The FDA has gotten a lot of heat lately for all kinds of things, and perhaps 90% of what they are blamed for is not their fault or responsibility. When infrequent side effects show up in large post-marketing studies, or manufacturers adulterate a product leading to deaths, there is no FDA with as many people and as much money as you can provide that could stop this from happening. The Vioxx story was not the FDA’s problem, and the sad thing is really that Vioxx is a good drug but it just needed to have the right patient population. For the severe arthritic patient with bleeding ulcers and a good heart, the drug is very useful and beneficial. Merck with all the great work their scientists did getting there messed up the process in the end. Nothing the FDA would have done could have foreseen or stopped it, and it is naive to believe that lots of mindless random safety analyses will catch this kind of thing.
Similarly, inspecting Chinese manufacturers of heparin could never have discovered an intentional adulteration scheme in which a fake compound that could not be detected by usual means (part of the plan in choosing the compound) was added to reduce the cost. An inspection would not have reliably found this as few manufacturers would have said, “…and here is where we add the fake heparin material to our product.”
When someone consciously decides to cheat, they can beat inspections. Inspections are important yes, but cheaters cannot be fully stopped by FDA. Manufacturers have the legal responsibility to make product that is pure and unadulterated, and the FDA’s job is to make sure that the basic rules are being followed. Inevitably, there are people who will try to cheat, and they do get caught and are disbarred from the industry and subject to criminal prosecution.
Our goal for CURETHEPROCESS is to help point to and make the changes in organization and regulatory process needed to accelerate the effective development of rare disease treatments. This should not mean and does not mean that we don’t highly respect the very difficult job that an FDA reviewer has to do each day, despite the noise and rancor going on about them. Both developing drugs for treatments (the job of biotech and innovation) and reviewing them to assure the right products reach patients (the task of FDA) are truly noble endeavors as we take science discoveries and treat patients. What is nobler than that?
FDA’s responsibility is also enormous and the pressure high. The great challenge is that there is no perfect data or information on anything. There are always ambiguities and uncertainties and in the midst of these situations, the FDA has to make a call whether to risk something and treat, or risk nothing and subject the patient to their unmitigated disease. These are difficult choices, and rarely is the information available to know everything that may happen. For rare diseases, we just need to make sure there are enough of the right people in place, and that we give these people the right process tools on how to review rare disease drugs, and then let them do that most difficult step which is to approve (or not) the drugs as best they can.
That is the goal of the CURETHEPROCESS campaign.
But we also need to support the FDA for all the difficult work they do every day.
