Public Engagement and Clinical Trials New Models and Disruptive Technologies: Workshop Summary (2012) / Chapter Skim
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2 Framing the Problem
2 Framing the Problem
Pages 5-16
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From page 5... ...
Growth in funding and the ongoing rapid pace of scientific discov ery have led to a major increase in the number of clinical studies, with 1 This section of the summary is based on presentations by Deborah Ascheim, Associate Professor, Department of Health Evidence and Policy, Mount Sinai School of Medicine; Annetine Gelijns, Co-Chair, Department of Health Evidence and Policy, Mount Sinai School of Medicine; and Juan Lertora, Director, Clinical Pharmacology, NIH Clinical Center. 2 Explanatory randomized trials generally measure the effects of a treatment (its efficacy)
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Barriers to Patient Recruitment Clinical trials experience significant obstacles in patient recruitment. BOX 2-1a Elements for Successful Clinical Trials Well-defined clinical trial goals and target patient population • Realistic patient accrual strategy • Community outreach and education, building trust • Community/patients advisory boards • Well-informed community physicians and other health care professionals • Effective patient retention strategy • Protocol implementation support team • a Material presented by Juan Lertora, Director, Clinical Pharmacology, NIH Clinical Center.
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/American Heart Association (AHA) guidelines found that a large proportion of recommendations in current cardiovascular guidelines were based on expert opinion or case studies, and not evidence from random ized clinical trials comparing therapies (Tricoci et al., 2009)
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Some of the reasons individuals do not pursue clinical investigation in the academic medical setting include • conomic disincentives such as inadequate reimbursement for trial e activities and reduced time available to provide patient care; • ogistical disincentives that derive from a lack of infrastructure and l support; and • ultural disincentives, which manifest themselves in the decisions c of tenure and promotion committees that often have a bias against clinical trials activity as a prestigious or beneficial academic pursuit. Lack of Community Physician Involvement There is also a lack of engagement on the part of community physicians.
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The analysis of trial results generally relies on statistical measures of "central tendency" -- averages -- and their strength, as measured by standard deviations and significance tests. An unavoidable weakness of this approach, Juan Lertora, Director, Clinical Pharmacology, NIH Clinical Center, commented, is that its generalizability is limited because the "realworld" patient population is heterogeneous, so results of a treatment are likely to be more scattered than in a study population of demographically and clinically similar individuals.
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NIH Resources for Extramural and Intramural Researchers Lertora noted that NIH can share with outside researchers some resources it has developed to support its own intramural clinical trials. These resources can facilitate protocol development and trial imple mentation for researchers who need support beyond what their home institutions can offer.
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Clinical Trials and New Drug Development At the outset, clinical trials' importance in the regulatory process was underscored by panelist Leslie Ball, Acting Director of FDA's Office of Scientific Investigations. Clinical trials produce the data that support FDA determinations whether a drug should be approved, and they determine what information will appear on the product label.
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standard of health care is generally high, commented Richard Murray, Head of Global Center for Scientific Affairs, Merck & Co. It is increasingly difficult to identify patients to participate in studies who have not yet had some type of treatment for their condition and, because new treatments are likely to represent only incremental improvements, large numbers of patients are needed to achieve statistical validity.
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a Based on presentations by Carol Horowitz, Associate Professor, Department of Health Evidence and Policy, Mount Sinai School of Medicine; Robert Michler, Surgeon-in-Chief, Professor and Chairman, and Director, Center for Heart and Vascular Care at the Montefiore Medical Center/Albert Einstein College of Medicine; Richard Murray, Head of Global Center for Scientific Affairs, Merck & Co.; and Greg Simon, Senior Vice President, Patient Engagement, Pfizer Inc., and workshop discussions.
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monies to be used for this purpose.6 In response to the discussion of whether private or public payers should increase their support for the clinical trials enterprise, Bruce Vladeck, Senior Advisor, Nexera, Inc., described the need for strong, meaning ful trial standards to overcome the problem of trials of widely varying quality. He commented that criteria are needed to identify trials both of adequate scientific quality and sufficient importance, in terms of the pub lic's health, to warrant public support.
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To reach this population, the Cystic Fibrosis Foundation has worked with the medical community to establish more than 110 CF care centers nationwide, about 80 of which now can conduct clinical trials, said Joan Finnegan Brooks, President, Patient-Focused Market Research, and a person with CF. This network has successfully reached out and engaged the CF community to participate in clinical trials, resulting in several new therapies to manage the disease.7 Similarly, the Alzheimer's Association has promoted patient involvement in clinical studies (Alzheimer's disease affects some 5.4 million Americans)
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16 PUBLIC ENGAGEMENT AND CLINICAL TRIALS makes them willing to participate in a trial, even if they understand it will not help them, said Geiger. According to Simon, in an environment where scientists are many and trial participants are scarce, patient groups are starting to organize their own clinical trial networks and offer them to scientists, "because they realize their registries, their tissue banks, their biobanks, and their experience are the key resources."
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