Engaging Patients Across the Spectrum of Medical Product Development

Hunter, Nina L.; O’Callaghan, Kathryn M.; Califf, Robert M.

doi:10.1001/jama.2015.15818

Cited by 54 publications

(40 citation statements)

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“…Modern clinical development algorithms of candidate technology incorporate multi-disciplinary assessment by healthcare providers, developers, regulators, and payers [74] and seek active patient engagement [75]. This evolving landscape heralds an evolution in the medical product development and authorization lifecycle of novel therapies, from a paradigm focused on the therapeutics to a holistic evaluation that integrates the patient within a healthcare regimen.…”

Section: Discussionmentioning

confidence: 99%

Stem cell therapy for heart failure: Ensuring regenerative proficiency

Terzic

Behfar

2016

Trends in Cardiovascular Medicine

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Patient-derived stem cells enable promising regenerative strategies but display heterogenous cardiac reparative proficiency, leading to unpredictable therapeutic outcomes impeding practice adoption. Means to establish and certify the regenerative potency of emerging biotherapies are thus warranted. In this era of clinomics, deconvolution of variant cytoreparative performance in clinical trials offers an unprecedented opportunity to map pathways that segregate regenerative from non-regenerative states informing the evolution of cardioregenerative quality systems. A maiden example of this approach is cardiopoiesis-mediated lineage-specification developed to ensure regenerative performance. Successfully tested in pre-clinical and early clinical studies, the safety and efficacy of the cardiopoietic stem cell phenotype is undergoing validation in pivotal trials for chronic ischemic cardiomyopathy offering the prospect of a next-generation regenerative solution for heart failure.

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Section: Discussionmentioning

confidence: 99%

Stem cell therapy for heart failure: Ensuring regenerative proficiency

Terzic

Behfar

2016

Trends in Cardiovascular Medicine

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“…Additionally, the CDRH has developed guidance for patient involvement in regulatory decision making 11 and recently announced the formation of a Patient Engagement Advisory Committee, which will provide further patient perspectives on FDA policies. 12 In parallel, there has been growing emphasis on incorporating patient-reported outcomes (PROs) into device studies, reinforcing the importance of PROs in the regulatory process. The CDRH has also partnered with the Medical Device Innovation Consortium, a public-private partnership focused on medical device regulatory science, to develop a framework for incorporating preference data throughout the device development cycle and a catalog of valid scientific methods for evaluating patient perspectives in the regulatory context.…”

Section: Workhopmentioning

confidence: 99%

Stimulating Patient Engagement in Medical Device Development in Kidney Disease: A Report of a Kidney Health Initiative Workshop

Hurst

Chianchiano

Upchurch³

et al. 2017

American Journal of Kidney Diseases

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New technologies challenge current dialysis treatment paradigms as devices become smaller, more portable, and increasingly used outside of the dialysis clinic. It is unclear how patients will view this care transition, and it will be important to consider patient and care partner perspectives during all aspects of development for novel dialysis therapies, from design and clinical trials to regulatory approval. To gain insight into this area, the Kidney Health Initiative (KHI)—a public-private partnership between the American Society of Nephrology, the US Food and Drug Administration, and nearly 80 member organizations and companies dedicated to enhancing patient safety and fostering innovation in kidney disease—convened a workshop of patients, care partners, and other kidney community stakeholders. The workshop included background presentations followed by focused small-group discussions in three areas (device design, clinical trials, and regulatory approval). Participants explored how to involve patients throughout the lifecycle of a medical device, including discussions of how patients can influence device design, assist in the planning and implementation of clinical trials, and provide input to impact regulatory decisions. Patients were engaged in the workshop discussion and interested in sharing their perspectives, but they recommended additional efforts around education, communication, and outreach in these areas.

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“…His keynote address underscored that patient preferences and perspectives are important to the FDA. 20 …”

Section: Cultural Change Among Medical-product Sponsorsmentioning

confidence: 99%

Patient Engagement at a Tipping Point—The Need for Cultural Change Across Patient, Sponsor, and Regulator Stakeholders: Insights From the DIA Conference, “Patient Engagement in Benefit Risk Assessment Throughout the Life Cycle of Medical Products”

Smith

Hammad

Metcalf

et al. 2016

Ther Innov Regul Sci

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Benefit-risk assessment is the foundation for decision making throughout the life cycle of medical products. Because patients are the beneficiaries of the efficacy of medical treatments and also bear their possible risks, their perspectives and judgments about value and the relative importance of benefits and risks should be at the heart of the medical decision-making process. Patient engagement is now at a tipping point; there have been a growing number of patient engagement initiatives over the past several years, but there remains the need for a common language, alignment on engagement approaches and best practices, and a shared vision regarding a desired future state. This article discusses insights gleaned from the DIA conference, ''Patient Engagement in Benefit-Risk Assessment throughout the Life Cycle of Medical Products'' (September 2015). It highlights the changes that will need to occur within the patient, medical-product sponsor, and regulatory cultures in order for patient engagement to become integrated into the medical-product development process and life cycle maintenance. Furthermore, it emphasizes that reaching the desired future state will require a conscious commitment from all stakeholders to work collaboratively to develop shared solutions and to map a common path forward.

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Engaging Patients Across the Spectrum of Medical Product Development

Cited by 54 publications

References 1 publication

Stem cell therapy for heart failure: Ensuring regenerative proficiency

Stem cell therapy for heart failure: Ensuring regenerative proficiency

Stimulating Patient Engagement in Medical Device Development in Kidney Disease: A Report of a Kidney Health Initiative Workshop

Patient Engagement at a Tipping Point—The Need for Cultural Change Across Patient, Sponsor, and Regulator Stakeholders: Insights From the DIA Conference, “Patient Engagement in Benefit Risk Assessment Throughout the Life Cycle of Medical Products”

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