Developing New Drugs for Cancer Patients

¶ … Cancer Drugs

For the past several decades, the news about cancer in the United States has been increasingly positive and that trend has continued unabated over the past decade. According to the National Cancer Institute at the National Institutes of Health, the overall incidence rate for men has declined by an average of 0.6% every year between 2004 and 2008, while for women, the incidence rate has dropped 0.5% per year from 1998 through 2006 and leveling off from 2006 to 2008 (National Cancer Institute). Just as importantly death rates from cancer have declined since 1975 and have sharply decreased between 19944 and 2008 for both men and women as shown in the graph below from the National Cancer Institute (National Cancer Institute).

There are myriad reasons for this decline in cancer deaths, including better education regarding healthy living, improved diets, and better treatment. Among the treatment options are improved drugs, which have come a long way towards extending life and enhancing quality of life in patients in the past few decades. For this reason, it is important to continue the push for new cancer drugs and to ensure that, once a new drug is found to be effective, it can quickly be given to the patients most in need of treatment.

Research and Development of Cancer Drugs

The process by which a drug goes from the earliest testing and design phase to being available to cancer patients can be a time-consuming and expensive undertaking. The process begins with a sponsor, usually a drug company, making the decision that a particular drug is worth pursuing based on the overall interest in the cancer targeted by the drug, the preclinical data in both cell lines and animal models, and the attractiveness of its properties (Chabner & Longo, 2010, p.29). Also, many companies may determine whether or not their investment is worth the risk, based on demand for the drug. If the drug cannot return the cost of development and a profit, many companies will drop the research at that point.

Once these hurdles have been cleared, the company must then coordinate with the U.S. Food and Drug Administration, the National Cancer Institute, and other academic investigators to set up clinical studies that need to be done before the drug is approved for use (Chabner & Longo, 2010, p.30). Phase 0 is considered pre-clinical research and involves laboratory testing performed by the company and testing done on animals. Many drugs fail after this point because drugs that show promise in animals often behave much differently in humans (Chabner & Longo, 2010, p.30). A drug that makes it beyond phase 0 must then move on to phase 1 and phase 2 clinical trials. An estimated 25% of cancer agents that enter phase 1 trials will not pass to phase 2 (Chabner & Longo, 2010, p.30). These are the two most costly phases and many drugs entering this stage of development will never gain approval for consumer use, but will still end up exacting a heavy toll on the drug company's bottom line. Drugs that get this far and don't work out for a drug company are especially costly since they will return no profit at all, even though the company has invested many resources and funds.

A drug that shows significant anticipated activity through the first two phases is then passed to phase 3, typically the most arduous in the entire process. It is a difficult decision for a company to make regarding pursuing phase 3 testing, since a failure at this point could have devastating consequences for both the company and the patients exposed to the drug at this point (Chabner & Longo, 2010, p.35). A diagram provided by the FDA illustrates the process below:

Accelerated Approval

The FDA has had such stringent evaluation methods in place to ensure that the drug supply in the United States is the safest in the world, but it is also very costly and many companies argue that they have abandoned promising drugs because they were not confident of gaining approval. To combat this problem, the FDA implemented the Accelerated Approval regulations of 1992 in the hopes of expediting the approval process. Under this process, the FDA can approve drugs for the treatment of serious or life threatening illnesses before they are subjected to the rigorous testing required for regular approval (Chabner & Longo, 2010, p.41). In these cases, the FDA grants approval while obtaining an agreement for the company to conduct confirmatory post-approval clinical trials in a timely manner. Should the drug then fail to confirm results, the drug can be pulled from the market (Chabner & Longo, 2010, p.41).

This acceleration can greatly reduce the time and money spent on development of drugs, up to four years in some cases (Chabner & Longo, 2010, p.41). But not all companies perform adequate follow-up testing and the concern from many in the healthcare profession is that these drugs are not as safe as regularly approved drugs are before they are available to the public and could, therefore, do more harm than good for the patient. But a more recent study has found that these drugs approvals are happening less than they had previously and that their efficacy rate was similar to regularly approved drugs. However, due to the fact that the FDA has encouraged only those who have shown some positive results in phase 3 trials to seek accelerated approval, the development times are no longer accelerated, averaging just over seven years for both types of approvals (Richey, et al., 2009, p. 4403).

Benefits to Patients

For a diagnosed cancer patient, the promise of a drug that could reduce symptoms, shrink tumors, or even add to life expectancy is akin to a miracle in pill form. These patients have typically been overwhelmed by news about their odds of survival or remission and have sought any treatment which they believe may prolong life. This often includes alternative remedies that are not officially recognized or approved by the FDA. For these people, who in many cases are down to their last chance, the promise of new, potentially effective drugs is worth the risk, even if the FDA has not given its tacit approval.