Welcome to the era of the orphan drug

Authors: Mark Smith, Senior Medical Manager and Duncan Brearley, Associate Medical Writer

At the time of writing, it has been roughly two years since the outbreak of the COVID-19 pandemic, when we were first locked down with limited capacity to work, move around, and socialise. Thankfully, owing to the considerable research efforts of the global scientific community, vaccines have been rolled-out and the promise of normality is on the horizon. But now just imagine what might have happened if these research efforts had not taken place.

How would you be feeling if your lifestyle remained highly restricted and your prospects for the future were still unclear? This is life for many of the >300 million individuals who suffer from a rare disease.

There are currently over 7000 known rare diseases, but fewer than 1 in 10 people with a rare disease receive targeted treatment¹. Furthermore, it takes an average of 6–8 years for a person with a rare disease to receive a correct diagnosis². Diagnostic delay, in conjunction with an overall lack of efficacious therapies, often results in a significant psychosocial and financial burden for affected individuals and their caregivers.

However, the passing of the Orphan Drug Act by the FDA in 1983 and the reciprocal Orphan Drug Regulation by the European Union in 1999 has helped to incentivise drug development for rare diseases that, under normal circumstances, would be commercially non-viable.

Fast-forward around 30 years and we have entered a new era – an era of the orphan drug...

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