‘Orphan drugs’ are ascendant. What will that mean for people with rare diseases?

For most drugs, the path from the Petri dish to pharmacy shelves is paved with uncertainty. It’s a decade-long journey, with billions in investment dollars at stake, seemingly countless administrative hoops to jump through, and the risk of complete failure lurking at every bend. Ultimately, studies show that only a meager 10 percent of drugs in development — about 30 per year — make it to the finish line: approval by the US Food and Drug Administration (FDA).

Current trends in therapeutic development are pointing to a metamorphosis in the industry. Fierce competition between companies, economic factors, policy changes, and patent expirations on existing bestsellers are nudging pharmaceutical enterprises toward a focus on a new niche market: orphan drugs, which treat diseases effecting fewer than 200,000 Americans.

Orphan pharmaceuticals have historically been left on the back burner in research and development, owing to their limited profit potential. But in the next five years, the orphan drug market is set to be worth $209 billion (an increase double that of the overall prescription drug market growth) and to represent over 20 percent of all prescription drug sales. Last year, more than a third of drugs approved by the FDA had an orphan status.

A whopping $2.5 billion is required to bring a prescription drug to market

The ascent of orphan drugs as a significant portion of the drug market is the result of decades of activism. A whopping $2.5 billion is required to bring a prescription drug to market. To maximize returns, it has historically made sense to aim at the largest patient groups, with vaccines and cancer therapeutics being obvious choices. In the late 1970s, however, shifting attitudes and escalating public pressure prompted major legislative changes in a bid to prevent patients with rare conditions from being marginalized. Families and supporters of those fighting these illnesses rallied together in a coalition called the National Organization for Rare Disorders (NORD) which, along with other advocate groups, spurred legislative action to fast-track the creation of drugs, vaccines, and diagnostic agents for rare diseases.