The scientific purpose of a phase 1 trial is to establish the dosage level that is toxic. Agents in a phase 1 trial are not dosed as they would be in clinical practice. A second complaint leveled at investigators concerns the fact that similar agents tested in previous trials had failed to markedly reduce pain Bichell, Again, these complaints belie a poor understanding of the drug development process.
Small differences in chemical structure can make a large difference in pharmaceutical outcomes, and scientists never know when a compound will radically change medical practice. The nature of research means that many drugs will not be successful, but a few will be.
A final complaint lodged against the trial investigators suggested that some neurotoxicity in animal studies should have forced scientists to re-evaluate their plan for a phase 1 trial.
But, even the scientist lodging this complaint admits that these injuries have happened with other experimental agents not this particular class of drugs , and those agents caused no problems in human subjects Bichell, Of note, the sponsor has made changes regarding how they are notified of subject hospitalization due to concern that additional volunteers received a high dose after the first man was hospitalized.
So, how does what happened last year in France factor into current promises to speed access to new drugs? By scientific standards, the phase 1 trial in question did not fail. It established the outer limits of toxicity. The outcry over what happened in France highlights the differences between how scientists think and how most patients and health care professionals think. Most drugs in the development pipeline will never gain FDA approval. Scientists are concerned with insuring safety and efficacy through a rigorous scientific process that takes time.
What is a promising drug today may be a complete flop tomorrow. Expanding access requires that we accept a different standard of evidence i. Accepting different standards may make a lot of sense, but it will not mean access without risk. Perhaps the most important step we should take is to test drugs in people who suffer from the disease in question and not healthy volunteers.
At least then, the burdens will be borne by those who stand to benefit. Yet, any of these solutions will not change the fact that medical progress is not a straight line, and we are putting people at risk today for the sake of tomorrow. She is currently completing her medical school clerkships and plans to graduate in May Join the discussion!
Your comments and responses to this commentary are welcomed. The author will respond to all comments made by Thursday, March 9, With your participation, we hope to create discussions rich with insights from diverse perspectives. Thanks for writing this! I learned a lot about the 3 phases in clinical trials. I think the answer is complicated.
On the one hand, competent people often decide to engage in risky activities and have an obvious right to do so. On the other hand, there are limits to the choices we allow people to make. For instance, no one can sell himself into slavery. It is also interesting that healthy volunteers for phase 1 trials tend to be those who need the money or are disadvantaged.
This recognition is more social than financial but would hopefully lead to better support for volunteers. At the same time, particularly dangerous agents or protocols should in my view be limited to sick individuals.
You must provide your name and email address to leave a comment. Your email address will not be made public. References Bichell, R. Brennan, Z. Regulatory Affairs Professionals Society.
David Oakley was the first to receive the drug and at 3. His face was just round like a ball and his stomach was huge. It was pretty scary to see somebody you love so disfigured. Ryan Wilson spent four months in hospital and had his toes and parts of his feet and fingers amputated after battling the symptoms of pneumonia, septicaemia and dry gangrene. Your body is a temple. Something could have been tampered with, sabotaged, poisoned and that these folk might have been the victims of such foul play.
Parexel, the company which ran the clinic where the drug trial was carried out, was found to have acted within its protocols in an interim review by the Medicines and Healthcare products Regulatory Agency. A final report however stated the trial had not properly considered the safe dosage of the drug for humans. It is understood that some volunteers have received confidential compensation payments, but still suffer from weakened immune systems and other side effects, and will never know the true legacy of the drug which so nearly cost them their lives.
Main Menu U. Climate Crisis. Follow us. Ryan Wilson spent four months in hospital BBC. The thenyear-old had all of his toes and parts of his fingers amputated after the trial BBC.
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