There is some debate in medical circles about the use of OLE...

There is some debate in medical circles about the use of OLE Open Label Extension studies. Some Pharma companies are using them as a marketing tool.

However in our case I believe that the Ethics Committee should see fit to grant approval. It would be cruel and unusual punishment to suddenly discontinue use of a medication which is not yet approved but has been demonstrated in a clinical trial to be safe and potentially showing benefit to the patient.

If all 12 patients accept the offer to continue with the OLE then I think it is suggestive that they are observing a positive benefit.

From the NIH National Library of Medicine :

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC1200598/


Compassionate use

When a promising new drug is being tested for a serious problem, doctors and patients naturally want the drug to be available outside the period of the randomised controlled trial. Delay is inevitable between the completion of the trial and the granting of a product license, and participants who do well on the study drug will want to continue to take it. Research ethics committees often feel obliged to approve extension studies as the only practicable way to provide continued beneficial treatment.

However, prescribing a drug on compassionate grounds is not research, and research ethics are therefore not a valid structure through which to provide a treatment, however beneficial. Members of research ethics committees will have had training and experience in assessing the ethical acceptability of a research protocol, but they will not necessarily have the expertise to make ethical judgments about clinical decisions involving the long term use of novel treatments.

This may be the role of the institutional review board in some countries, but it is not the role of the UK research ethics committee system. Apart from their lack of relevant expertise, the research ethics committee is an independent body with no authority over clinical practice and no responsibility for decisions about practice.At the end of a participant's involvement in a phase III study a decision will need to be made about their continuing treatment.

The new drug may have been of appreciable benefit to the patient, and a mechanism is needed for the patient to receive the drug even if it is not yet licensed, especially if there are few other treatment options. However, this is not the role of an open label extension study, and research should not be used to mask the limitations of current regulations and procedures in drug development. Research ethics committees should not be expected to connive in such practices, no matter how laudable the intention.

Prescribing for compassionate use is possible on a named patient basis, and much has been published on the prescription of unlicensed and “off label” drugs. If researchers and clinicians believe there are grounds for the compassionate use of an as yet unlicensed product, drug manufactures should be prepared to supply the drug to the doctors of patients who have been shown to benefit. The costs would, arguably, be no greater than those for an open label study. Safety data would still be available from these patients through the usual monitoring systems.