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Advancing Therapeutic Development for Pulmonary Morbidities Associated with Preterm Birth

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Abstract

Chronic pulmonary and respiratory conditions associated with preterm birth are incompletely characterized, complicating long-term treatment and development of more effective therapies. Stakeholders face challenges in the development of validated, clinically meaningful endpoints that adequately measure morbidities and predict or represent health outcomes for preterm neonates. We propose in this paper a research agenda, informed by the input of experts from a 2018 workshop we convened on this topic, to advance endpoint and treatment development. We discuss the necessity of further evaluation of existing endpoints and the improved characterization of disease endotypes. We also discuss key steps to the development of optimized short- and long-term endpoints that can be linked to meaningful health outcomes. Finally, we discuss the importance of limiting variability in data collection and the application of new clinical trial endpoints as well as the critical nature of multi-stakeholder collaboration to advancing therapeutic development for this vulnerable patient population.

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Funding

The development of this manuscript was supported by the U.S. Food and Drug Administration (7U19FD004971-04). This manuscript reflects the views of the authors and should not be construed to represent FDA’s views or policies.

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Authors and Affiliations

  1. Duke-Margolis Center for Health Policy, Washington, DC, USA

    Sarah Sheehan MPA, Morgan Romine MPA & Gregory Daniel PhD, MPH, RPh

  2. U.S. Food and Drug Administration, Silver Spring, MD, USA

    Gerri Baer MD, Robert Lim MD, Elektra Papadopoulos MD, MPH, Michelle Campbell PhD & Susan McCune MD

  3. Critical Path Institute, Tucson, AZ, USA

    Lynn Hudson PhD

  4. Duke-Margolis Center for Health Policy, 1201 Pennsylvania Ave. NW Suite 500, Washington, DC, 20004, USA

    Sarah Sheehan MPA

Authors
  1. Sarah Sheehan MPA
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  2. Gerri Baer MD
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  3. Morgan Romine MPA
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  4. Lynn Hudson PhD
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  5. Robert Lim MD
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  7. Michelle Campbell PhD
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  8. Gregory Daniel PhD, MPH, RPh
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  9. Susan McCune MD
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Contributions

SM, GD, MR, and LH, were key to conceptualizing and designing the content of the workshop that informed this manuscript and reviewed and provided critical revisions to the manuscript during development. GB was key to conceptualizing and designing the content of the workshop that informed the manuscript, drafted and provided critical revisions during manuscript development. SS conceptualized, designed, and drafted the manuscript, revised the manuscript for important intellectual content in response to working group input, and supervised manuscript submission. RL, EP, and MC provided feedback and revised the manuscript for important intellectual content throughout the manuscript development process. All authors approved the final manuscript as submitted and agree to be accountable for all aspects of work.

Corresponding author

Correspondence to Sarah Sheehan MPA.

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Conflicts of interest

Dr. Daniel receives Consulting Fees from AbbVie, Reagan-Udall Foundation, and Genentech and is the Editor-in-Chief of TIRS. The authors have no other conflicts of interest to declare.

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Sheehan, S., Baer, G., Romine, M. et al. Advancing Therapeutic Development for Pulmonary Morbidities Associated with Preterm Birth. Ther Innov Regul Sci 54, 1312–1318 (2020). https://doi.org/10.1007/s43441-020-00153-z

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  • DOI: https://doi.org/10.1007/s43441-020-00153-z

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