FDA and Researchers Move Us Closer to New Clinical Model(s)

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

Comments

[Bruce Lehr]

Thanks for your comments Pharma Conduct Guy (Eric). Sometimes, thinks like extended release add real value and maybe even combo formulations at times (probably more germane to vaccines though) but many times innovation is used a shield for exclusivity without much patient benefit. Along those lines, I've enjoyed observing the back and forth that is going on in India now between foreign MNCs and local Indian companies with regard to their patent rules - which are effectively pushing a company to show data of improved efficacy vs previous therapy to gain a patent extension (see several posts on this blog) I think that would make for better progress and make R&D stretch more in their thinking.

[Pharma Conduct Guy]

Although many companies will claim that regulation stifles innovation, I guess that depends on how you define innovation.

If you take it to mean coming up with clever ways to game the patent system in order to increase exclusivity time and charge 10x over other available therapies, then I guess the companies could have a point.

Some examples of ways that companies extend their patents include 1) taking two separate drugs and combining them as a combination therapy (e.g. Pfizer's Caduet), 2) reformulating an active ingredient as an extended release version (many drugs), 3) studying pediatric indications for "adult drugs" (think anticholesterol or sexual disfunction), or 4) preparing an isomerically pure variant of a racemic blockbuster that is losing patent protection (e.g. AstraZeneca's Prilosec vs Nexium).

By requiring companies to show a 'significant' improvement either in safety or efficacy, drug companies will have more incentive to reallocate marketing budgets for RD. Perhaps innovation will increase.