Systems and Regulatory Issues
Guest Expert John Marshall, MD: How do we engage the 97%?
Please welcome this month’s AccrualNet Guest Expert, Dr. John Marshall. Dr. Marshall is a professor of oncology at Georgetown University’s Lombardi Comprehensive Cancer Center (LCCC), holds the title of Chief, Division of Oncology/Hematology, Georgetown University Hospital; and Director, Ruesch Center for the Cure of GI Cancers, Georgetown LCCC. Dr Marshall specializes in the care of patients with GI malignancies and is recognized globally as a leader in the clinical research and development of new agents and approaches for gastrointestinal cancers. Personalized therapy is central to Dr Marshall’s philosophy for cancer and he hopes to implement it as a standard-of-care. With a purpose to reform healthcare policy and decide where our value in medicine resides, Dr Marshall proposes that we need a new approval process for novel therapies, one that incorporates magnitude of benefit and value. He wants all clinical researchers to think of patients as their partners; he believes that researchers need to understand that without patients, the oncology world will not progress in its quest for the cure.
Enjoy John’s comments below and consider posing your questions to this well-qualified expert!
John Marshall’s Post:
For a few years now, I have recognized the need for scientific forums for both research and lay audiences, focused on state-of the-art cancer care and advocacy, as well as general cancer medicine-related discussions. In December 2012, the Ruesch Center for the Cure of GI Cancers hosted just such a meeting; its annual symposium, “Fighting a Smarter War against Cancer: Engaging the 97%―Empowering Patients through Clinical Research.” The symposium addresses two related issues - can we improve clinical trial accrual from the current average of 3% of the cancer patient population to, say, 25%, and what would be the impact on clinical research?
Participants in the symposium represented key stakeholders―the federal government (NCI), industry sponsors, payers, healthcare providers, clinical research organizations, Georgetown University policy makers, and, often forgotten, patients. Sessions were moderated by noted authors and leaders in the clinical research community. The symposium conclusions--
We need improved clinical trial education and communication tools for patients. The most widely referenced public source for clinical trial information, clinicaltrials.gov, was found to be confusing, difficult to navigate, and replete with medical language, rendering it of little use to patients. Consent forms were considered even more problematic, mostly striking fear in patients, requiring their physicians and research teams to “interpret” the study risks for each patient.
We recommend developing a standard incentive model for clinical research participation. Medical providers and institutions spend uncompensated time, effort and resources on research. Physicians are currently incentivized based on consumption of healthcare, but incentives should be based on the primary metric of patient outcome and clinical research activity. Ideas for physician compensation include relative value unit (RVU) credits and “Level 6” classification for patient-care visits related to clinical research. Success in clinical research should be rewarded, not just for individual cancer centers but for rapidly growing and merging healthcare systems.
We suggest support and recognition for patients. Although participation in clinical research comes with additional time commitments and risk, we provide patients and their families with little additional support. Trial budgets should include financial supports to assist participants with expenses such as transportation, childcare, and lost wages associated with trial participation. We believe this extra expense would be offset by increased speed of accrual. Healthcare systems should develop patient partnering strategies to help address the increased needs of the patients participating in trials. And finally, we should develop a national recognition strategy such as creating a clinical research participant month and letting our “soldiers” in our war on cancer board planes first, along with our military.
Clinical trials should be designed with a greater treatment benefit for patients, incorporating personalized approaches whenever possible. It is clear that accrual improves when there is the potential for “substantial therapeutic improvement”―language recently adopted by the FDA to define a drug candidate for “breakthrough” designation. Clinical trials of a new therapeutic intervention should be smaller, have the potential for substantial therapeutic improvement, have minimal control arms, be designed to enable faster accrual, have faster reporting, and be designed to facilitate faster drug approval. Tissue acquisition and related reimbursement must also be addressed. While costs of radiologic scans are covered by insurers, repeat tissue acquisition is not. Standardized biopsy protocols have been developed and validated, using time-sensitive and homogeneous procedures. Using these protocols, tissue analysis will be critical to our success, making revisions to this aspect of our healthcare system essential.
We must revise our data sharing processes and laws. Over 60% of all oncology practices currently use electronic medical records (EMRs). However, this information is stored in silos all over the country, in different formats, with little consistency or quality controls, and maybe most importantly, with HIPAA preventing data sharing. We need to use this information to find connections among match patient diagnoses, treatments, outcomes and personalized genetic information.
We propose master protocols similar to that used in the I-SPY 2 clinical trial in breast cancer. In these trials, the master or parent protocol supports several smaller, enriched, personalized sub-protocols. This allows patient tumor tissue to be analyzed for several key molecular pathways and, based on the tumor’s individual protein expression, patients are assigned to a personalized therapy matching their individual molecular profile. In order to develop such master protocols, the following changes are recommended in our clinical research infrastructure and culture: centralized institutional review board clearance for these protocols (a process which is gaining momentum around the US); centralized/standardized research pharmacies; centralized/standardized medical staff training; centralized/standardized/simplified data collection, not only for those patients on trials but also for patients who opt for the standard of care as this group could serve as a contemporary control arm.
In conclusion, the United States must re-structure and unite its cancer clinical research systems to take advantage of the scientific discovery progress. Value and patient treatment outcomes should become the primary metrics of healthcare success, while the metric of healthcare consumption (the more tests and treatments, the more revenue) should be removed from the equation. All stakeholders share the common goal of finding a cure for increasingly common, often fatal cancers and we should fight a smarter war against cancer as a team.