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Delivering Value in Rare Diseases

Posted by | 10:56am on Monday, February 29, 2016 | No Comments
Hidden Value of Orphan Drugs

Debates about high drug prices have dominated many of the headlines this year.  Raise the price of a drug 5,000% and you will definitely get everyone’s attention, including the feds.

It’s unfortunate to see the occasional bad actors getting so much press, given the inspiring progress made by all of the companies and communities diligently working to find treatments, especially when you consider those with rare diseases. Thirty million people in America are impacted by rare disease and 95% of those disorders lack treatment. Don’t let the headlines mislead you. The real news is that continued investment in rare disease innovation helps everyone.

We hope you’ll share our 4th annual Rare Disease Infographic that focuses on these innovations:

  • Record levels of investment leading to record levels of FDA approvals
  • NIH and advocacy groups funding early research
  • Research on orphan drugs that opens the door to new treatments for common ailments
  • New distribution models that reduce costs through direct patient engagement and personal support
  • Foundations and compliance experts working to accelerate the approval process by improving research and public policy

As the number of orphan drug designations and approvals rises, there will likely be more questions raised about the cost.  The customary response is that research is more challenging in rare diseases or that knowledge gained from rare informs the common, both of which are true.

But as the emphasis on reducing costs in healthcare grows, perhaps the best response orphan drug makers can make is to demonstrate value. That isn’t just about costs.  It’s about patient experience, quality of care and maximizing outcomes.

Understanding how best to accomplish this requires a paradigm shift. Delivering value in orphan drugs requires a fundamentally different strategy, because rare patients and families face a whole host of financial, medical and lifestyle challenges that require a sophisticated system of support.

In the end, it will be the patients themselves that teach us what good looks like in rare disease. Whether it’s a matter of determining meaningful endpoints for clinical trials or designing a program to increase adherence, the best way to deliver value is to listen to them.


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