I read a couple of articles this evening that gave me hope. We're starting to see some innovative thinking in how to conduct clinical trials that really make sense to me. These new approaches to clinical trials seem long overdue and if adopted sooner rather than later makes me believe in a brighter future.
First in a recent public meeting supporting the process toward renewal of the Prescription Drug User Fee Act (PDUFA), William Vaughn, health policy adviser at Consumers Union, told the agency it is past time when companies "must test new drugs against current therapies, not placebo". In other words, we should begin testing drugs versus the current best in class to prove we have something better and not another "me-too". That would be a step forward for patients I believe. See Pharma Times story.
Much more exciting (and more immediate), researcher at MD Andersen are conducting the Battle lung cancer study. The study is looking a biomarkers from tumor biopsies of 255 patients in the study at defined short intervals during the study. The patient's disease is assessed to see if the drug regimen he/she is on is working or if the treatment needs to be changed. This is a new adaptive trial design that is expected to enhance the chance of success of drugs in mid- to late- stage trials. (See Fierce Biotech)
We all know about the number of drugs that have been failing to meet their clinical endpoints in phase II and III studies. This type of design would better allow clinicians to match drugs to the patient or patient class that would be respond to a particular therapy. It's another step closer to personalized medicine and could save drugs from failure by targetting them to the right set of patients.
Couple this with an approach where you actually evaluate the treatement versus the best options out there - and hey - we might have something.
Posted by Bruce Lehr April 22nd 2010