Personalised medicine is a “necessity not a luxury”, but there are numerous obstacles currently in place hindering true advancement in this area, industry has been told.
Speaking at last week’s joint BIA/MHRA Personalised Medicine Conference, Sir Gordon Duff, chairman of the UK’s Commission on Human Medicines said there was no clear plan or leadership when it came to personalised medicines and this was particularly seen within the clinical trials landscape.
“We tend to think of stratified medicine as new but stratification occurs on the market by trial and error – the patient says it doesn’t work so the doctor gives something else. But we need to understand how to do this at the development phase rather than in the clinic. We need to move upstream and stratify populations in clinical development with biomarkers. We need to stratify in Phase I and II and then on the market with a Phase III/IV type trial. As a result we’ll probably produce more and better drugs – that’s where we need to head.”
But in order for this to work, Sir Gordon said there were several things that needed to change, including a need for a more adaptive approach to clinical trials, the use of all available information, early authorisation in exchange for post-market commitments and a risk-based approach to regulation.
“Progress needs to be based on multiple convergent changes. We have progressed but not as a united front with all stakeholders. We need public involvement – an awareness and willingness to share data. We need healthcare systems – the NHS could be seen as one large clinical trial. In development, the model needs to change and there’s a need for collaboration. And regulators need to take on a facilitator role. We are at the beginning of a new era in convergent healthcare.”
Dr Tom Lillie, international therapeutic area head, oncology, at Amgen, agreed, saying there was “great inequality” in the way healthcare was approaching personalised medicines.
“It’s very disappointing we haven’t made more progress but our current trial and regulatory framework is not set up for that,” he said.
He feared development of personalised medicines was currently quite risky with the emphasis being on “single agent activity” when a “combination approach was empirically desirable”. For example, different cancers have different genetic signatures, he said, and that points to combination therapy.
Furthermore, there were too many studies generated on retrospective analysis, which slowed down development, cost more and was unsustainable, he said.
There was general consensus that the current clinical trial framework did not fit with the development of genomic drugs and there was possibly the need for a “radical rethink” of how clinical trials should be done.
However, Dr Ian Hudson, director of licensing division of the Medicines and Healthcare products Regulatory Agency, said there was flexibility in the system. “Companies need to talk to regulators for advice on what trials might be needed. We know large studies are impossible for many orphan diseases. I think regulators are more flexible than we are given credit for.”
But Dr Eric Abadie, chairman of the European Medicines Agency’s committee for Medicinal Products for Human Use, acknowledged there were a lot of open questions with the answer ‘it depends’. For example, questions like, is retrospective biomarker identification adequate for regulation and are biomarker negative patients required in clinical trials? Likewise, Seren Phillips_from the National Institute for Health and Clinical Excellence, said there were still a lot of issues to be resolved.
