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Use of Patient-Reported Outcomes to Understand & Measure the Patient Experience of Novel Cell and Gene Therapies

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Abstract

Patient reported outcomes (PROs) are the gold standard for assessing patients’ experience of treatment in oncology, defined in the 21st Century Cures Act as information about patients’ experiences with a disease or condition, including the impact of a disease or condition, or a related therapy or clinical investigation on patients’ lives; and patient preferences with respect to treatment of their disease or condition [1]. PROs provide a comprehensive assessment of the benefits and risks of new medical products, as well as essential data to inform real-world use. Although RCTs are the ultimate source for information for evaluating products in development, they are not always feasible for rare diseases with few or no effective treatment options available. Thus, it is important to consider other measures that can help to improve the strength of evidence for cell and gene therapies targeting rare indications. While collection of PROs and other patient experience endpoints does not resolve the difficulty of conducting trials in small populations, doing so contributes empirical evidence that informs both product development and patient access. Additionally, including routine collection of PROs in registries may provide supplemental data to further characterize the benefit:risk profile of cell and gene therapies at follow-up times that would be infeasible to operationalize in a clinical trial setting.

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Acknowledgements

During the entirety of this work Alicyn Campbell worked exclusively with Patient Relevant Evidence as Founder and Strategic Lead. At the time of manuscript submission, Alicyn has added an additional role of Head of Digital Health for Oncology R&D at AstraZeneca.

Funding

Funding was not received for the preparation of this commentary.

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Authors and Affiliations

  1. Friends of Cancer Research, 1800 M St. NW Suite 1050S, Washington, DC, 20036, USA

    Laura Lasiter PhD, Mark Stewart PhD & Jeff Allen PhD

  2. Patient Relevant Evidence, San Francisco, USA

    Alicyn Campbell MPH

  3. University of North Carolina at Chapel Hill, Chapel Hill, USA

    Ethan Basch MD

  4. Clinical Outcomes Solutions, Tucson, USA

    Stacie Hudgens MSc

  5. Amgen, Inc, Thousand Oaks, USA

    James J. Wu MSc, MPH

  6. University of Pennsylvania, Philadelphia, USA

    Allison Barz Leahy MD

  7. Children’s Hospital of Philadelphia, Philadelphia, USA

    Allison Barz Leahy MD

Authors
  1. Laura Lasiter PhD
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  2. Alicyn Campbell MPH
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  3. Ethan Basch MD
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  4. Stacie Hudgens MSc
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  6. James J. Wu MSc, MPH
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  7. Allison Barz Leahy MD
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  8. Jeff Allen PhD
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All authors were involved in the conception, drafting & revising, and approval processes for this commentary. Each author agrees to be accountable for all aspects of the work.

Corresponding author

Correspondence to Laura Lasiter PhD.

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Conflict of interest

Dr. Basch reports grants from the National Cancer Institute and the Patient Centered Outcomes Research Institute, outside the submitted work; and Dr. Basch is on the Editorial Board of the Journal of the American Medical Association and consults on research projects at Memorial Sloan Kettering Cancer Center, Dana Farber Cancer Institute, Centers for Medicare & Medicaid Services, and Research Triangle Institute. Dr. Basch is a Scientific Advisor for Sivan Healthcare, CareVive Systems, and Navigating Cancer. Authors Laura Lasiter, Alicyn Campbell, Stacie Hudgens, Mark Stewart, James J. Wu, Allison Barz Leahy, and Jeff Allen have nothing to disclose.

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Lasiter, L., Campbell, A., Basch, E. et al. Use of Patient-Reported Outcomes to Understand & Measure the Patient Experience of Novel Cell and Gene Therapies. Ther Innov Regul Sci 54, 1566–1575 (2020). https://doi.org/10.1007/s43441-020-00184-6

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  • DOI: https://doi.org/10.1007/s43441-020-00184-6

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