Significant Benefits Await those Willing to Embrace Adaptive Methodologies



Pharma IQ
03/21/2011

Since the European Medicines Agency and the United States Food and Drug Administration (FDA) clarified their positions on adaptive clinical trials, their use has become more widely accepted within the pharmaceutical industry.

There are still of course barriers which exist. Some continue to question the integrity of the findings from trials where the results are unblinded and are subject to a quick analysis, while there is also still limited understanding about the trial design itself.

However, for those who overcome these hurdles, significant benefits await.

"By enabling better, earlier decisions, agile clinical development provides a far faster and less costly path to market, by as much as forty percent compared to traditional approaches.

"These gains in efficiency reduce investment risk and make it possible to treat currently underserved populations, representing a wealth of opportunity for those willing to embrace adaptive methodologies," Dr Michael Rosenberg, president and chief executive officer of Health Decisions, explained.

Adaptive designs in oncology clinical trials

One area in which adaptive clinical trials are seen to hold particular benefits is in the field oncology.

The Battlecancer study, which was hailed as providing a significant breakthrough in the use of biomarkers, used an adaptive design. Following the analysis of tumour biopsies from the 225 patients enrolled in the trial, researchers at the Anderson Cancer Centre randomly assigned participants therapies.

Results were then analysed after eight weeks to assess how the therapies were working, switching to what they believed to be a more appropriate therapy if improvements were not being seen.

Tyler Jacks, resident of the American Association of Cancer Research, told the Wall Street Journal: "This is the future. This is how drugs will be developed and clinical trials organized."

Pharmaceutical company Merck is also looking to use adaptive clinical trial designs within its Oncology Collaborative Trials Network, which will carry out 30 to 40 studies on investigational drug candidates throughout the year.

"Despite recent advances, there remains an urgent need for effective treatments for cancer, and the pace of traditional drug development often lags far behind the latest science," Malcolm Moore, from Princess Margaret Hospital and Ontario Cancer Institute, said.

Personalised medicine

Personalised medicine has taken off in a big way in recent years. Research conducted by the Tufts Centre for Drug Development found 94 percent of United States-based biopharmaceutical companies were investing in this area, with the majority of these experiencing positive results.

The Biomarkers Consortium, a public private partnership including the FDA, National Institutes of Health and a number of major pharmaceutical companies, last year launched what it called a "highly-anticipated" clinical trial, which combined both personalised medicine and adaptive design for the treatment of women with fast advancing breast cancer.

The I-SPY 2 trial involves taking biomarkers from tumours to identify which treatments were likely to be most effective, then using information collected from early trial participants to form decisions on how to treat later stage patients and remove ineffective therapies.

Anna D Barker, deputy director of the National Cancer Institute, and co-chair of The Biomarkers Consortium Cancer Steering Committee, said: "This will allow us to finally design advanced, smaller and less expensive Phase III trials that test the right drugs in the right patients."

However, while such studies hold a great amount of promise, there are still those with a note of caution.

Lesley Seymour of the clinical trials group of the National Cancer Institute of Canada in Ontario told the journal Nature these studies can take up to six months longer to plan and be hampered by a lack of access to qualified statisticians.

"They have been hyped by some people, but we're still trying to determine what potential these trials have," he said.

There are many though who see the further development of adaptive clinical trials as key to the growth within personalised medicines. For a pharmaceutical industry which no longer has the development of blockbuster drugs as an option, they can also boost the incentive to develop riskier therapies for small patient populations.