Growing Trend Toward Specialty Drugs

Cincinnati—Although <1% of patients use specialty medications, they represent >15% of drug spend, and the percentage is likely to increase. In each of the past 3 years, the FDA has approved more specialty than traditional drugs.

The trend is expected to continue, according to Aimee Tharaldson, PharmD, senior clinical consultant of emerging therapeutics at Express Scripts. Dr. Tharaldson discussed specialty drugs in a contemporary issues session at the AMCP meeting.

Express Scripts defines specialty drugs as costing >$500 per month, having limited distribution and requiring frequent dosing adjustments, intensive clinical monitoring and patient training, compliance assistance, and specialized handling and administration.

Dr. Tharaldson said the introduction of biosimilars may help contain the costs and proliferation of specialty drugs. The Patient Protection and Affordable Care Act (ACA) contained a provision called the Biologics Price Competition and Innovation Act, which created an abbreviated approval pathway for biologics.

In February 2012, nearly 2 years after the ACA passed, the FDA issued draft guidelines for biosimilars, which are products that are highly similar to or interchangeable with FDA-approved biologics. More guidelines are expected next year, according to Dr. Tharaldson, who added that she expects legal hurdles before biosimilars are approved.

Until then, Dr. Tharaldson said healthcare professionals will see more specialty drugs, which have only recently become prevalent. She noted the following disease states have seen significant increases in FDA-approved specialty drugs from 2004 through 2012: hereditary angioedema, renal cell carcinoma, multiple sclerosis, rheumatoid arthritis, and pulmonary arterial hypertension.

Of specialty drugs in the pipeline, 25% are indicated to treat cancer, and there is a trend toward oral medications for oncology, rheumatoid arthritis, multiple sclerosis, and hepatitis C.

In 2011, the total per member per year (PMPY) cost of specialty drugs was $172, with the following categories being the most expensive: inflammatory conditions ($41), multiple sclerosis ($33), cancer ($25), HIV ($18), and growth deficiency ($7).

Dr. Tharaldson said the PMPY spend for specialty drugs is expected to reach $200 this year and could reach $300 by 2014. Meanwhile, the PMPY spend for traditional drugs is forecasted to remain consistent at approximately $800 through 2014.

As of September 2012, the FDA had approved the following 13 specialty drugs (8 were oral medications and 5 were for intravenous administration) this year: Inlyta^® (axitinib) for kidney cancer, Erivedge^™ (vismodegib) for skin cancer, Kalydeco^™ (ivacaftor) for cystic fibrosis, Korlym^™ (mifepristone) for Cushing’s syndrome, Omontys^® (peginesatide) for anemia, Elelyso^™ (taliglucerase alfa) for Gaucher disease, Perjeta^™ (pertuzumab) for breast cancer, Kyprolis^™ (carfilzomib) for multiple myeloma, Zaltrap^® (ziv-aflibercept) for colorectal cancer, Stribild^™ (elvitegravir, cobicistat, emtricitabine, tenofovir disoproxil fumarate ) for HIV, Xtandi^® (enzalutamide) for prostate cancer, Bosulif^® (bosutinib) for chronic myeloid leukemia, and Aubagio^® (teriflunomide) for multiple sclerosis.

Dr. Tharaldson also discussed the specialty drug pipeline for several classes. For inflammatory conditions, she said to expect more oral drugs in general, injectable biologics to treat rheumatoid arthritis, psoriasis, and inflammatory bowel disease, and possibly drugs to treat lupus and gout.

For multiple sclerosis, Dr. Tharaldson predicts there will be more oral disease-modifying drugs, expanded uses for drugs that are already approved, new therapies for secondary progressive disease, and an influx of generics and biosimilars.

There will also be more oral drugs for cancer, according to Dr. Tharaldson, as well as targeted therapies and vaccines. Other areas to watch include HIV (novel integrase inhibitors, single-tablet regimens, and therapeutic vaccines), hepatitis C (more oral protease inhibitors, novel interferons, and direct acting antivirals), and cystic fibrosis (drugs to increase mucous clearance, treat the underlying disease, and fight infections).