The drug was still experimental, but clinical trials suggested it could be a lifesaver for patients with a lethal form of blood cancer called multiple myeloma.
And those patients were clamoring to get it. They overwhelmed drug maker Janssen Pharmaceuticals with requests for the medication.
Most companies don’t know how to handle such requests. Often, it’s the richest patients, or the best connected, or those who run the most compelling social media campaigns who end up getting the drug. Everyone else is out of luck.
A better solution would be to change the way the FDA approves drugs. Instead of the Go/No-Go method they currently use why not implement the rolling approval process that has been suggested for well over 2 decades?
With rolling approvals the PharmaCos are allowed to put the drug in the field under very tight yet available restrictions and the doctors who are prescribing the drugs must consistently provide data to the FDA and the drug company and any deviation from either expected outcome or compliance is a trigger to stop allowing new admissions. By using the rolling process the better the results the more are allowed to join the testing process. In addition instead of a deadline of approval which could shift the balance of stock and profits the drug simply slides into the society and becomes integrated into the formulary as it gains success and acceptance. Instead of selected high profile (read EXPENSIVE) university only type facilities each licensed doctor who has a patient in need can participate with the rolling process if he/she the patient and the PharmaCo agrees. In terms of costs it is FAR less costly to give some drugs to patients then it is to pay a university team a few million dollars to supervise “a clinical trial”
The so – called ” Compassionate ” Use system for pre – approval access to drugs in this country is broken . The situation in Europe ( Bigger Gov.t Bureaucracy) and around the world is even worse. My wife is a Stage IV breast cancer survivor. Which is normally a death sentence , but new drugs are changing the situation . However the System is not keeping up . My wife is alive today because of a new 21st century drug , Kadcyla. We could not get compassionate use in 2010. We could not wait for the Slow FDA and the up-tight , Profit Driven Pharma industry in 2010. The FDA shockingly denied accelerated approval for the drug and it was not available in Boston . So we went to Wash. , DC 16 times at our own expense to get the drug . Her cancer disappeared by 2011. We stopped the drug in 2014. She is cancer – free.
We applaud both Janssen and the NYU team for starting to change the System . The FDA is trying to get quicker – we need more pharma co.’s to follow Janssens’ lead . This drug is just one of many , every yr. , that still take 7 – 10 yrs to get through the System . Even then they often have conditional approval from the FDA limiting access for many yrs. after . We pt. activists do have some differences with Dr. Caplan and his NYU team – he gives the FDA a free pass, and does not factor in the FDA’s bureaucratic slowness ; their Regulatory Power contributing to co.’s reluctance to grant compassionate use. His words about the ” the rich ( not factually proven ) and ” connected ” , pt.’s who are ” savvy with social media ” tend to cast blame on the pts., the victims of this broken system . We do appreciate his initiative. But this is just a drop in the bucket . Much more reform needs to come .
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