is a cardiologist based in Raleigh, North Carolina, and CEO of . In addition to his weekly video analyses on ѻý, he is the official medical expert at WNCN in Raleigh and makes frequent guest appearances on other national media outlets such as Fox News and HLN. The opinions expressed in this commentary are the author's.
The following transcript has been edited for clarity:
Last week in the Wall Street Journal, reporter to initiate a pilot program that gives dying cancer patients an opportunity to take experimental, unapproved drugs and therapies. While patients have been granted access to experimental drugs in the past, they are almost always part of a study or clinical trial -- this leaves many patients who do not live near a clinical trial site or meet specific enrollment criteria with few, if any, options. Compassionate use permission is sometimes granted but the drug maker has to provide permission first -- this can be a cumbersome and lengthy process -- and many patients just do not have that time left. Under the pilot, there is now a single call center and a single point of contact with a much shorter application process.
There are valid arguments on both sides of this issue and the way in which drugs are approved and made available to consumers. Here is what critics and proponents have to say about this issue of the longstanding drug approval process.
First, the pros:
1. The FDA is dedicated to making sure that patients are safe when it comes to new drugs through a rigorous review and approval process, and this review provides physicians and patients with a certain degree of confidence in a particular therapy.
2. Many drugs that are developed often have unseen and unknown side effects -- the FDA approval and surveillance process helps to identify these and alert both doctors and patients when appropriate.
3. The regulation of drug development by the FDA ensures that there is a standard process that all manufacturers must follow -- there are no short cuts.
Then, the cons:
1. The complicated process at the FDA can make it very difficult to get potentially life-saving drugs approved and in the hands of doctors to treat critically ill patients.
2. Many patients who have no options have died waiting for a potential new drug to be approved.
3. The lack of regulation of these drugs -- or a fast-tracked approval process -- may result in patient harm.
4. Pharmaceutical makers may take less regulation as an opportunity to take a short cut and put lives at risk.
Here is my take:
1. It's all about balance. We must balance patient safety with allowing for compassionate use of a new drug for patients with incurable diseases -- when a patient has no chance for recovery in the eyes of a treatment team, and there is a promising -- but not yet approved therapy out there -- I believe that we should allow them the opportunity for access. This must be accompanied by a thorough shared decision process involving patient, family, and physician. The FDA should make this process easy and less cumbersome. Time spent on the phone and filling out lengthy forms may result in "death while waiting."
2. The bigger issue at hand, beyond approval for compassionate use of cancer drugs, is that the FDA must streamline the approval process for all drugs and eliminate the redundancy in paperwork, and limit the expense to developers. As it stands now, many smaller companies cannot afford to go to the FDA with promising drugs until they obtain more funding or are acquired due to the enormous expense involved. This limits innovation.
3. There must be a better way to prioritize and categorize drugs for patients who are out of treatment options. These "high value" and high potential drugs must be identified earlier in the process and the FDA must be ready to allow their use in advance of a dying patient's request.
4. For patients with little or no hope, newly developed drugs can provide the hope that is needed to get them through the next week, the next day, or even the next hour. Let's cut down on the red tape and the bureaucratic delays and do just that -- give patients HOPE.