Recasting the Mold
The historical reticence of pharma firms to pursue orphan drugs seems to have originated with conflicting molecular domains. The pharmaceutical industry evolved with a small molecule bias. Large molecule biologics, which make up the majority of orphan drugs, do not lend themselves to the industry’s molecular R&D comfort zone.
Moreover, big pharma’s small molecule therapeutics tend to target diseases with sizable patient populations. The large commercial infrastructures that drug companies have established to address these markets provide them significant competitive barriers. The small, concentrated patient populations that define orphan drug markets do not provide similar operating leverage. Thus, as long as new drug opportunities existed in markets that fit the established market mold, big pharma seemed willing to pass up orphan drug opportunities despite the financial attractiveness.
A deeper understanding of the molecular basis for disease will likely make the billion-dollar pill obsolete. Advances in genetic profiling and biomarker discovery may bring a greater appreciation that broad disease categories more likely reflect an aggregation of numerous, albeit slight, genetic variants. Accordingly, future therapeutics may be developed to target specific variants.
While personalized medicine may not extend to the individual, a single remedy for the masses seems destined to become more “mass-less”. Another perspective might postulate that, as currently defined, today’s orphan indication may be much more commonplace in the future.