NORD at Capitol Hill Hearing: Patients Have Unique Perspective That Needs to be Heard

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NORD Board Member and Scientific Adviser Marshall Summar, MD, underscored the importance of the patient voice in his invited testimony on behalf of NORD at a July 11 hearing hosted by the House Energy & Commerce Committee Subcommittee on Health as part of the 21st Century Cures initiative.

“When we ask patients ‘What is the worst thing about your disease?’, we are often surprised by their answers,” Dr. Summar said. The direct experiences of patients and their families are unique and extremely important to the process of drug discovery and development, he added.

On behalf of NORD, Dr. Summar presented nine recommendations for accelerating the pace of medical innovation and improving the lives of patients and their families.  The nine recommendations are:

  • Establish a Commission and National Plan to Determine Priorities, Methods, Resource Needs and a Consistent Agenda on Rare Disease Registries and Natural History Studies
  • Significantly Reform the Institutional Review Board (IRB) System for Assessing New Therapies
  • Ensure that All Current Laws that Increase Patients’ Involvement are Implemented Fully
  • Ensure Sufficient and Consistent Resources, Direction and Funding for the Food and Drug Administration and National Institutes of Health
  • Commission a “National Plan for Rare Diseases”
  • Enhance the Focus on Clinical Trial Design and Endpoint Development within the NIH Division of Clinical Innovation within the National Center for Advancing Translational Sciences (NCATS)
  • Create an “Orphan Protected Class” within the Medicare Part D Program
  • Establish Clearer Federal Policies with Regard to Off-label use of Drugs
  • Ensure Reimbursement for Medical Foods