Approvals and Access Highlight the Year Ahead in Specialty

Following a year in which nearly 40 specialty drugs were approved by the FDA, 2019 also is expected to be huge in the specialty market, with more therapies for cancer, multiple sclerosis, and orphan conditions on the horizon. The question remains as to how the US health care system will absorb the high cost of these new specialty drugs to ensure access to the patients who so desperately need them. In 2017, specialty medications accounted for 46% of total pharmaceutical spending in the United States, as specialty spend grew 9.3% while spending on traditional medicines dropped 4%, according to the IQVIA Institute’s report Medicine Use and Spending in the U.S.: A Review of 2017 and Outlook to 2022.

Spending on specialty drugs is poised to overtake traditional medications by 2020, driven by innovative new treatments such as gene therapy, which restores an individual’s abnormal or mutated gene to treat or cure a disease. Although these drugs are revolutionary, they come at an extremely high cost. In her latest specialty pipeline report on page 11, Aimee Tharaldson, PharmD, discusses Novartis’ AVXS-101 (Zolgensma), a DNA-based gene therapy that treats children with spinal muscular atrophy. Tharaldson notes that the treatment is projected to cost between $1 million and $3 million for the one-time intravenous infusion. With this and other lifesaving treatments on the horizon, new approaches to funding health care must be considered to improve the lives of patients in need.

As such, Dan Leonard, president and chief executive officer of the National Pharmaceutical Council, states in his latest article on page 26 that improved patient survival and well-being should drive spending decisions, with funding based on what provides the greatest benefit to patients. Following the release of the Trump administration’s blueprint to lower drug prices, it is clear that major changes loom across the health care space, with specialty pharmacy at the center of this shifting landscape. In his editorial on page 5, our editor-in-chief, Dan Steiber, PharmD, takes a look at some of the tactics the administration may employ to address drug pricing and encourage the growth of value-based care.

With 2019 promising to bring significant change to health care, it’s more important than ever to see how the latest legislative developments might affect specialty pharmacy. Staying on top of these trends is our mission in this journal and on our daily news website, SpecialtyPharmacyTimes.com. Follow us online, here in print, and in our e-newsletters to ensure that your pharmacy is ready to thrive in the year ahead.

Mike Hennessy, Sr

CHAIRMAN & CEO