AJMC Review of Specialty Pharmaceuticals Finds New Drugs Prove Their Value

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Specialty pharmaceuticals are changing the lives of patients with cancer and chronic conditions, but their high cost and increased used has drawn the scrutiny of payers. A review in this month's issue of The American Journal of Managed Care, which examined studies involving therapies for rheumatoid arthritis, multiple sclerosis, and breast cancer, found that when these drugs are used with the right patients, the value for patients is high.

PLAINSBORO, N.J. — Specialty pharmaceuticals occupy a growing share of pharmaceutical treatment, and could account for more than half of all pharmaceutical sales by 2017. But are these expensive drugs worth the cost?

A review article in this month’s issue of The American Journal of Managed Care, which examined studies involving drugs for that account for most of the spending in this area, found that specialty therapies can make a huge different in patients’ lives, and can be cost-effective over the long term if matched with the right patients.

Led by Martin Zalesak, MD, PhD, the authors examined studies involving 10 drugs for rheumatoid arthritis, eight for multiple schlerosis, and 10 for breast cancer. The authors defined “specialty pharmaceuticals” as those therapies that cost more than $600 per month, required close monitoring, which could include frequent dose adjustments; and required special handling such as temperature control or limits on where the dose could be administered. The authors present calculations for cost-effectiveness in each disease area, based on assigning well-accepted values to each year of improved quality of life.

“Although these new therapies are more expensive than older therapies, the fact that their cost-effectiveness results can be favorable indicates that these costs must be viewed in light of the patient benefits,” the authors conclude. “It is important, however, to note that achieving convincing benefits and cost-effectiveness is not always straightforward, but can instead require identifying the most effective agents and the most appropriate patients.”


The review found that patient benefits and cost-effectiveness were most clear cut in rheumatoid arthritis, a disease for which experts say early treatment and use of disease-modifying antirheumatic drugs (DMARDS) makes remission possible for most patients. This avoids disabling joint damage and other costly side effects.

For multiple schlerosis, the review found cost-effectiveness would have been enhanced if patients who did not respond had discontinued therapy. In the area of breast cancer, the review found especially strong evidence of cost-effectiveness in studies involving trastuzumab.

Increased scrutiny from payers is due not only to the cost of individual drugs, but also to the ever-growing list of diseases that may involve specialty pharmaceuticals, the authors note. “Although specialty biopharmaceuticals have historically been associated with rare medical conditions, they are increasingly being developed and used for the treatment of a number of chronic conditions,” they write.

About the Journal

The American Journal of Managed Care, now in its 20th year of publication, is the leading peer-reviewed journal dedicated to issues in managed care. In December 2013, AJMC launched The American Journal of Accountable Care, which publishes research and commentary devoted to understanding changes to the healthcare system due to the 2010 Affordable Care Act. AJMC’s news publications, the Evidence-Based series, bring together stakeholder views from payers, providers, policymakers and pharmaceutical leaders in the areas of oncology, diabetes management, respiratory care, and immunology and infectious disease.

CONTACT: Mary Caffrey (609) 716-7777 x 144