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FDA and Patient Interactions On Benefit-Risk Determinations

FDA and Patient Interactions On Benefit-Risk Determinations



Presented to the Regulatory Education and Action for Patients (REAP) Coalition on 7/25/12

Presented to the Regulatory Education and Action for Patients (REAP) Coalition on 7/25/12



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  • AC Meetings - , where they offer the patient perspective, ask questions, and give comments to assist the committee in making recommendations. Consultants – assist the clinicians and scientists who review data submitted to determine whether the product’s benefits outweigh the potential risks
  • Webiste: Medical Product Development 101 Library of Written & Multimedia Educational Resources Updates on FDA actions, upcoming meetings, opportunities to comment (i.e. dockets) Interactivity
  • Today I am going to discuss the guidance document entitled, Factors to Consider When Making Benefit-Risk Determinations in Medical Device Premarket Approval and De Novo Classifications. This guidance document was published in its final form on March 27, 2012.
  • This guidance document was developed to provide greater clarity for FDA reviewers and Industry regarding the factors FDA considers when making benefit-risk assessments in PMA applications and de novo premarket submissions for medical devices. FDA believes that the appropriate application of the factors listed in this guidance will help FDA believes that the full implementation of the guiding factors described in this guidance will facilitate transparency, consistency, and predictability of the premarket review process.
  • This guidance addresses two main avenues for a sponsor to gain market entry for a new medical device: PMAs and de novo petitions. Under Section 513(a) of the Federal Food, Drug & Cosmetic Act, or the “FD&C Act,” FDA determines whether PMA applications provide a “reasonable assurance of safety and effectiveness” by “weighing any probable benefit to health from the use of the device, against any probable risk of injury or illness from such use.” Other factors also apply. FDA reviews the valid scientific evidence submitted in the application to determine whether it supports the intended use of the device. This evidence may come from a range of information sources, such as clinical data, animal studies, and non-clinical data. Scientific data reviewed by FDA includes clinical studies, non-clinical studies, and the intended use of the device. Wherever eligible, the Sponsor may submit a de novo classification petition in order to gain market entry for a new medical device. For de novos, FDA considers the risk profile of a device to make a classification determination for the device under Section 513(a)(1) of the FD&C Act. FDA makes a risk-based classification determination after consideration of all risks of the device, such that the risks may appropriately be mitigated through the general and/or special controls. A complete benefit-risk assessment is then carried out as part of the assessment of device's safety and effectiveness. . Devices that reach the market via a de novo classification petition serves as predicates for future submissions through the 510(k) program.
  • This guidance was developed by the benefit-risk working group, made up of FDA Staff from the Office of Device Evaluation (ODE), the Office of Surveillance and Biometrics (OSB), the Office of In Vitro Diagnostics (OIVD), and the Office of Science and Engineering Laboratories (OSEL). This group’s effort led to the issuance of the draft guidance in August 2011, which was published and available for public comment The comment period was open until November 15, 2011, at which time the docket closed and comments were collated, addressed, and incorporated as appropriate into the final guidance. The final version of the guidance was published on March 27, 2012.
  • The guidance document addresses and defines multiple factors FDA considers important in making benefit-risk determinations. Several examples are included within the guidance document to demonstrate how the factors are applied in making benefit-risk determinations. A worksheet is provided at the end of the guidance. This worksheet is intended to capture how reviewers consider the factors described within the guidance when making benefit-risk assessments.
  • The factors I will now describe are considered within the intended use of the device when assessing the benefit-risk profile. I will present each factor, followed by examples of questions the FDA reviewers consider for each factor described. The guidance document contains a worksheet that provides additional questions for each factor.
  • This guidance characterizes the benefit-risk determinations into three primary categories: the factors that characterize the benefits of the device; the factors associated with the risks of the device; and, finally, additional factors that FDA considers while weighing the probable benefits and risks.
  • The guidance document helps FDA reviewers assess the extent of the probable benefit using several factors, including the type of benefits, the magnitude of the benefits, the probability of the patient experiencing one or more of the benefits, and the duration of the benefits. These factors are considered individually and in the aggregate. The type of benefit can be measured directly, or by using endpoints or surrogate endpoints. Knowing what primary endpoints or surrogate endpoints were evaluated and what value physicians and patients place on the benefit are important considerations. Additionally, understanding the impact of the benefit to public health may be helpful for therapeutic devices and early diagnosis of a disease. FDA often assesses magnitude of benefits along a scale or according to specific endpoints or criteria, or by evaluating whether a pre-identified health threshold was achieved.
  • Another measure of benefit is the probability of the patient experiencing one or more benefits. Based on the data provided, does the study predict the which patients will experience a benefit or the probability that a patient will experience a benefit. Understanding the variation in public health benefits for different subgroups is important because different patient subgroups may experience different benefits or different levels of the same benefit. A final measure of benefit is the duration of the effect of the benefit. A treatment whose benefit lasts longer is more desirable than a treatment that must be repeated to preserve the benefit.
  • FDA determines the Risk profile of the device by taking into the severity, types, number and rates of harmful events; the probability of a harmful event; the duration of each harmful event; and the risk from false positive or false negative results, as in the case of diagnostic devices. These factors are considered individually and in the aggregate. The severity, types, number and rates of harmful events refers to events that result directly from the patient’s use of the device. Examples of questions considered by the reviewer include: what are the device-related serious adverse events? What are the device-related non-serious adverse events? And, what other procedure-related or indirect complications to which a patient be subjected? The probability of a harmful event can be assessed by understanding what the probability is that a patient in the intended population will experience a harmful event. Reviewers also consider the question, “are patients willing to accept the probable risk of the harmful event, in exchange for the probable benefits of the device?”
  • Another factor FDA considers when making benefit-risk determinations is the availability of alternative treatments or diagnostics. FDA takes into account how effective these other treatments are and the risks they pose to patients. The use of risk mitigation strategies, when appropriate, can minimize the probability of a harmful event occurring. The reviewer will determine if the sponsor has identified ways to mitigate risks through product labeling or education programs. For in vitro diagnostics, risks may be mitigated by the use of complementary diagnostic tests.

FDA and Patient Interactions On Benefit-Risk Determinations FDA and Patient Interactions On Benefit-Risk Determinations Presentation Transcript