Rare and Orphan Disease Challenges… And How Interim Can Help

Road sign that says "Challenges Ahead" and shows the silhouette of a businessman about to jump over a hurdle.

Road sign that says "Challenges Ahead" and shows the silhouette of a businessman about to jump over a hurdle.Jordan WarshafskyBy Jordan Warshafsky, Partner, Ashton Tweed

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In 2015, the FDA awarded over $19 million in research grants to boost the development of products to treat rare diseases. Recently, there have been many similar pushes to provide treatments for patients with little or no treatment options. However, working with orphan diseases presents a new set of challenges in the life science industries.


Unfortunately, developing treatments for diseases with a small patient population cannot offset the cost of R&D, clinical trials, and manufacturing – making these therapies difficult to price. Commercialization for these products also proves to be an unprecedented challenge in the pharmaceutical world. Lastly, the diagnosis of rare diseases is difficult in and of itself, causing additional strain to companies hoping to provide therapies for these diseases.


In such an already challenging field, companies can encounter a variety of unforeseen obstacles including leadership changes, regulatory speed bumps, and funding failures. Fortunately, interim executives are available to aid companies working on highly specific therapies. Many rare disease companies utilized interim leadership in 2015 to safely get their companies through a transition or emergency.


It’s impossible to have full-time experts of every field on staff. But this hiring strategy takes that impossibility one step closer to reality. Often your company needs more than just a consultant’s advice, it needs experts at your disposal who actually execute solutions. This is where interim talent can solve your problems.


Your rare disease company can hire highly experienced executives on a short-term basis to help you through each step of the process to bring your product from discovery through commercialization. Your company gains all this without the stress of compensating an executive salary indefinitely. Think of it as borrowing a tool from a toolbox – right when you need it. This saves time and money for both your company and eventual patients receiving treatment.


This strategy is flexible and wide-ranging to fit your company’s needs. You can have an Interim Chief Financial Officer implement a financial strategy, an Interim Chief Commercial Officer work out a production method, an Interim Head of Clinical Affairs sort through clinical trial complications – expert leadership to use how and when you require it. For examples, see the case studies below:



Progress is underway for rare and orphan disease treatments and companies are overcoming obstacles like these worldwide. Interim talent is a proven tool that should be considered by companies hoping to address the unmet needs of patients around the world. It’s been a proven asset for life sciences companies large and small.


Looking for executive leadership with a narrow or specific background for your company? Take a look in our toolbox, or rather our Life Sciences Executive Talent Bank, by contacting Ashton Tweed today.


Share your insights! Contact jamesrudman@ashtontweed.com to contribute your life sciences article as a guest writer.


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