Orphan drug development suffers from high late stage attrition rates, a challenge stifling all of pharmaceutical innovation. Experience goes a long way in order to identify and avoid mistakes that can cause your orphan drug development to go belly up or be shelved just prior to market approval.
While there is no one-solution-fits-all answer to successful drug development, here are some key points to bear in mind and potential mistakes to avoid in order to achieve late stage success for your orphan product.
· Make sure you present clear endpoints for you trial.
· Patient exaggeration of benefit.
· Not discussing regulatory strategy with regulatory authorities early enough in the process
· Incorrectly assuming there is a lower bar for demonstrating efficacy in orphan diseases
· Preparing the various stakeholders well in advance
· Forget to make a strong sales partnering globally before drug approval
· Not assessing outside factors that might contribute to patients decline – how can you monitor his lifestyle, food or medication.
· No support from patient advocacy groups and not engaging the patient community.
· Not having patients involved from the get-go & driving the study
· Switching or widening label to early
If you think the list needs up dating and there are glaring mistakes that you feel need to be avoided, we welcome your comments below.
In the meantime, take a look at recommended strategies to put in place to avoid late stage attrition rates during orphan drug development