From 2001 through 2016, medical device sponsors submitted 61,439  premarket notifications and FDA cleared 51,028 devices for market. In making its assessment  whether to approve, or clear, a submission, FDA relies on the sponsor to  provide supporting data as part of the submission. The  guidance also states that FDA was in the process of developing tools  to be used by both agency staff and its stakeholders to periodically  assess the implementation of the least burdensome principles. FDA Frequently Requested Additional Information to Support Medical Device Reviews, and Sponsor Disagreements Often Related to Least Burdensome Requirements

		FDA Issued Deficiency and Additional Information Letters for a Significant Proportion of PMAs and 510(k)s

FDA requested sponsors provide additional information for a majority of  the PMAs and 510(k)s it reviewed. Although FDA did not have readily available  data on appeals that occurred prior to 2013, the agency was able to  provide information about the 63 appeals of significant decisions that  occurred from 2013 through 2016. According to medical device industry representatives,  sponsors may not always pursue an appeal, so the number of official  appeals may not represent the extent of least burdensome-related issues  that sponsors experience. Of these 33 appeals, FDA agreed, or partially agreed with the sponsor for  11 appeals, which resulted in FDA overturning the decision or reopening  the file and continuing the review. FDA Offered Some Training on the Least Burdensome Requirements, and Evaluates its Training for Effectiveness

		FDA Offered Some Early Least Burdensome Training at Limited Times, and Has Incorporated Related Information in Broader Training

FDA officials indicated that training specific to the least burdensome  requirements was held in the years following the enactment of FDAMA in  1997. In response to the 21st Century Cures Act, enacted in December 2016,  FDA is providing mandatory online training specific to the least  burdensome requirements. FDA officials  told us that, as of October 31, 2017, 91 percent of CDRH staff had  received the new least burdensome specific training. FDA Is Implementing Evaluations of All Training Courses for Medical Device Review Staff, including Courses that Address the Least Burdensome Requirements

While FDA has not had processes in place to evaluate its medical device  training, it is implementing such processes for all training, including  courses related to the least burdensome requirements. In addition to its current training evaluation plan, FDA is also required by  the 21st Century Cures Act to conduct an audit of the training and its  effectiveness in implementing the least burdensome requirements. FDA is Taking Steps that May Improve Its Requests for Additional Information Overall, but Has Not Fully Evaluated Its Implementation of the Least Burdensome Requirements

		FDA Is Implementing Processes to Improve the Consistency and Clarity of Its Requests for Additional Information during Medical Device Reviews

Some stakeholders and others have raised concerns about the  consistency and clarity of FDA’s requests for additional information during  medical device reviews. Representatives of one of the organizations representing  the medical device industry noted the high percentages of medical device  submissions that involve a letter, and some of their member companies  have said that FDA reviewers may request additional information as a  result of intellectual curiosity rather than a “need to know.” In addition, the  independent assessment’s 2014 report, funded by FDA as part of  MDUFA III, found inconsistent decision-making among FDA review staff  throughout various stages of the review process, including additional  information requests. FDA Has Not Developed Metrics to Evaluate Implementation of the Least Burdensome Requirements, and While a New Audit Process Could Aid Oversight, Its Scope Is Still Unclear

FDA has not established performance metrics that would allow it to  evaluate its implementation of the least burdensome provisions. While FDA implemented  guidance and training related to the least burdensome requirements  following the passage of FDAMA in 1997, it has taken few steps to  develop performance metrics to evaluate the extent to which reviewers  are using a least burdensome approach when reviewing medical device  submissions. While planned audits of FDA’s medical device review  process have the potential to provide the agency with evaluation tools  through which to assess performance, these audits are still early in their  development and the extent to which they will allow FDA to assess  implementation of the least burdensome requirements is unclear. In this regard, HHS concurred with our recommendation  that it develop performance metrics and use them to evaluate the  implementation of the least burdensome requirements, such as during its  planned audits of medical device deficiency letters. At that time, we will send copies of this report to the  Secretary of Health and Human Services and other interested parties.
Why GAO Did This Study

Determining that a new medical device is safe and effective is a substantial investment of time and resources for the sponsor and FDA, the agency that regulates medical devices. FDA relies on the device sponsor to provide supporting data at the time of its original submission, and the agency can request additional information during the review. The Federal Food, Drug, and Cosmetic Act, as amended, requires that when FDA requests additional information from sponsors, the agency consider the least burdensome means of evaluating a medical device.
GAO was asked to provide information on FDA's implementation of the least burdensome requirements in its medical device review process. This report (1) describes FDA's requests for additional information and sponsor disagreements, (2) describes its least burdensome training efforts, and (3) describes FDA actions to improve its requests for additional information and examines the extent to which it has evaluated its implementation of the least burdensome requirements. GAO reviewed FDA documents and guidance and interviewed agency officials. GAO also interviewed officials from four relevant medical device manufacturing associations.

What GAO Found

Since 1997, the Food and Drug Administration (FDA) has been required to consider the least burdensome means of evaluating certain types of medical devices for marketing, including when requesting that sponsors—generally manufacturers—seeking to market their medical devices provide information in addition to what was provided in their submissions. GAO found that, from 2001 through 2016, FDA issued letters asking sponsors to provide such information for a majority of the more than 62,000 medical device submissions that it reviewed. Sponsors may formally disagree with the request on the grounds that it is not the least burdensome method needed for FDA to review the submission. For example, sponsors appealed FDA decisions internally to agency management 63 times from 2013 through 2016, and of these, FDA identified 33 such appeals in which the sponsor raised an issue related to least burdensome requirements. FDA agreed or partially agreed with the sponsors in 11 of these appeals. Medical device industry representatives noted that these appeals may not fully represent the number of such disagreements, because applicants are generally concerned that an appeal would damage their relationship with FDA and potentially negatively affect future device applications.
FDA provided staff training that was specifically dedicated to addressing the least burdensome requirements from 1997 through 1999. Since 1999, FDA has not offered a course dedicated to the least burdensome requirements, but has incorporated related concepts into other training programs, such as in a training mandatory for most new reviewers. In response to the 21st Century Cures Act, enacted in 2016, FDA is providing new least burdensome training to all relevant employees, and said that 80 percent had received the training as of October 2, 2017. Although FDA did not specifically evaluate the effectiveness of past training on least burdensome requirements, it is implementing an evaluation of all device-related training, including the new least burdensome training. It also plans to complete a required audit of training on least burdensome requirements by June 2018.
FDA has not specifically evaluated implementation of the least burdensome requirements. However, in response to broader evaluations, such as an independent assessment of its medical device review process, the agency is in the early stages of developing processes that may improve its requests for additional information. For example, FDA plans to conduct an audit of letters requesting additional information. FDA is developing the audit's methodology and expects it will assess whether the agency's process was followed. However, due to their early stage, the extent to which these efforts will allow FDA to assess implementation of the least burdensome requirements is unclear. In 2002, FDA stated that it planned to periodically assess the implementation of the least burdensome principles, and federal internal control standards identify the importance of performance metrics for such assessments. However, the agency has yet to develop performance metrics to do so. Until such measures are developed and used, FDA will not be able to evaluate whether it effectively and consistently applies a least burdensome approach in its medical device reviews.

What GAO Recommends

GAO is making one recommendation that FDA develop and use performance metrics to evaluate the implementation of the least burdensome requirements. The Department of Health and Human Services agreed with GAO's recommendation.