Clinical Trial Design, Drug Development, and Policy Issues of Importance to Cancer Advocates Ellen L. Stovall National Coalition for Cancer Survivorship (NCCS) Institute of Medicine Workshop October 5, 2007
Patient Advocates and Clinical Research n Views about patient participation in clinical research are not universal n Clinical trial design is one factor but remains an unproven barrier n Many studies have been done to assess physician and patient attitudes to clinical trial participation
Physician & Patient Attitudes toward Clinical Trials n Lack of physician and patient awareness of open clinical trials and the unavailability of protocols to both the patient and physician at the time of consultation [Metz, et.al. 2005] n Lack of participation can be categorized generally as an absence of knowledge about clinical trials; unwillingness to be randomized; time constraints; distance from treatment center; insurance denial; and distrust of the medical establishment [Metz, et.al. 2005]
Attitudes of Advocates & the Public Toward Drug Development n The Unrealized Promise of Biotechnology l l l Gleevec example raised hope, but has not been easily replicated. Even successful follow-on drugs like Herceptin Tarceva, and Avastin are limited in their impact on patients. Cancer is typical example of personalized medicine high drug prices and high co-pays & present access challenges to patients who could benefit from them
Unrealized Promise of Biotechnology n The way our country has funded and conducted research is being challenged i.e. ongoing debate over NIH reorganization and importance of clinical trial participation (e.g., CMS/NCD reconsideration n Everyone government, academia, industry must recognize that our efforts have fallen short of what was promised to taxpayers and patients.
Threats to Future Progress: Excessive Avoidance of Risk n People with life-threatening diseases like cancer are less likely to be concerned about newly discovered safety issues than patients who take drugs for other conditions n No medical intervention is entirely free from risk, and pharmaceutical innovation may be deterred if decision makers focus too much on risk avoidance.
Threats to Future Progress: Discrepancy in How Advocates View Access to New Therapies n NCCS has always advocated maximum access for people with cancer, and believe that access to new therapies should be based on sound and reliable medical evidence. n Access issues differ among advocacy groups, e.g., Abigail Alliance/Washington Legal Foundation call for individual access to unapproved therapies by those who can pay for them as a Constitutional right.
Regulatory & Financing Issues n complexities which include past and current Medicare clinical trials coverage decisions; n the adherence to standards for granting accelerated approval; n the status of FDA s issuance of final guidance on expanded access programs n oversight regarding compliance with FDA policies regarding review of new cancer indications by the Oncologic Drugs Advisory Committee.
Potential Remedies n Prioritize and expand access to research protocols that transcend individual interests for the common good n Create an incentives and rewards-based practice environment for individual physicians to engage in clinical research n Advocates should work with FDA & clinical trials sponsors on their drug development programs and clinical trial designs to allow for expanded access to innovative cancer treatments
Potential Remedies n Developing a public policy approach that takes a longer view and is not swayed first to one extreme and then another by sensationalist media or political coverage n Forging new partnerships among government, academia, industry, providers and patients, based on more open sharing of information and honest assessment of relative strengths and weaknesses of various ongoing initiatives.