Is it Time to Rethink Clinical Trials? FDA Thinks So.

Is it Time to Rethink Clinical Trials? FDA Thinks So.

A few weeks ago, I wrote about the need for patients to double check the Inclusion and Exclusion Criteria, when evaluating clinical trials.  Companies and Sponsors who are conducting clinical trials, develop Inclusion and Exclusion Criteria that make clear who should be included in the clinical trials and who should not be included. This is done for a number of reasons.  One reason is to maximize the chance of success of the clinical trial.  If everyone were able to take part in the clinical trial, the study would need to increase in size for statistical reasons, and the cost of the study would increase.  These costs would need to be passed onto those who need the treatments, after approval, i.e. the patients.  No one wants to see the costs of medicines increase from where they are at this time.

Another reason for the Inclusion and Exclusion Criteria, is to ensure patients who would suffer harm from taking part in the clinical trial, due to concomitant illnesses, are not included in the clinical trial.

FDA would like to change the way drug developers approach the design and conduct of clinical trials in terms of inclusion and exclusion criteria   A new series of guidelines have been issued. These guidelines seek to encourage drug developers to redesign their clinical trials to include patients with organ (liver, kidney, heart) dysfunction, HIV/Hepatitis B, Hepatitis C, brain metastases, and concomitant malignancies. These are all conditions that would normally be automatically considered grounds for exclusion from most clinical trials.  It will be interesting to see the impact of this guideline.

One of the concerns of these blanket Exclusions is that they make the recruitment of patients into clinical trials more challenging. The argument is that if these blanket exclusions are removed, more patients would be able to take part in clinical trials.  This is a tight rope to walk.  Patients who might potentially benefit from a treatment can still receive the treatment on a compassionate use basis, if this is appropriate.  For instance, including patients with HIV infection in clinical trials not intended to evaluate the treatment of HIV infection would be challenging because healthcare professionals may not be trained for the management of these patients.  It will be interesting to see how these new guidelines are adopted by companies and sponsors.

What are your thoughts on these new guidelines?


Lorna Speid, Ph.D.

President, Putting Rare Diseases Patients First!(R)