Benefit–Risk Assessment With the Patient in Mind

Myrl Weinberg
iHealth Connections, 2011;1(1):21–3
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Abstract

People who take medications on a daily basis understand there are risks associated with drugs and they often are willing to take those risks in exchange for a better life. It is only when you consider both the benefit and the risk of using a drug—or the benefit–risk of not using the drug—and frame that scenario in the context of the condition being treated that policy makers, patients, and providers can make an effective judgment about a treatment. None of the main regulatory authorities has issued benefit–risk criteria for the approval of new treatments. So as agencies create an objective and predictable framework for assessing the benefit–risk of new drugs, it should ensure the perspective of the patient community is well-represented in policy discussions.
Disclosure The author has no conflicts of interest to declare.
Correspondence: mweinberg@nhcouncil.org

Life is full of trade-offs. No one understands that better than a person with a chronic disease or disability. For more than 133 million people in the US living with a chronic condition, the delicate balance between benefits and risks associated with a treatment plan is more than a topic of theoretical debate.

People who take medications on a daily basis understand that there are risks associated with drugs and they often are willing to take those risks in exchange for a better shot at a more normal, productive, and happy life. One patient with multiple sclerosis who was interviewed by National Public Radio (NPR) said he was eager to try whatever offered him the first real glimmer of hope for a cure, a sentiment frequently expressed by individuals with chronic diseases and disabilities.1 Both sides of the benefit–risk equation play into a patient’s decision-making process. The public discussion on new treatments, however, is often concerned more with the risk and not the benefit.

It is only when you consider both the benefit and the risk of using a drug—or in some cases the benefit–risk of not using the drug—and frame that scenario in the context of the condition or conditions being treated, that policy makers, patients, and providers can make any effective judgment about a treatment.

Surprisingly, none of the main regulatory authorities—the EU, US, or Japan—has issued a list of benefit and risk criteria that they apply to the approval of new treatments. According to the European Medicines Agency (EMA) and its Committee for Medicinal Products for Human Use (CHMP), guidance on the principles and methodology for benefit–risk assessment is also lacking.2

Policy makers are wrestling with finding the right balance that acknowledges the need to protect the public and yet provides the hope that people with chronic diseases and disabilities seek. The Food and Drug Administration (FDA) Center for Drug Evaluation and Research (CDER) acknowledges that the approval process for new treatments could benefit from more structured discussions on a range of issues to better gauge the benefit–risk assessment.3

Now is the optimum time to take action because of the need to reauthorize the Prescription Drug User Fee Act (PDUFA). PDUFA allows the FDA to collect fees from companies that produce certain human drug and biological products and these fees fund about half of the costs for the review of new drugs.4 PDUFA reauthorization legislation must be passed by Congress in 2012 in order for the program to continue.

Recent discussions on the future of PDUFA have focused on changes that could be made to improve the review process, including the development of a more objective and predictable framework for assessing the benefit–risk of new drugs.

References:
  1. Kroen G, Doctor challenges cause of MS and treatment, National Public Radio, 2011. Available at: www.npr.org/2011/01/31/133247319/doctor-challengescause- of-ms-and-treatment (accessed March 23, 2011).
  2. Committee for Medicinal Products for Human Use, Reflection paper on benefit-risk assessment methods in the context of the evaluation of marketing authorisation applications of medicinal products for human use, 2008. Available at: www.emea.europa.eu/docs/en_GB/document_library/Reg ulatory_and_procedural_guideline/2010/01/WC500069634 .pdf (accessed March 23, 2011).
  3. Mullin T, Food and drug administration, Benefit-risk and patient-focused drug development, Presented at: PDUFA V Stakeholder Meeting, Silver Spring, Maryland, 11 January 2011.
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  5. Food and Drug Administration, Guidance for industry format and content of proposed Risk Evaluation and Mitigation Strategies (REMS), REMS assessments, and proposed REMS modifications, 2009. Available at: www.fda.gov/downloads/Drugs/GuidanceComplianceReg ulatoryInformation/Guidances/UCM184128.pdf (accessed March 23, 2011).
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