How Patient Preferences Contribute to Regulatory Decisions for Medical Devices

  • March 22, 2018

In August 2017, for the first time we cleared an expanded indication for a home hemodialysis machine so it could be used without a care partner being present, a decision based in part on asking kidney patients about their tolerance for risk.

Home dialysis may improve a patient’s quality of life, allowing them to undergo the lengthy treatments in the comfort of their home without the need to travel three times a week to a dialysis center.  However, due to a risk of rare but serious events associated with performing hemodialysis alone in the home, we had previously required the presence of a care partner during the treatment.

During our August 2015 public workshop as part of the Kidney Health Initiative, a public-private partnership, multiple patient representatives argued that the care partner requirement effectively ruled out home treatment for those patients who lived alone or who could not afford to hire a care partner.

We told them that we were willing to reconsider this issue but needed a systematic way to evaluate risk.  A medical device developer, NxStage, approached us at the meeting to propose a patient-centric approach.  During the pre-submission process, we worked with NxStage to design their robust patient survey that could quantify the level of risk that patients would accept in exchange for doing hemodialysis in the home alone instead of at a dialysis center.  The survey used a weighting method described in the Patient Preference Framework developed by the Medical Device Innovation Consortium (MDIC), a nonprofit that operates in partnership with FDA to improve the medical technology environment.  This data helped inform the review of NxStage System One’s expanded indication that allows for home hemodialysis treatments without the presence of a care partner.

The System One is the latest example of how we have been successfully implementing our Patient Preference Initiative, designed to identify and develop methods for assessing patient valuations of benefit and risk related to specific device types and specific illnesses and conditions that can be used to inform product review decisions.

Collecting qualitative feedback from patients is another important technique and proved helpful in enhancing the safety of the Dexcom G5 Continuous Glucose Monitoring (CGM) System and Animas Vibe System, a continuous glucose monitor with an insulin pump, for children.  FDA discussed with patients, care partners, and patient groups their concerns about the safety of using an insulin pump in the young pediatric population.  These conversations included how the device would be used once approved by FDA for the young pediatric population and what kind of safety considerations might be relevant.

Based on this feedback, FDA worked with the company to develop additional risk mitigation strategies that included a lockout feature to prevent unintended boluses by young children playing with the insulin pump.  Patient preference information led to a safer device on the market, and parents can now have greater confidence with managing their children’s diabetes.

These are good examples of how medical device companies are leveraging different types of patient preference information to support product submissions, information which is likely to vary depending on the device, the disease, the level of risk, and its purpose in the submission.

We invite companies to start a conversation with FDA about using patient preference information to support your submission.  Patient preference is also an evolving area of regulatory science, supported in part by the nonprofit MDIC, and FDA encourages further research in this field.

Jeffrey Shuren, M.D., J.D., is Director of FDA’s Center for Devices and Radiological Health

Anindita Saha, is Director of External Expertise and Partnerships at FDA’s Center for Devices and Radiological Health

Martin Ho, M.S., is Associate Director for Quantitative Innovation at FDA’s Center for Devices and Radiological Health

REFERENCE:  FDA Voice; 25 SEP 2017; Jeffrey Shuren, M.D., J.D., Anindita Saha, and Marin Ho, M.S.

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