The complexity of many orphan diseases in terms of treatment options (or lack of), their chronic manifestation and the management of symptoms, make them among the most challenging diseases to manage. A growing number of potential orphan disease therapies, are being developed as biologics requiring injection as the dominant route of administration.
Getting the drug on the market and into the hands of the patient as quickly as possible is a key driver for pharmaceutical companies, and this is extremely important for such orphan diseases where there is often such a lack of therapies. Unfortunately, thinking about the way such therapies are to be delivered is often not a dominant part of the total disease solution.
Underestimating the importance of instilling confidence and well-being in such patient populations is crucial to long term compliance with treatment. The healthcare world is shifting towards payment for outcomes and the orphan disease area is unlikely to escape this.
Whitepaper provided by: Kate Hudson-Farmer, Senior Manager