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Regulating Between the Notes: The US FDA and the Evolution of the Patient Voice Through Twenty-First Century Regulatory Science

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References

  1. Combined FDA and Applicant Briefing Document NDA# 216660. https://www.fda.gov/media/157186/download. Accessed 9 Aug 2020.

  2. AMX0035 in Patients with Amyotrophic Lateral Sclerosis (ALS) (CENTAUR) https://clinicaltrials.gov/ct2/show/NCT03127514. Accessed 9 Aug 2022.

  3. Sarepta Therapeutics web site. https://www.sarepta.com/. Accessed 9 Aug 2022.

  4. Guidance for Industry. Patient-reported outcome measures: use in medical product development to support labeling claims. https://www.fda.gov/media/77832/download. Accessed 9 Aug 2022.

  5. Health-Related Quality of Life (HRQOL), US Centers for Disease Control, Last accessed August 9, 2022.

  6. CDER patient-focused drug development. https://www.fda.gov/drugs/development-approval-process-drugs/cder-patient-focused-drug-development. Accessed 9 Aug 2022.

  7. Sarepta and the regulation of hope. https://morningconsult.com/opinions/sarepta-regulation-hope/. Accessed 9 Aug 2022.

  8. Redberg RF, Kesselheim AS, Califf RM. Are they safe? Drugs and devices receiving accelerated approval by the FDA. http://jamanetwork.com/learning/audio-player/14650592. Accessed 9 Aug 2022.

  9. Pitts PJ, Brady P. From the valley of death to the crossroads of opportunity: a discussion of evolving benefit/risk evaluation standards. https://doi.org/10.1177/2168479018774556. Accessed 9 Aug 2022.

  10. The FDA has devalued the gold standard on R&D. And that threatens everyone in drug development. https://endpts.com/the-fda-has-devalued-the-gold-standard-on-rd-and-that-threatens-everyone-in-drug-development/. Accessed 9 Aug 2022.

  11. FDA's Accelerated Approval Program Webpage. https://www.fda.gov/drugs/information-health-care-professionals-drugs/accelerated-approval-program. Accessed 9 Aug 2022.

  12. FDA's Breakthrough Therapy Webpage. https://www.fda.gov/patients/fast-track-breakthrough-therapy-accelerated-approval-priority-review/breakthrough-therapy. Accessed 9 Aug 2022.

  13. https://www.amazon.com/NEXT-NORMAL-Peter-J-Pitts-ebook/dp/B09SMVJ71W. Accessed 9 Aug 2022.

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  1. Center for Medicine in the Public Interest, 757 Third Avenue, New York, NY, 10017, USA

    Peter J. Pitts

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  1. Peter J. Pitts
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Correspondence to Peter J. Pitts.

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Pitts, P.J. Regulating Between the Notes: The US FDA and the Evolution of the Patient Voice Through Twenty-First Century Regulatory Science. Patient 15, 611–613 (2022). https://doi.org/10.1007/s40271-022-00599-3

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