clinical research: safety & end points

[Guest guest]

During an interview on Schorr's " Patient Power "

website, Dr. M. Keating of M.D. made a specific call

for patients to advocate for govt. deregulation to allow for

quicker patient access to new drugs. Keating's call was

general in nature and I would like to see a more nuanced

discussion by doctor/researchers as to the pros and cons of

what sort of regulation is appropriate to preserve a climate

of safety yet speed up bringing promising drugs to market.

On the safety side of the equation, no one would want a

repeat of the Thalidomide scandal or the more recent UK

clinical trial where six healthy volunteers were seriously

damaged by cytokine storm from a monoclonal antibody.

(see link) http://classic.the-scientist.com/news/display/23275/

This tragedy also demands the examination of govt.

regulatory (MHRA) failures for prevention of a re-occurrence

of a similar event, proving that just because regulations

exist it does not mean guaranteed safety.

That said, we need to have a re-evaluation of the way in

which new drugs are compared to standard therapies. A good

example would be the class of Kinase Inhibitors (PCI-32765,

CAL-101 & AVL-292) which act in an entirely different way to

alleviate the cancer burden of patients from Standard Chemo

or Monoclonal Antibody therapies. The so called " End Points "

of drug efficacy measurement may need to be re-examined and

re-defined as well as the data gathering criteria (read CT-

scans) that are required to show efficacy leading to FDA

approval. We don't want Clinical Trial volunteers whose

epitaphs will read: " Cancer well controlled with good

quality of life for several years by new Kinase Inhibitor -

patient died of Acute Scan Syndrome (wouldn't that be a kick

in the " ASS " ).

Improvement in these two areas could speed up new drug

approval AND safety for Trial volunteers while achieving the

intended goal of getting new life saving drugs to the

broader patient population much faster than is the current

slow and bureaucratic train we are now forced to ride.

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