The growth of specialty medications, including orphan drugs, is a topic everywhere you turn; at conferences, in health journals and blogs, and in newspapers and on the news. Innovative and transformative medicines are coming to market in therapeutic areas where there was previously either no treatment or only minimally effective treatments. More medications for orphan or rare diseases, many of which are diseases in children, are being approved at a faster rate than ever before (15 orphan drug approvals in 2015 and 19 in 2016). These innovative medicines provide hope to patients and families living with an often devastating illness, that their previously untreatable disease may be managed in such a way that they can live a more normal and longer life, even if there isn’t a cure. Orphan medications can treat populations with as few as one hundred patients or less in the US, and may come with high price tag.
Payers and manufacturers are dealing with this dynamic in ways that are both similar and distinct. Payers manage with prior authorizations, requirements for medical necessity for medical benefit products and narrow specialty pharmacy networks, to ensure appropriate use and control costs. Manufacturers implement limited or exclusive specialty pharmacy networks and provide patient services (HUB, financial assistance where permitted) to try to provide an optimal patient experience.
I will be at the sPCMA conference Tuesday through Thursday this week in Orlando, FL and will be speaking on Thursday morning with Bill Roth, Founding Partner, Blue Fin Group, on the topic of Orphan and Rare Disease Products and Exclusive Pharmacy Networks. I look forward to the dialog around the opportunities and challenges these transformative medicines bring to the health care arena.