The U.S Food and Drug Administration (FDA) has created a new ‘breakthrough’ designation for cancer drugs, so that any that are deemed to be successful can effectively be fast-tracked through the trial process and brought to market quicker.
Many respected physicians in the field of cancer research backed the move, including Robert Schneider of New York University Cancer Institute. While that is understandable, the backing of pharmaceutical giants such as Pfizer has many people talking. Is public health at risk by giving new drugs early clearance so that such companies can cash in? That is the view of some analysts, although we wanted to look at the story behind these drugs before coming up with a definitive opinion.
Clinical Testing Background
Ordinarily, the process from initial research, to trials, to bringing a drug to market is typically around a decade, with a cost of around $1billion. However, the trend in recent years, as seen with new drugs such as Xalkori and Zelboraf, has been for such drugs to be approved in a fraction of the time. This has seen more cancer drugs being brought to market for a fraction of the previous cost. This is not just something that has happened for one or two new products. Schneider commented, “We’re going to see this as a sea change over the next five years.”
Even as scientists and medical professionals have come to understand the make-up of cancer and how it behaves in the body, it has remained a challenge to develop better treatments than those currently available. It is hoped that, as more drugs are developed and cleared for medical use quicker, agonizing treatment options such as chemotherapy will become much less necessary.
Naturally, there is a fear among the public that a shorter period from research to market means a lack of understanding of side effects and potential complications. While this is recognized by the industry, they believe that the immediate benefits of saving someone’s life outweigh any risks. Addressing this, Mace Rothenberg of Pfizer said, “The accelerated development of new drugs can be a double-edged sword. As you move more quickly some questions may be unanswered.”
One of the key drivers of such change has been the way in which trials take place. Ordinarily, the purpose of a ‘Phase 1’ human trial is to test the dosage of a drug that the body could safely consume. Now, such trials determine how effective such drugs are from day one. How is this done? Simple. Cancer patients are the clinical testing group.
Among all of the issues that quicker availability of treatments will bring, this is one of the most controversial.
However, ask yourself the question:
If you have endured chemotherapy, one or more operations, and tried various other treatments, and death was staring you in the face, would you not give it a shot? Would you not want a friend or family member to try, if it could save them?
It is on this basis that many new drugs are tested. However, it is not a case of consultants across America simply giving a new treatment to all cancer patients. The growth of knowledge and information means that we can identify which drugs have the potential to work better with certain cancers. The chances of terminal cancer patients getting a drug that works actually increases massively, while, longer-term, any ineffective treatments will be identified quicker, and therefore the money and time dedicated to them withdrawn. From a patient and their families’ perspective, it also means they are likely to avoid being administered treatments that do not work.
Speaking about the possibilities such a trial process brings to the world of medicine, Dr. Michael Davies, of MD Anderson Cancer Center, Houston, commented, “You can see positive signals much more quickly, and clinically you can spare patients for whom the drug is not likely to work.”
This new attitude towards cancer treatment is potentially leading us towards an era of ‘personalized medicine.’ Clearly, there is not going to be a cancer drug developed for everyone, however as medical databases become further populated with evidence that shows a particular drug works in patients with a particular gene mutation, for example, doctors will be able to get to the correct course of treatment quicker than ever, crucial to the survival chances of cancer patients.
Cancer drugs will now be developed to target specific areas of tumors, and as they are a much more focused treatment the time to get these approved will fall dramatically. Xalkori, for example, went from research to market in a little over four years, and is now widely used as a treatment for lung cancer. The process for Zelboraf took a similar amount of time.
In terms of the drugs themselves, the unanswered question is that relating to side effects. There will be, naturally, a lower level of understanding when it comes to new drugs, especially if certain stages of the trial process are bypassed entirely. With such treatments, it must be appreciated that it could take many years to build up a full picture of side effects, although to back up Pfizer’s perspective, surely the act of saving a life renders a shortfall of knowledge in this area irrelevant.
The Financial Question
Cynicism and suspicion will always exist around pharmaceutical companies. None of the major providers being unwilling to specify how much money is saved with these new processes has helped this. It is easy to think that, with the potential for millions of cancer patients’ lives to be saved, that such an issue is not important, yet it is central to everything we have discussed.
Should we be concerned with how much money Pfizer or Johnson & Johnson save or make in the coming years? Not directly, no. We should be concerned with how those figures will affect the cost of drugs made available to hospitals and cancer patients, however. The FDA does not factor finances into its decision making process, which means pharmaceutical companies could simply grow their profits, if they choose to do so.
If savings in the trial process translates into these new drugs becoming available to millions during the ‘sea change’ of the next five years, as Schneider referred to it, then we will have truly breached a new frontier of cancer treatment.
Should it go towards the bottom line, however, there is a real danger cancer treatment could become a glorified social engineering exercise.
This opportunity to change cancer treatment forever is one that should not be missed.