This week we highlight clinical holds on cell and gene therapies, clinical trials and patient experience, artificial intelligence and identifying bone fractures, and more. Each week we highlight five things you need to know about in the life sciences industry. Here’s the latest.
The Food and Drug Administration can place a trial on a clinical hold for any number of reasons including safety concerns, incorrect investigator brochures or other gaps in the Investigational New Drug application. Usually, these holds are short term and are resolved so the trial can proceed once the FDA’s concerns have been addressed. A recent uptick in holds point to one new trend: cell and gene therapies represent a higher percentage of clinical holds than the percentage of overall trials.
Beyond just safety and efficacy, there are nearly endless decisions that go into the design of a clinical trial. The FDA is considering new guidance that will formalize another set of considerations focused on risk-benefit analysis and understanding the patient’s experience with their disease and with the proposed treatment. This framework will help the FDA evaluate the candidate better in the context of the condition, other treatment options and how risks associated with the treatment will be managed.
The FDA this week approved software from the French company Gleamer that can automatically identify fractures in X-rays. The system reduces the volume of undetected fractures in X-rays by 30%, which promises better outcomes for patients and a reduction in misdiagnosis for one of the most common reasons people visit the emergency room.
The Federal Trade Commission recently failed to advance a review of the practices of pharmacy benefit managers (PBMs) but is expected to either adjust the proposal to get additional support, or to hold a new vote once a fifth member of the commission is approved by the Senate. PBMs have often been the target of complaints by the pharma industry but now independent pharmacies are joining the chorus and asking for change.
As companies are entering trials with promising new gene therapies, a nagging concern has been whether effects of these treatments will be durable. For one of Intellia’s Phase 1 trials, the data has shown that the effects of the treatment have lasted up to 12 months. This specific candidate is still in trials and there is likely a long road ahead before patients could benefit from it, but given the cost and complexity of gene therapies, this could be promising news.
Read our industry outlook for additional life sciences insights.