scholarly journals Digital Health Technology to Measure Drug Efficacy in Clinical Trials for Parkinson’s Disease: A Regulatory Perspective

2021 ◽  
pp. 1-5
Author(s):  
Leonard Sacks ◽  
Elizabeth Kunkoski

Digital health technology (DHT), including wearable and environmental sensors, video cameras and other electronic tools, has provided new opportunities for the measurement of movement and functionality in Parkinson’s disease. Compared to current standards for evaluation of the disease (MDS-UPDRS), DHT may offer new possibilities for more frequent objective measurements of the duration, severity and frequency of disease manifestations over time, that may provide more information than periodic clinic visits. However, DHT measurement are only scientifically and medically useful if they are accurate, reliable and clinically meaningful. Verification and validation, also known as analytical validation and clinical validation, of DHT performance is important to ensure the accuracy and precision of measurements, and the specificity of findings. Given the wide range of clinical manifestations associated with Parkinson’s disease and the many tools and metrics to assess them, the challenge is to identify those that may represent a standard for use in clinical trials, and to confirm when digital measurements succeed or fall short of capturing meaningful benefits during drug development.

Author(s):  
Anthony E. Lang

ABSTRACTParkinson’s disease is associated with a variety of neurotransmitter disturbances which may be further altered by its treatment with dopamine agonists. Based on this information a wide range of pharmacological approaches have been used in search of newer treatment alternatives and in hopes of reducing complications of long-term levodopa use. This paper reviews the various therapies which have had some success in the management of Parkinson’s disease, other than levodopa and dopamine agonists. Special emphasis is placed on the many unresolved questions and controversies that exist in this area of neuropharmacology.


Author(s):  
Daniel J. van Wamelen ◽  
Jirada Sringean ◽  
Dhaval Trivedi ◽  
Camille B. Carroll ◽  
Anette E. Schrag ◽  
...  

2019 ◽  
Author(s):  
Solomiia Bandrivska

Parkinson's disease (PD) - is a chronic progressive degenerative disease of the central nervous system, the main clinical manifestations of which are hypokinesia, rigidity, resting tremor, postural disorders, which as the disease progresses grow and eventually lead to immobility of patients [Stok V. N., Fedorova N.V., 1997, Jahno N.N., 1995]. Modern anti parkinson therapy is mainly symptomatic and, apparently, does not affect the current degeneration process. Therefore, even with the optimal treatment today, disability of the patient is inevitable in the future. In addition to classical motor disorders, the cause of disability can be a wide range of non-motor disorders (mental , vegetative , sensory).


2020 ◽  
Author(s):  
Solveig K. Sieberts ◽  
Jennifer Schaff ◽  
Marlena Duda ◽  
Bálint Ármin Pataki ◽  
Ming Sun ◽  
...  

AbstractMobile health, the collection of data using wearables and sensors, is a rapidly growing field in health research with many applications. Deriving validated measures of disease and severity that can be used clinically or as outcome measures in clinical trials, referred to as digital biomarkers, has proven difficult. In part due to the complicated analytical approaches necessary to develop these metrics. Here we describe the use of crowdsourcing to specifically evaluate and benchmark features derived from accelerometer and gyroscope data in two different datasets to predict the presence of Parkinson’s Disease (PD) and severity of three PD symptoms: tremor, dyskinesia and bradykinesia. Forty teams from around the world submitted features, and achieved drastically improved predictive performance for PD status (best AUROC=0.87), as well as tremor (best AUPR=0.75), dyskinesia (best AUPR=0.48) and bradykinesia (best AUPR=0.95) severity.


2021 ◽  
pp. 1-11
Author(s):  
Lisa Damron ◽  
Irene Litvan ◽  
Ece Bayram ◽  
Sarah Berk ◽  
Bernadette Siddiqi ◽  
...  

Background: Hispanics are under-represented in Parkinson’s disease (PD) research despite the importance of diversity for results to apply to a wide range of patients. Objective: To investigate the perspective of Hispanic persons with Parkinson disease (PWP) regarding awareness, interest, and barriers to participation in research. Methods: We developed and administered a survey and qualitative interview in English and Spanish. For the survey, 62 Hispanic and 38 non-Hispanic PWP linked to a tertiary center were recruited in Arizona. For interviews, 20 Hispanic PWP, 20 caregivers, and six physicians providing service to Hispanic PWP in the community were recruited in California. Survey responses of Hispanic and non-Hispanic PWP were compared. Major survey themes were identified by applying grounded theory and open coding. Results: The survey found roughly half (Q1 54%, Q2 55%) of Hispanic PWP linked to a tertiary center knew about research; there was unawareness among community Hispanic PWP. Most preferred having physician recommendations for research participation and were willing to participate. Hispanics preferred teams who speak their native language and include family. Research engagement, PD knowledge, role of family, living with PD, PD care, pre-diagnosis/diagnosis emerged as themes from the interview. Conclusion: Barriers exist for participation of Hispanic PWP in research, primarily lack of awareness of PD research opportunities. Educating physicians of the need to encourage research participation of Hispanic PWP can address this. Physicians need to be aware of ongoing research and should not assume PWP disinterest. Including family members and providing research opportunities in their native language can increase research recruitment.


2021 ◽  
pp. 1-6
Author(s):  
Mark Tomishima ◽  
Agnete Kirkeby

After many years of preclinical development, cell and gene therapies have advanced from research tools in the lab to clinical-grade products for patients, and today they constitute more than a quarter of all new Phase I clinical trials for Parkinson’s disease. Whereas efficacy has been convincingly proven for many of these products in preclinical models, the field is now entering a new phase where the functionality and safety of these products will need to stand the test in clinical trials. If successful, these new products can have the potential to provide patients with a one-time administered treatment which may alleviate them from daily symptomatic dopaminergic medication.


2021 ◽  
Vol 11 (1) ◽  
pp. 43
Author(s):  
Piyush Varma ◽  
Lakshanaa Narayan ◽  
Jane Alty ◽  
Virginia Painter ◽  
Chandrasekhara Padmakumar

Introduction: Parkinson’s disease is a heterogeneous clinical syndrome. Parkinson’s disease in older persons presents with a diverse array of clinical manifestations leading to unique care needs. This raises the need for the healthcare community to proactively address the care needs of older persons with Parkinson’s disease. Though it is tempting to categorise different phenotypes of Parkinson’s disease, a strong evidence based for the same is lacking. There is considerable literature describing the varying clinical manifestations in old age. This article aims to review the literature looking for strategies in personalising the management of an older person with Parkinson’s disease.


Neurology ◽  
2003 ◽  
Vol 60 (8) ◽  
pp. 1234-1240 ◽  
Author(s):  
B.M. Ravina ◽  
S.C. Fagan ◽  
R.G. Hart ◽  
C.A. Hovinga ◽  
D.D. Murphy ◽  
...  

Sign in / Sign up

Export Citation Format

Share Document