When clinical trials end, the participants typically stop taking the study drug until it receives approval from their country’s regulatory agency. But if patients are benefitting from the drug and have no other treatment options without it, they are sometimes allowed to continue taking the drug as part of an open-label extension (OLE). People are only allowed to participate in an OLE if they’ve completed the initial portion of the study.
OLEs often follow phase 3 trials, the last phase before regulatory approval. But it is not uncommon for them to follow phase 1 or phase 2 trials for ALS/MND treatments. Many organizations are strongly advocating for the inclusion of some form of OLE in all industry-supported clinical trials.
OLEs are an act of compassion for people with serious illnesses like ALS/MND who participate in clinical trials. ALS/MND is not only severe, but clinical trial participants are making a sacrifice by willing to volunteer knowing there is a chance they may end up on placebo. In these cases, everyone should have access to an OLE that could maximize the potential benefit of the treatment, should it ultimately prove effective.
Scientific Gain
Besides benefitting trial participants directly, a well-designed OLE can also provide useful data about the therapy that could benefit more people. OLEs often extend a trial for months, allowing researchers to collect more information on the long-term efficacy, tolerability, and safety of the drug. However, the data’s validity is limited by the fact that participants know they are receiving the treatment. This could create a placebo effect that makes the drug’s benefit appear greater only because participants believe it should work.