FDA News: Medical device needs for rare diseases, Accelerating next generation sequencing-based tests, Essure sale retsriction – Drug and Device Digest


Unmet medical device Needs for patients with rare diseases

FDA, National Center for Advancing Translational Sciences (NCATS)/Office of Rare Diseases Research (ORDR) at NIH sought to better understand medical device needs of patients with rare disease

  • Generate meaningful data to inform patients, practitioners, policymakers, and device developers
  • Needs, barriers, and incentives

Online survey of four clinician groups

  • satisfaction with current devices
  • unmet needs for specific rare diseases
  • impediments to medical device development

Survey Respondents: 1,342 clinicians


  • Patients with rare diseases face numerous unmet needs
  • Device needs of pediatric patients – grow with child, be modified to smaller size, less invasive
  • Creating entirely new devices needed vs. modifying/repurposing existing devices
  • Limitations included lack of sensitivity/specificity, cumbersome and invasive
  • Costs of research, lack of profitability, challenges of conducting trials are challenges



FDA finalizes guidances to accelerate the development of reliable, beneficial next generation sequencing-based tests

Finalized two guidances for  efficient development of novel technology that scans DNA to diagnose genetic diseases-next generation sequencing (NGS)

  •  Recommendations for designing, developing, and validating tests; for continued advancement of individualized, genetic-based medicine
  • Modern and flexible framework

Use of Public Human Genetic Variant Databases to Support Clinical Validity for Genetic and Genomic-Based In Vitro Diagnostics

  • Reliance on clinical evidence from FDA-recognized public databases to support clinical claims e.g. ClinGen

Considerations for Design, Development, and Analytical Validation of Next Generation Sequencing (NGS)–Based In Vitro Diagnostics (IVDs) Intended to Aid in the Diagnosis of Suspected Germline Diseases

  • Recommendations for designing, developing, validating tests to diagnose individuals with suspected genetic diseases

Based on extensive feedback from the public and stakeholders; continuation of creating regulatory efficiencies in the development and review of NGS tests


CaptureFDA Restricts the Sale and Distribution of Essure

Order to restrict the sale and distribution of the Essure device

  • Ensure  all women provided with adequate risk information so that they can make informed decisions
  • Taking this step because some women were not being adequately informed of Essure’s risks before getting the device implanted
  •  Boxed Warning including perforation of the uterus and/or fallopian tubes, identification of inserts in the abdominal or pelvic cavity, persistent pain, and suspected allergic or hypersensitivity reactions
  • FDA closely evaluating new information on the use of Essure; requires additional, meaningful safeguards to ensure women are able to make informed decisions

New Essure labeling

  • Restricts sale and distribution to only health care providers and facilities that provide information to patients about the risks and benefits of this device
  • Review “Patient-Doctor Discussion Checklist – Acceptance of Risk and Informed Decision Acknowledgement”
  • Patient and physician required to sign
  • FDA will review and monitor Bayer’s plan to ensure compliance of restriction


Image credit: FDA

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