Orphan drugs : future viability of current forecasting models, in light of impending changes to influential market factors

Abstract

Interviews were conducted to establish a baseline for how orphan drug forecasting is currently undertaken by financial market and industry analysts with the intention of understanding the variables typically accounted for in such a model. A literature search formed the basis of subsequent interviews conducted with experts from industry, payers, providers, legislators, patient groups, and the FDA. Discussion then focused on elements of the market which are poised to change in the short-term, how such changes might be reflected in existing models, and/or how these models may instead need to be modified to adapt to the new environment. We hypothesized that impending changes in the healthcare sector would indeed impact the legitimacy of current forecasting models, and that significant changes would need to be introduced to account for these new market forces. Our hypothesis, however, was not confirmed, in that although much of the literature and, indeed, public outcry over rising healthcare costs in general and drug prices in particular make a strong case for implementing changes in the orphan market via payers, government, or other actors, an assessment of healthcare experts regarding market changes over the next five years revealed a general consensus that meaningful change will likely not occur during this timeframe for orphan products, with the exception of a possible increase in pharmacoeconomic requirements for drugs which are only marginally effective. Thus, current orphan drug forecasting models constructed for use by financial and industry analysts correctly avoid discounting for these potential changes, as they will likely not face significant changes in the US until closer to a ten year time horizon. Potential exceptions to this conclusion depend on implementation and regulatory treatment of the fields of personalized medicine and gene therapy, as developments in these areas may closely interact with existing orphan drug legislation. Our results have significant implications for all companies and stakeholders entering or currently operating in the orphan market, and open the door for further quantitative and qualitative analysis.