A few years ago we had two situations developing in parallel. Both related to an open label extension study following a pivotal registration study. In the first instance, the Sponsor believed that it was improper to deprive participants in the parent study the benefit of continued access to the drug in an open label extension study at the end of the parent study. In the second instance, for a Pediatric Investigational Plan (PIP), the Sponsor’s KOLs felt it was highly unethical to not allow an open label extension study.
We circle back to the first study. It was a double blind randomized (1:1) placebo controlled pivotal study. Patients who completed the pivotal study insisted on knowing their treatment assignment in the parent study. Their contention was that if they were randomized to placebo in the parent study, they would consider participating in the open label study.
However, if they were randomized to drug in the parent study, they would determine themselves if they benefited from the drug in the parent study before consenting to participate in the open label extension study. Now, as this was a study in a rare indication, the pace of enrolment was slow. The initial participants had completed the 12-month pivotal study, even as new participants were being enrolled into the study. To protect the pivotal study, it was important that we not disclose randomization until all patients had completed the study and the database had been unblinded.
So the Sponsor thought it was unethical to not allow participants the opportunity to be ‘rewarded’ with an open label extension study. The participants thought it was unethical of the Sponsor to offer them an open label study without disclosing to them the treatment assignment in the parent study. Needless to say, the Regulatory position prevailed – i.e treatment assignment could not be disclosed, leaving behind unhappy participants.
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