In a March 27 article in the New England Journal of Medicine, a team led by physicians at Massachusetts General Hospital revealed that a new cancer drug from Novartis has shown exciting clinical results in a small trial of lung cancer patients. While additional trials are necessary before the drug can obtain approval from the Food and Drug Administration, this type of success story demonstrates why research to develop new cancer therapies is critically important.
Researchers in academia, biotech and pharmaceutical companies are making remarkablediscoveries to help identify new drugs and drug targets for cancer patients. Many new compounds are under investigation — including those that inhibit the growth of cancer cells, block the blood supply to tumors and prevent tumors from evading the immune system.
Even as scientists seek to bring new cancer treatments to market, however, drug patent issues are holding back some researchers. A major hurdle is in combination drug trials that test two or more therapies at once. Pharmaceutical companies often shy away from trials that have great potential, because the drugs may not generate profits if they are used together with a generic drug or a drug patented by a different company.
Recently, there have been major advances in our understanding of how cancer progresses. As scientists have sequenced thousands of cancer genomes, patterns are starting to emerge. One clear insight we have gained is the likelihood that no single drug will be able to defeat cancer. The reason most cancers become drug resistant and come back is because their DNA mutates quickly. Cancer cells that are not killed by the drugs survive, continue to grow and replace the cells that have been wiped out.
So how can we beat the evolution of cancer cells? Most cancer researchers believe that the way to do it is to use the same approach that holds HIV in check for AIDS patients: with combinations of drugs. These drug cocktails, if used appropriately, may someday control many cancers because the cells resistant to one drug will be sensitive to another.
The great challenge is figuring out which drug combinations are likely to work the best. But when it comes time for clinical trials that are necessary to bring drug combinations to market, there are two major hurdles.
The first hurdle occurs when two drugs have been patented by two different pharmaceutical companies. When this is the case, quite often neither company wants to fund the trial. This reluctance is because only 8 percent of new drugs obtain FDA approval, clinical trials can take many years to complete, and there can be legal conflicts regarding ownership and publication of results — resulting in the fear of litigation from the other company. There can also be squabbles over how to divide expenses and profits, with one or both parties looking for a bigger cut of the potential financial gains, and both putting profits before patients.
The second obstacle can arise when there is no patent protection, and thus no financial incentive to test the cocktail and market it if it works. There is only a financial incentive to move forward if all patents are active and held by a single company, and with drug combinations, this is often not the case.
For example, a 2013 study showed that patients who take a generic diabetes drug called metformin have a much lower risk of dying from prostate cancer. However, since the patent for metformin expired in 2003, no drug company has been interested in funding studies of combination therapies that include metformin because there will not be a new, patented drug at the end of the study.
Currently, there are at least 38 clinical trials testing metformin in therapeutic combination studies, but not a single one of them is sponsored by a drug company. Instead, cancer centers, hospitals and universities are searching for support from the government and foundations to finance the trials on their own.
Meanwhile, drug companies are looking for ways to modify metformin, so that they can patent those versions and test them. Those modified versions may or may not perform better than metformin, or have fewer side effects. But they will definitely be more expensive for patients. Development and testing of these drugs will also take considerable time and money.
Of course, patents and intellectual property are essential to the process of bringing new treatments and drugs to patients. My point is not that we should eliminate financial incentives. Those incentives are necessary to pay for the huge cost of ensuring that drugs are both safe and effective for patients, and to fund the research behind new discoveries that will someday defeat cancer.
While pharmaceutical companies have a legal responsibility to their shareholders, they also have a moral responsibility to do their best to help patients’ lives — even if it may not be the most profitable course of action. The mission statements of drug companies usually have two goals: the second focuses on growing their business, but invariably the first goal is something like “to help people…live longer,” “to care and cure” or “to save and improve lives.”
Cancer patients do not have the luxury of waiting until legal battles can be settled, or derivatives of existing drugs can be patented. Cancer drug combination trials should go forward without delay, and drug companies should be in the forefront of making that happen. Doing so will not only save lives, but also generate the good will and favorable publicity that could improve the pharmaceutical industry’s poor public image.
It is time for drug companies to put helping patients first.