What is the future of the orphan drug industry?

In Advocacy, Clinical Development by Tim Peplow

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Over the past few weeks, through a series of eBooks, we have been highlighting some of the most researched rare diseases. Now we wait with baited breath as to which competing orphan drugs reach the market first – those for Duchenne Muscular Dystrophy immediately spring to mind.

But while we’ve been looking into current rare disease research, we’ve also been asking ourselves some questions about the future of the industry. Which of the rare diseases will the next wave of orphan drugs treat? How will increased competition impact pricing and reimbursement levels in cases where one rare disease has multiple treatment options?

Take part in the poll below – where will the next wave of orphan drugs come from?

And there are more questions than that. By the time the next wave of orphan drugs are granted safety and efficacy approval, will there be a patient market as we know it today? Might today’s successes end up in long term failure to treat all rare diseases? Indeed, the rare disease industry, whilst growing at an enviable rate, has only just found its feet as a viable business model. Could it be said that the successes of Alexion and alike may never be emulated, as payers’ budgets become tighter under the economic pressure? As it stands, is there room on the market for more premium priced orphan drugs?

Perhaps you might think these fears are unfounded. Perhaps there will be a long and illustrious future ahead for all rare disease researchers and orphan drug developers. After all, with an estimated 7000 rare diseases, and an enthusiastic and influential patient advocacy body backing each and every one of them, surely good science in disease areas of completely unmet medical need will be good for all?

Take part in the poll below – where will the next wave of orphan drugs come from?

The orphan drug sector is no longer the emerging and uncharted opportunity for drug developers that it once was. It’s an established business arena that has witnessed the growth of billion dollar biotech companies created from one solely marketed orphan drug. The industry must now ask itself where the next opportunities lie and which rare diseases demand the greatest attention. Having naturally targeted rare diseases with well characterised natural histories, developers must now endeavour to differentiate themselves and go even rarer into the disease spectrum. The long-sighted sustainable approach needs to embrace the remaining 7000 rare diseases. It’s for this reason that we’re asking for your thoughts. Where will the next wave of orphan drugs come from? Which of the rare diseases will they treat?

Below is a poll (it might take a little while to load, our apologies) to which we have added 15 rare diseases that we think could be part of the next wave. The disease might either already have an orphan medicinal product in early stages of development, the disease pathogenesis might be well known, or there might be a strong patient advocacy body campaigning for the cause. Whatever your reasoning, vote for a disease already on the list or add a rare disease “new idea” of your own (if you are having problems adding your own idea, you can leave it as a comment and we will add it for you):

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The World Orphan Drug Congress Europe brings together pioneers from the orphan drug community, including key opinion leaders from the payer, HTA, public health and patient advocacy bodies. Find out more about the World Orphan Drug Congress Europe 2013, 14-15 November 2013, Geneva.