Carolyn Vachani, MSN, RN, AOCN
Last Modified: May 18, 2002
"Miracle Cure for Cancer," "New Drug Gives Hope to Cancer Patients". Headlines like these make us cringe as health professionals, but are reported in the media on a daily basis, playing on the desperation of many cancer patients. Often times, patients do not hear that a drug or test is still in early development, or the bad outcomes that were experienced during clinical trials. This has drawn attention in recent years to the process of Food and Drug Administration new drug approval and expanded access use of promising agents. A panel discussion held Saturday afternoon at the American Society of Clinical Oncology?s Annual Meeting set out to explain the process and the issues surrounding it. The panel was moderated by Stephen Carter, MD, of the Northern California Oncology Group, and included Richard Pazdur, Director of Oncology New Drug Approval for the Food and Drug Administration, Peter Eisenberg, MD, Community Oncologist, and Ellen Stovall, National Coalition for Cancer Survivorship.
Promising new agents are defined as medications that have demonstrated clinical efficacy in a particular cancer. Both Industry and the FDA use "objective response" and "time to response" as a measure of efficacy in new agents. This efficacy can be met through anecdotal evidence, early clinical trial results, and even extensive trial evidence that otherwise does not meet the FDA?s approval (as in the case of oxaliplatin). Medications that meet these criteria often gain public attention, and an outcry for expedient approval by the FDA. There are two paths a company can consider to get these medications to patients in need; expanded access programs and accelerated approval.
Expanded access programs are put in place to help patients receive a medication, currently in a clinical trial that they are not eligible for. Often times, this medication is undergoing FDA review, which can take as long as 6 months. In order to be considered for expanded access, the therapy must treat a serious or life-threatening illness, for which no other treatments are available. The drug must be involved in ongoing clinical trials, and by offering expanded access, accrual to these trials should not suffer. In addition, the company must be pursuing FDA approval "with due diligence". If these requirements are met, the FDA may allow temporary distribution of the agent, to patients meeting company-specified criteria. Despite being treated off study, data must be collected on these patients, most importantly, adverse events. Issues surrounding expanded access include: who absorbs the cost, how will this distribution affect the drug supply, the impact on clinical trial recruitment, and the fear of negative outcomes effecting future FDA approval. The panel expressed concern that over-use of expanded access will ultimately delay medications getting to market by hurting trial accrual, delaying the drug from reaching possibly thousands of patients.
Accelerated approval differs greatly from expanded access in that it is an FDA approval that is not temporary. These medications are given approval based on positive results of a marker that is considered predictive of outcomes. The disease being treated has few other therapeutic options available to patients. Once a medication is given accelerated approval, there must be subsequent clinical trials to prove efficacy and investigate adverse events. Unfortunately, not all medications can take one of these paths to availability, and there may be many months between the release of trial results and the time a medication comes to market. This has lead to increased pressure from the media and public to allow more expanded access and faster medication approvals. This too, has drawbacks.
Dr. Richard Pazdur, of the FDA, agrees that patients need access to promising therapies, but should have the knowledge and information necessary to make an informed decision. Dr. Peter Eisenberg commented that one way to expedite clinical trial accrual and approval may be to expand investigator credentials to more community physicians. Dr. Eisenberg shared that a majority of cancer patients are being treated in the community and these patients should also have access to clinical trials.
The Cancer Leadership Council (CLC), comprised of advocacy groups and healthcare workers, is a national group dedicated to addressing issues of importance in cancer care. The CLC attempted to form a consensus statement on access to promising new agents, but was unsuccessful. "Where you stand depends on where you sit," stated panelist Ellen Stovall, herself a 30 year cancer survivor and CLC member. As a patient, you want fair, unrestricted access to safe therapies that could save your life. As an industry representative, you must worry about company interests, and as the FDA, there is a duty to protect patients. Dr. Pazdur summed up the feelings of the panelists, stating that there must be a balance between the expectations of patients and society and the right of this vulnerable population to be protected against abuse.
The panel agreed that the issues of access to new therapies and media focus on these therapies require attention from the oncology community. Great strides have been made through the accelerated and expanded access programs, but this topic is one that is sure to be discussed at future ASCO meetings.
Oncolink's ASCO Coverage made possible by an unrestricted Educational Grant from Bristol-Myers Squibb Oncology.
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