Characterizing and Communicating Uncertainty in the Assessment of Benefits and Risks of Pharmaceutical Products Workshop Summary (2014) / Chapter Skim
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5 Communicating Uncertainty
5 Communicating Uncertainty
Pages 43-60
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From page 43... ...
• Implementing standard procedures for developing and com municating regulatory decisions could improve the accuracy and impact of risk communication strategies. • Public information at the time of a drug's approval can be improved by clearly conveying benefits, harms, and uncertain ties and concisely highlighting what is known, what is still being studied, and what is unknown.
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From page 44... ...
A number of workshop participants indicated that communicating uncertainty is at the heart of many of the issues and challenges associated with benefit–risk assessments. OVERVIEW OF RISK COMMUNICATION1 The intent of effective risk communication is to help people make informed decisions; it does so by providing orderly, structured ways to understand the uncertainties inherent in their choices, explained Fischhoff.
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From page 45... ...
As the literature of risk communication demonstrates, Fischhoff noted, scientific rigor is needed to create accurate messages. Underlying any risk communication is an implicit decision tree representing the choice that the communicator seeks to inform (see Figure 5-1)
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From page 46... ...
Fischhoff noted that current support for risk communication efforts is largely haphazard and episodic. Establishing dedicated resource centers, akin to the statistics units (or "cores")
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From page 47... ...
• Treatment options - Medication, surgery, in-hospital treatments, cognitive therapy, "nutraceuticals," possible inclusion in clinical trial • Family decisions and family attitudes • Information sources - Peers, co-workers, friends - Internet (ranging from well informed, seemingly well The informed, and not even pretending to be well informed) - Support groups for various conditions, and these can be very well-organized, professionally facilitated support groups that have a particular intention in terms of informing about the choices - Loosely organized online discussion groups - Media coverage • Other factors - Insurance coverage, reimbursement, out-of-pocket costs, dis ability vs.
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From page 48... ...
This leads to what Woloshin called the standard misconception that "new is better." This misconception is reinforced in advertisements targeting physicians and consumers that promote a drug's newness as representing extra benefit, rather than extra uncertainty, noted Woloshin. In an attempt to offset this misconception, regulators in Europe and the United Kingdom require companies to include a "black triangle" warning next to the name of a new drug on all prescriber and consumer information, alongside the statement "This medicinal product is subject to additional monitoring."6 The intent is to alert the public that despite a rigorous approval process, the drug is "under probation," as Woloshin 5 This subsection is based on the presentation by Lisa M
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From page 49... ...
. Woloshin explained that while the concept of accelerated approval is very useful and important, information conveying that there is extra uncertainty due to the accelerated nature of the approval is often buried in the various communication tools deployed by FDA.
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Approximately half (48.9 percent) of the pivotal clinical trials for novel therapeutic agents approved by FDA between 2005 and 2012 included a surrogate outcome as the primary endpoint (Downing et al., 2014)
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From page 51... ...
Because of the fundamental uncertainty of surrogate outcomes, Schwartz said, it is important that patients understand the inherent uncertainty when drug approvals are based on surrogate outcomes. The same randomized trial mentioned above (Schwartz and Woloshin, 2011)
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From page 52... ...
Schwartz indicated that FDA could improve the consistency and impact of its communications by prominently featuring information about postmarketing requirements in press releases, information for prescribers, and labeling. Schwartz also noted the importance of including information about the direction of the uncertainty -- for example, if the uncertainty is not that the effect of a drug on an outcome is unknown, but that there is an open question about a potential harm and that FDA is requiring specific postmarketing studies to better understand the magnitude of the problem.
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From page 53... ...
Schwartz, Professor, Departments of Medicine and Community & Family Medicine, Dartmouth Medical School; and Co-Director, Center for Medicine and the Media, at The Dartmouth Institute for Health Policy and Clinical Practice, and Steven Woloshin, Professor, Departments of Medicine and Community & Family Medicine, Dartmouth Medical School; and Co-Director, Center for Medicine and the Media, at The Dartmouth Institute for Health Policy and Clinical Practice.
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From page 54... ...
Also in the initial FDA press release was a statement that the approval of Tysabri was based on positive results seen in patients after 1 year of treatment: The approval of Tysabri is based on positive results seen in patients after one year of treatment. This product received accelerated approval because it appears to provide substantial benefit for patients with a seri ous disease.
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From page 55... ...
Evidence shows that press releases can influence media reporting. Schwartz and Woloshin compared medical journal press releases with news coverage and showed a strong association between what was in the press release and what appeared in the subsequent news stories (Schwartz et al., 2012)
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From page 56... ...
Presentation at the IOM workshop series on Characterizing and Communicating Uncertainty in the Assessment of Benefits and Risks of Pharmaceutical Products. to the Tysabri case study, Schwartz noted that FDA issued another press release at the time Tysabri was withdrawn from the market in 2005: During the review of Tysabri for remarketing approval, FDA conducted an intensive analysis of possible adverse events that might be related to the effect of the drug on the immune system.
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From page 57... ...
Today, with a total of 10 FDA-approved MS therapies on the market, Fox generally suggests 2 or 3 therapeutic options for each 11 This section is based on the remarks by Carmen Bozic, Senior Vice President, Clinical and Safety Sciences, Biogen Idec; Robert Fox, Staff Neurologist, Mellen Center for Multiple Sclerosis and Vice-Chair for Research of the Neurological Institute, Cleveland Clinic; Alice Hughes, Deputy Director for Safety, Division of Neurology, CDER, FDA; Gavin HuntleyFenner, Human Factors and Safety Consultant, Huntley-Fenner Advisors; Joyce Korvick, Deputy Director for Safety, Division of Gastroenterology and Inborn Error Products, Office of Drug Evaluation III, CDER, FDA; and Cynthia Sitcov, Patient Representative & Voting Member, FDA Central and Peripheral Systems Advisory Committee.
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From page 58... ...
a benefit lexicon should be established and could include a benefits table to accompany the adverse events table in a medical product's package insert, and (3) the context in which medical products are used matters and could be better understood with individual stakeholder mapping.
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From page 59... ...
Building on the workshop discussions earlier in the day regarding patients' ability to understand quantitative estimates, Bozic and Robert Temple, Deputy Director for Clinical Science, and Acting Deputy Director, Office of Drug Evaluation I, CDER, FDA, suggested that industry and regulators could explore opportunities to include meaningful quantification of benefit and risk into medication guides and other communication tools.
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