Ann: Percheron launches post-trial access program, page-43

Time is muscle and time is both precious and limited so no one wants to stay on a course that is leading to no where

What is the post trial access program? We know that it refers to the provision of an investigational treatment to clinical trial participants following trial completion, but beyond that surprisingly its not entirely black and white. In fact, it appears that the regulations for continuing to treat a patient post trial are unclear or non-existent. Consequently we have recourse in the Percheron announcement, to the Declaration of Helsinki. The declaration provides an international ethical framework that addresses the need for country governments to make provision for post trial access for all participants who still need an intervention identified as beneficial in the trial.

The paper by Doval et al provides a little more colour and connects post trial access to compassionate grounds. The investigational drug should offer significant benefit, where termination of the treatment would lead to deterioration in the patient's overall condition.

It appears, however, that the sponsors of the investigational drug must decipher how to apply the principles within the Declaration of Helsinki. According to one of the world's leading healthcare companies, consideration for post trial access will depend upon the patient's medical need contingent upon meeting certain conditions. My cherrypicked list include;

• the patient must have a serious or life threatening illness or condition
• the request must be made via the trial investigator
• the investigational drug must have resulted in a clinical benefit to the patient based upon a benefit / risk analysis by the investigator
• the investigator has determined that post trial access is the best medical option for the patient.

So, back to the interim data to be released in December that could result in an application for accelerated approval. Percheron management make the point - quite rightly - that they don't know what the data looks like because as we know its a double blind placebo controlled trial. Maybe, post trial access requests happen all the time and one shouldn't read too much into the requests being made by the trial investigators. Alternatively, maybe shareholders have been given a remarkable insight into the efficacy of avicursen that the Principal Investigator commented may have a valuable role to play in the treatment of DMD.