BETHESDA, MD (June 1, 2012) – The Food and Drug Administration (FDA) has drafted a regulatory guidance describing a new  way of conducting breast cancer drug trials that promises to reduce substantially the time and cost of getting new treatments to patients.  The approach is based on a trial design being tested in the I-SPY 2 TRIAL, an innovative Phase II breast cancer trial being conducted under the auspices of the Biomarkers Consortium, a public-private partnership led by the Foundation for the National Institutes of Health (FNIH) that includes representatives from NIH, FDA, and multiple pharmaceutical companies and academic research centers.

Patients with early-stage breast cancer have typically had to wait for years to receive new cancer drugs, which are usually tested first in patients with later stage metastatic disease and approved for use in more curable early stage cancer only after additional randomized clinical trials. The draft guidance, which is described in the current issue of the New England Journal of Medicine, establishes a potential new pathway for accelerated approval of drugs tested prior to surgical removal of tumors in certain types of high-risk patients with localized, early-stage disease. FDA signaled it may now grant approval of new drugs that have shown clinical benefit, based on data from patients receiving this type of “neoadjuvant” treatment whose invasive cancer has disappeared by the time of surgery (“pathologic complete response”). I-SPY 2 results, together with an appropriately powered follow-on phase III study in the biomarker populations identified in I-SPY 2, could be sufficient for accelerated regulatory approval of an investigational agent under the new guidance.

“Better options for patients with high-risk breast cancer are urgently needed,’’ said Janet Woodcock, MD, Director of the Center for Drug Evaluation and Research at FDA. “The FDA guidance explains how a promising drug identified in trials such as I-SPY 2 could be evaluated for FDA approval, so patients could have rapid access if the drug proved better than current treatments.’’

The I-SPY 2 Trial, which is being led by Laura Esserman, MD, MBA at the University of California at San Francisco (UCSF) and Dr. Donald Berry, PhD at MD Anderson Cancer Center in Houston, uses specific genetic signatures – biomarkers – in the tumors of patients to select those who are most likely to benefit from testing using the new approaches.  The biomarkers are also incorporated into a unique “adaptive” trial design that allows researchers to measure the relative benefit of treating patients with different tumor profiles with a specific drug and guide treatment assignments for subsequent trial participants. I-SPY 2 can test new treatments with significantly fewer participants and in half the traditional time, which will dramatically lower costs under the new guidance.

The use of the I-SPY 2 design as a basis for accelerated drug approvals was first discussed in an article in the December issue of the Journal of the American Medical Association co-authored by Drs. Esserman and Woodcock.

“We are truly excited to see that the FDA is supportive of trials like I-SPY 2,’’ said Dr. Esserman, the co-principal investigator of I-SPY 2. “This really moves us much closer to getting the right drugs to the right patients now, and at a time when they can be cured.’’

The trial, which was launched two years ago, is screening multiple cancer drugs at 19 major cancer research centers across the country.  Scientists from the National Cancer Institute, FDA, pharmaceutical and biotechnology companies, as well as breast cancer patient advocates also contributed to the design of the trial, which is managed by FNIH and Quantum Leap Healthcare Collaborative with support from Quintiles, a global biopharmaceuticals services provider.  Funding for I-SPY 2 is provided by non-profit foundations including The Safeway Foundation, several pharmaceutical companies, and other private sector and philanthropic donors.

FDA is accepting public comment on the new recommendations through July.

To view a copy of the draft FDA Guidance, click on this link - http://www.quantumleaphealth.org/wp-content/uploads/2012/06/FDA-Guidance-draft-May-2012.pdf

For the full report, go to the February 2, 2012 issue of Clinical Cancer Research, http://clincancerres.aacrjournals.org/content/18/3/638.full

The Commentary starts by stating the problem with getting new drugs to market today – “The development of new drugs is becoming increasingly expensive—and oncology drugs, in particular, have a high clinical failure rate. The current return on capital investment in drug development by US public companies was recently reported as less than 0.3%.  The low probability of success, coupled with rapidly accelerating expenses, means that drug development is increasingly the purview of only 2 organization types: a few large companies and myriad small, venture capital–funded start-up firms. At an estimated cost of $1.0 billion to $1.8 billion for developing a successful new drug, funding for such risky ventures, particularly for oncology drugs, may diminish.”

The methodology behind the I-SPY 2 Trial is cited in this Commentary as one important example of how to dramatically speed up the process.

To read the full Commentary, click here:  JAMA article (Dec 2011).full

Watch the video on NBC’s website:

http://video.msnbc.msn.com/nightly-news/45730121/#45730121

UCSF’s Carol Franc Buck Breast Care Center is one of the recipients of the Safeway Breast Cancer Awareness campaign funds.  “We are so grateful to Safeway for its leadership and vision, and to the company’s customers for every dollar they donate,” said Laura Esserman, M.D., Professor of Surgery and Radiology, and Director of the Carol Franc Buck Breast Care Center at the University of California, San Francisco. “Safeway is providing the critical resources to build systems to revolutionize breast cancer care.”

Read the full article here:

http://www.marketwatch.com/story/safeway-raises-145-million-for-breast-cancer-2011-11-08

Watch Potential Breakthroughs in Breast Cancer Research on PBS.

ISPY 2 is one of three breakthrough breast cancer clinical trials highlighted in this video.

BioCentury This Week opens an important dialog on “Regulatory Innovation” in its Sunday, Nov. 28 broadcast, devoting the entire program to a conversation with Dr. Mark McClellan, Director of the Engelberg Center for Health Care Reform at the Brookings Institution, and the only person to head both FDA and Medicare.  Dr. McClellan discusses obstacles in the current clinical trials process, and the resulting obstacles for drugs reaching patients in a timely and cost-effective manner.

Dr. McClellan specifically highlights the I-SPY2 Clinical Trial as a leading example of the type of regulatory reform necessary to move the industry forward.

The program can be viewed Here

The design of the I-SPY 2 trial represents a major advance in breast cancer research with promising implications for other cancers and other diseases. Quintiles will make its grant in support of I-SPY 2 through QuantumLeap Healthcare Collaborative, a 501(c)(3) non-profit foundation and a co-project manager of the trial.

• Click Here to read The full Press Release

]]> http://www.ispy2.org/archives/why-black-plates-had-to-go/feed 0