Ask yourself whose responsibility it is to create a strategy that will recruit patients. If you said “the investigator” you are wrong. It is the sponsor’s responsibility to create a strategy that will be successful, and if it is not, to readjust it and to apply necessary resources to insure it succeeds.
Open-label clinical trials often mislead a company as the data obtained have a much greater likelihood of being positive than if the same trial was done in a double blind manner. This may lead to a company wasting years of effort and millions of dollars until hey recognize that the drug really did not demonstrate efficacy in a true double blind study.
Insure that any compassionate plea protocols are in your company’s interests. This requires a balance of ethical responsibilities with practical issues. The practical issues include resources required, value of the data obtained and whether your program is being slowed and allowing competitors to catch up or move ahead of you.
Bert Spilker, PhD, MD is an independent consultant who was most recently the Senior Vice President of Scientific and Regulatory Affairs for PhRMA. He is the founder of Bert Spilker & Associates (BS&A).
