangeability offered by biosimilars and biobetters will provide greater cost savings and in some cases, fewer adverse side effects.

Cancer drug development is a major trend in the specialty market, with 25% of the pipeline consumed by oncology treatments. Orphan drugs are also another focus in specialty. Oral specialty medications have been developed for rheumatoid arthritis, multiple sclerosis, hepatitis C, and cancer, effectively shifting spending away from the medical benefit to the pharmacy benefit.

Citing data from Express Scripts’ 2011 Drug Trend Report, Dr. Tharaldson stated that by 2014, 25% of spend will be attributed to specialty drugs, with the top 3 conditions (inflammatory conditions, multiple sclerosis, and cancer) accounting for more than 2/3 of spend. Less than 1% of patients use specialty drugs but represent 18% of spend. Until 2014, cancer treatments, oral therapies, and specialty medications for orphan diseases are expected to dominate FDA approvals.

Near-Term Specialty Pipeline

Inflammatory Conditions

Drugs to watch: tofacitinib, apremilast, Fos-D (fostamatinib), baricitinib, Actemra (tocilizumab), Arcalyst (rilonacept), Ilaris (canakinumab), Ozespa (briakinumab), LymphoCide (epratuzumab), sarilumab, secukinumab, sirukumab, vedolizumab.

In this class, oral drugs are expected to compete heavily with injectable treatments, and injectable biologics for rheumatoid arthritis, psoriasis, and irritable bowel disease are expected to be released. In addition, more specialty medications for lupus and gout are expected to be developed. Of particular note is Lupuzor (forigerimod acetate), a small peptide (as opposed to a monoclonal antibody) that modulates the immune system and has a unique mechanism of action.

Multiple Sclerosis (MS)

Drugs to watch: dimethyl fumarate (BG-12), laquinimod, masitinib, PEG Interferon beta-1a, Tceina (tovaxin), Copaxone generic (glatiramer acetate), ponesimod, Zenapax (daclizumab).

For MS, health care providers should expect more oral disease modifying drugs. Some drugs that are already currently marketed and approved for other indications—such as Zenapax—are expected to be approved for an MS indication in 2013. In the coming few years, generics and biobetters for the treatment of this condition are expected to impact the market, and new therapies for secondary-progressive MS are slated to be introduced. Last month, Aubagio (teriflunomide) was approved for relapsing remitting MS.

Cancer

Drugs to watch: cabozantinib, omacetaxine, ponatinib, dabrafenib, trametinib, actimid, T-DM1, tivozanib, Allovectin-7, Alpharadin (radium-223 dichloride), belinostat, Herceptin SQ (trastuzumab), Lucanix Vaccine (belagenpumatucel-L), MAGE-A3 ASCI vaccine, masitinib, Tvec Vaccine, tivantinib, Tovok (afatinib).

There is significant development in the cancer pipeline, and according to Dr. Tharaldson, half of the specialty pharmaceuticals in the entire pipeline are being developed for use in cancer. Many of the cancer drugs in the pipeline are administered orally. There are targeted therapies and vaccines in the works, and medications set to be released in 2013 will address niche/orphan cancer types and common cancer types. Dabrafenib and Trametinib, both from GlaxoSmithKline, are expected to be approved to treat metastatic melanoma with BRAF V600 mutations on February 3, 2013.

HIV

Drugs to watch: elvitegravir, cobicistat, Reyataz (atazanavir)/cobicistat, dolutegravir, dolutegravir/abacavir/lamivudine, Prezista (darunavir)/GS-7340/Emtriva (emtricitabine)/cobicistat, AGS-004, GS-7340.

In order to address the growing HIV population, more single tablet regimens are expected to be approved starting in 2013. In addition, there will be new novel integrase inhibitors in the pipeline, as well as the appearance of a therapeutic vaccine. Elvitegravir and dolutegravir, if approved, will be fierce competitors to Isentress (raltegravir).

Hepatitis C

Drugs to watch:

Novel interferons: INF-alpha-2b XL, interferon omega, locteron, peg-interferon lambda.

Direct acting antiviral pipeline: ABT-072, daclatasvir, filibuvir, GS-5885, GS-7977, mericitabine, setrobuvir, VX-222, ABT-333, BI-207127, PSI-938, tegobuvir, IDX-184.

Protease inhibitor pipeline: sovaprevir, BI-201335, TMC-435, vaniprevir, ABT-450, asunaprevir, danoprevir, GS-9256, GS-9451.

The development of oral protease inhibitors continues to grow, and several direct antivirals to treat hepatitis C are progressing. Novel interferons will hit the market in approximately 2 years. Dr. Tharaldson predicts an all-oral regimen for hepatitis C will come in the near future, but warns that nucleotide polymerase inhibitors, or “nucs,” should be watched closely for class effects, as a drug in this class has already been pulled from development as a result of a severe safety/toxicity issue.

Cystic Fibrosis (CF)

Drugs to watch: Bronchitol (inhaled mannitol), Tobi Podhaler, Aeroquin (levofloxacin inhalation solution), ataluren, Arikace (amikacin liposomal), Aerosurf (lucinactant), lumacaftor.

New drugs in the pipeline for CF work by increasing mucus clearance, treating the underlying disease, and fighting infections. Before the approval of Kalydeco (ivacaftor), CF was an orphan condition with no available treatments. Unfortunately, Kalydeco is only for patients 6 years and older who have the G551D mutation in their CF gene, and is not for use in people with other mutations in the CF gene. Lumacaftor, an oral tablet which will be used to treat patients with the F508del mutation, was recently studied in combination with Kalydeco, according to Dr. Tharaldson, and this medication from Vertex is likely to be approved in 2015.

Other notable specialty drugs that are in the pipeline include treatments for conditions such as pulmonary arterial hypertension, Cushing’s disease, bleeding disorders, hypercholesterolemia, short bowel syndrome, urea cycle disorders, nephropathic cystinosis, chronic fatigue syndrome, and hemophilia B.

For more information on the specialty drug pipeline, see Dr. Tharaldson's article, Near-Term Specialty Highlights.