Researcher: Patients with Mesothelioma, Other Rare Cancers Would Benefit from Faster and Smaller Clinical Trials

Marie-Cecile Le Deley at a conference in France

A radical shift in the way clinical trials are conducted may be the key to getting new drugs to market, according to one researcher. This concept may especially provide a positive outlook for patients of rare diseases and cancers.

Marie-Cecile Le Deley, M.D., Associate Professor at the Institut Gustave-Roussy, Villejuif, France, told an organization of professional oncologists during a conference that performing smaller, more frequent clinical trials will help get drugs in the hands of patients sooner, especially those drugs for rare cancers like mesothelioma.

Le Deley explained to her colleagues at the 2011 European Multidisciplinary Cancer Congress that her research has shown a benefit to reducing current clinical requirements that mandate a need for large test samples and definitive evidence over the course of many years.

Le Deley is proposes to reduce the number of people that participate in a clinical trial, to relax the evidential criteria that requires trials to achieve a certain statistical success and to speed up the time of the trial process.

Patients who have rare and fatal cancers may have a prognosis that brings as little as a few months of life expectancy. Because some clinical trials require years to complete, that duration does not help these kinds of patients.

Mesothelioma is caused by exposure to asbestos. There are only 2,000 to 3,000 cases diagnosed a year in the United States.

Current clinical trial regulations demand long testing periods to ensure more accuracy of results and also to provide more safety of a drug or treatment.

For researchers focused on rare cancers, testing new potential treatments is also complicated by the high cost of researching over a long test period. Both are associated with trials for individual treatment options.

“We found that there were important gains in survival attributable to a strategy of conducting more trials with smaller sample sizes and relaxed evidential criteria compared with those required under traditional trial designs,” Le Deley said.

So what are the risks of this new approach? As with any experimental procedure with small test populations, faster test periods and less stringent evidence criteria, results may be less definitive.

However, because tests could be conducted more frequently, conclusive results could be reached quicker than traditional clinical trials by running many of these smaller tests at the same time.

For mesothelioma patients and other people diagnosed with rare, life-threatening diseases, smaller and faster clinical trials may offer an opportunity for them to get access to effective drugs faster than before. For pharmaceutical companies, less-populated and quicker clinical trials can reduce drug development costs because of less resources and personnel that will be required.

Because of the economics, large pharmaceutical and drug companies often do not invest in the research and development of drugs that cure ailments of smaller populations. If a cancer is rare, the potential market of people who would buy or use the drug may not yield enough revenue to make the the R&D process profitable for the company.

In 1983, to help ensure that patients with rare diseases would not be ignored, the U.S. government passed the Orphan Drug Act, which gives drug companies incentives to develop drugs for small markets of individual or rare disorders. Those incentives include federal tax credits, guaranteed seven-year monopoly rights and waivers on drug approval application fees and annual FDA fees.

Le Deley’s argument comes in the wake of a push by a rare-disease organization to ramp up awareness to the shortfalls of the current clinical trial process.

“There are still more than 6,000 rare diseases with no approved medications at all. These diseases affect as many as 30 million Americans. Most of these diseases are chronic, life-threatening or lifespan-reducing,” Peter Saltonstall wrote in a June letter to the Journal of the American Medical Association. Saltonstall is president and CEO of the National Organization for Rare Disorders.

Because clinical trials remain as the largest step in the drug development process, changes in policy and regulation of these research studies may determine the outcome of how fast drugs reach the people who need them.

“We hope that regulators will take this into account and re-examine their procedures in the interests of getting new, effective treatments to selected groups of patients as quickly as possible,” Le Deley said.


Mark Hall joined the Mesothelioma Center as a writer in 2011. Prior to joining the content team, Mark graduated from the University of Florida and then spent several years writing about business, entrepreneurship and technology for various online publications.

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