Study Assesses Specialty Pharmaceuticals Value


Specialty therapies for rheumatoid arthritis, multiple sclerosis, and breast cancer tended to have positive functional and clinical outcomes for patients.

Specialty therapies for rheumatoid arthritis, multiple sclerosis, and breast cancer tended to have positive functional and clinical outcomes for patients.

For patients with rheumatoid arthritis, multiple sclerosis, or breast cancer, specialty therapies tend to have positive clinical and functional outcomes that can make them worth the cost, the results of a study suggest.

The study appeared in the June 2014 edition of The American Journal of Managed Care.

“Our review of the 3 largest specialty pharmaceutical categories—RA, MS, and BC—suggest that these novel therapies hold great promise for arresting disease progression and improving quality of life,” the researchers wrote. “These findings raise the possibility that specialty pharmaceuticals for other disease areas might likewise confer substantial patient benefit.”

Researchers evaluated published studies on specialty pharmaceuticals in rheumatoid arthritis, multiple sclerosis, and breast cancer, and compared the treatment efficacy of specialty medicines to the previously available, non-specialty standards of care. They also analyzed cost-effectiveness data for specialty pharmaceuticals.

Researchers excluded HIV therapies from the analysis, because there were no non-specialty therapies for the disease.

The analysis included 10 specialty pharmaceuticals for rheumatoid arthritis, 8 specialty treatments for multiple sclerosis, and 10 specialty treatments for breast cancer.

The analysis of rheumatoid arthritis studies showed substantial and statistical improvement in clinical and functional outcomes compared to disease-modifying drugs. The cost-effectiveness analysis found a ratio of $47,500 per quality-adjusted life year, which was more favorable than the $100,000 per quality-adjusted life year that is generally accepted.

Although studies tended to support the clinical efficacy of multiple sclerosis therapies, functional findings were less conclusive, researchers noted. In addition, the cost-effectiveness for multiple sclerosis therapies also tended to exceed the $100,000 per quality-adjusted life year threshold.

“We expect that the cost-effectiveness of specialty pharmaceutical treatments for MS might also be improved by monitoring patients and discontinuing treatments among nonresponders,” the researchers wrote. “Such a strategy could protect most of the population’s clinical and functional gains while decreasing aggregate costs.”

Despite favorable results on functional metrics for breast cancer therapy, most studies of specialty medications yielded statistically inconclusive results. Data on breast cancer treatment cost-effectiveness was limited as well, but tended to show favorable results, researchers said.

According to the authors, the study results show the patient benefits associated with specialty pharmaceutical use. They further concluded that cost-effectiveness should be viewed in light of therapy benefits.

“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 researchers wrote. “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 most appropriate patients.”

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