The Patient Motivation Pyramid and Patient-Centricity in Early Clinical Development

2021 ◽  
Vol 16 ◽  
Author(s):  
Thijs van Iersel ◽  
Jocelyn Courville ◽  
Cathalijne van Doorne ◽  
Remco A. Koster ◽  
Christina Fawcett
Keyword(s):  
Trial Design ◽  
Clinical Development ◽  
Comprehensive Overview ◽  
Case Examples ◽  
Patient Motivation ◽  
Digital Medicine ◽  
Clinical Research Center ◽  
Potential Benefits ◽  
Early Clinical Development ◽  
Patient Centric

Background: It is increasingly recognized that patients should be involved in the design of clinical trials. However, there is a lack in agreement of what patient-centricity means. Methods: In this article a Patient Motivation Pyramid based on Maslow’s theory of human motivation is introduced as a tool to identify patient needs. This pyramid is used to make a comprehensive overview of options to implement patient-centric trial design. The Pyramid with the described options can help to identify patient-centric activities suitable for a given drug development. The current article further describes potential benefits of patient-centric trial designs with an emphasis on early clinical development. Especially in early clinical development during which trials have many assessments per patient, and the safety and clinical efficacy are uncertain, patient-centric trial design can improve feasibility. Finally, we present three case examples on patient-centric trial design. The first example is seeking patient input on the trial design for a First-in-Human trial which includes patients with Immune Thrombocytopenic Purpura. The second example is the use of a video link for home dosing. The final example is the use of digital medicine in a trial in heart failure patients. Results: A comprehensive overview of patients’ needs can be accomplished by building a Patient Motivation Pyramid as a tool. Patient input can lead to improved endpoints, improved feasibility, better recruitment, less dropout, less protocol amendments, and higher patient satisfaction. The use of digital medicine can lead to a trial design with much less visits to the clinical research center in early clinical development, and in a later development phase even to a complete virtual trial. Conclusion: We recommend using the Patient Motivation Pyramid as structural approach for identifying elements of patient-centricity. Secondly we recommend to start using patient-centric approaches in an early phase of the medicine’s lifecycle.

scholarly journals Parkinson’s Patients’ Tolerance for Risk and Willingness to Wait for Potential Benefits of Novel Neurostimulation Devices: A Patient-Centered Threshold Technique Study

2021 ◽  
Vol 6 (1) ◽  
pp. 238146832097840
Author(s):  
Brett Hauber ◽  
Brennan Mange ◽  
Mo Zhou ◽  
Shomesh Chaudhuri ◽  
Heather L. Benz ◽  
...  
Keyword(s):  
Trial Design ◽  
Patient Characteristics ◽  
Risk Tolerance ◽  
Level Of Evidence ◽  
Treatment Benefit ◽  
Study Results ◽  
Potential Benefits ◽  
Risk Thresholds ◽  
Time Motor ◽  
Threshold Technique

Background. Parkinson’s disease (PD) is neurodegenerative, causing motor, cognitive, psychological, somatic, and autonomic symptoms. Understanding PD patients’ preferences for novel neurostimulation devices may help ensure that devices are delivered in a timely manner with the appropriate level of evidence. Our objective was to elicit preferences and willingness-to-wait for novel neurostimulation devices among PD patients to inform a model of optimal trial design. Methods. We developed and administered a survey to PD patients to quantify the maximum levels of risks that patients would accept to achieve potential benefits of a neurostimulation device. Threshold technique was used to quantify patients’ risk thresholds for new or worsening depression or anxiety, brain bleed, or death in exchange for improvements in “on-time,” motor symptoms, pain, cognition, and pill burden. The survey elicited patients’ willingness to wait to receive treatment benefit. Patients were recruited through Fox Insight, an online PD observational study. Results. A total of 2740 patients were included and a majority were White (94.6%) and had a 4-year college degree (69.8%). Risk thresholds increased as benefits increased. Threshold for depression or anxiety was substantially higher than threshold for brain bleed or death. Patient age, ambulation, and prior neurostimulation experience influenced risk tolerance. Patients were willing to wait an average of 4 to 13 years for devices that provide different levels of benefit. Conclusions. PD patients are willing to accept substantial risks to improve symptoms. Preferences are heterogeneous and depend on treatment benefit and patient characteristics. The results of this study may be useful in informing review of device applications and other regulatory decisions and will be input into a model of optimal trial design for neurostimulation devices.

scholarly journals Blockchain Technology in the Food Industry: A Review of Potentials, Challenges and Future Research Directions

Logistics ◽  
2020 ◽  
Vol 4 (4) ◽  
pp. 27
Author(s):  
Abderahman Rejeb ◽  
John G. Keogh ◽  
Suhaiza Zailani ◽  
Horst Treiblmaier ◽  
Karim Rejeb
Keyword(s):  
Food Industry ◽  
Future Research ◽  
Journal Articles ◽  
Comprehensive Overview ◽  
Illicit Trade ◽  
Food Supply Chains ◽  
Blockchain Technology ◽  
Potential Benefits ◽  
Future Research Directions ◽  
Business Customers

Blockchain technology has emerged as a promising technology with far-reaching implications for the food industry. The combination of immutability, enhanced visibility, transparency and data integrity provides numerous benefits that improve trust in extended food supply chains (FSCs). Blockchain can enhance traceability, enable more efficient recall and aids in risk reduction of counterfeits and other forms of illicit trade. Moreover, blockchain can enhance the integrity of credence claims such as sustainably sourced, organic or faith-based claims such as kosher or halal by integrating the authoritative source of the claim (e.g., the certification body or certification owner) into the blockchain to verify the claim integrity and reassure business customers and end consumers. Despite the promises and market hype, a comprehensive overview of the potential benefits and challenges of blockchain in FSCs is still missing. To bridge this knowledge gap, we present the findings from a systematic review and bibliometric analysis of sixty-one (61) journal articles and synthesize existing research. The main benefits of blockchain technology in FCSs are improved food traceability, enhanced collaboration, operational efficiencies and streamlined food trading processes. Potential challenges include technical, organizational and regulatory issues. We discuss the theoretical and practical implications of our research and present several ideas for future research.

scholarly journals Spontaneous cervical epidural hematoma: Insight into this occurrence with case examples

2021 ◽  
Vol 12 ◽  
pp. 79
Author(s):  
Brian Fiani ◽  
Ryan Jarrah ◽  
Nicholas J. Fiani ◽  
Juliana Runnels
Keyword(s):  
Epidural Hematoma ◽  
Clinical Presentation ◽  
Treatment Algorithm ◽  
Region Of Interest ◽  
Sudden Onset ◽  
Neurological Deficits ◽  
Spinal Epidural Hematoma ◽  
Comprehensive Overview ◽  
Case Examples ◽  
First Case

Background: First characterized in the 19th century, spontaneous spinal epidural hematoma (SSEH) is known as the idiopathic accumulation of blood within the spinal canal’s epidural space, causing symptoms varying from general back pain to complete paraplegia. With varying etiologies, a broad spectrum of severity and symptoms, a time-dependent resolution period, and no documented diagnosis or treatment algorithm, SSEH is a commonly misunderstood condition associated with increasing morbidity. While SSEH can occur at any vertebrae level, 16% of all SSEH cases occur in the cervical spine, making it a region of interest to clinicians. Case Description: Herein, the authors present two case examples describing the clinical presentation of SSEH, while also reviewing the literature to provide a comprehensive overview of its presentation, pathology, and treatment. The first case is a patient with nontraumatic sudden onset neck pain with rapidly progressing weakness. The second case is a patient with painless weakness that developed while taking 325 mg of aspirin daily. Conclusion: Clinicians should keep SSEH in their differential diagnosis when seeing patients with nontraumatic sources of weakness in their extremities. The appropriate steps should be followed to diagnose and treat this condition with magnetic resonance imaging and surgical decompression if there are progressive neurological deficits. There is a continued need for more extensive database-driven studies to understand better SSEHs clinical presentation, etiology, and ultimate treatment.

scholarly journals Edaravone in Amyotrophic Lateral Sclerosis’Lessons from the Clinical Development Program and the Importance of a Strategic Clinical Trial Design

US Neurology ◽  
2018 ◽  
Vol 14 (1) ◽  
pp. 47 ◽  
Author(s):  
Said R Beydoun ◽  
Jeffrey Rosenfeld
Keyword(s):  
Clinical Trial ◽  
Clinical Trials ◽  
Amyotrophic Lateral Sclerosis ◽  
Trial Design ◽  
Development Program ◽  
Clinical Trial Design ◽  
Clinical Development ◽  
Disease Heterogeneity ◽  
Clinical Development Program ◽  
Lateral Sclerosis

Edaravone significantly slows progression of amyotrophic lateral sclerosis (ALS), and is the first therapy to receive approval by the Food and Drug Administration (FDA) for the disease in 22 years. Approval of edaravone has marked a new chapter in pharmaceutical development since the key trial included a novel strategic clinical design involving cohort enrichment. In addition, approval was based on clinical trials that had a relatively small patient number and were performed outside of the US. Edaravone was developed through a series of clinical trials in Japan where it was determined that a well-defined subgroup of patients was required to reveal a treatment effect within the study period. Amyotrophic lateral sclerosis is associated with wide-ranging disease heterogeneity (both within the spectrum of ALS phenotypes as well as in the rate of progression). The patient cohort enrichment strategy aimed to address this heterogeneity and should now be considered as a viable, and perhaps preferred, trial design for future studies. Future research incorporating relevant biomarkers may help to better elucidate edaravone’s mechanism of action, pharmacodynamics, and subsequently ALS phenotypes that may preferentially benefit from treatment. In this review, we discuss the edaravone clinical development program, outline the strategic clinical trial design, and highlight important lessons for future trials.

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