This document discusses strategies for optimizing patient recruitment in clinical trials, especially for rare diseases. It notes that patient recruitment is critical for drug and device approval but can be challenging for rare diseases where patients are spread out globally. New technologies like social media and mobile devices have helped increase awareness of studies. Trial designs are evolving to be more accessible and reduce patient burden, such as through multi-site trials. Collaboration between clinical research organizations, sites, and patient groups can also enhance recruitment. The future of recruitment looks encouraging as awareness and access continue to improve through technological advances.
5 years of “Rare” Progress Research: Cheryl Rockman-Greenberg, Max Rady College of Medicine, University of Manitoba
Rare Disease Day Conference 2020 March 9-10
IRCM’s Centre on Rare and Genetic Diseases in Adults
Sophie Bernard, M.D., PhD.
Head of the Rare Disease Clinic, IRCM Director, Genetic Dyslipidemias Clinic, IRCM Assistant Professor, Department of Medicine, Montreal University
Rare Disease Day Conference 2020 March 9-10
The utility of psychotropic drugs on patients with fetal alcohol spectrum dis...BARRY STANLEY 2 fasd
ABSTRACT
BACKGROUND: Treatment of the complications arising from Prenatal Alcohol Exposure (PAE) has largely been focused on psychosocial and environmental approaches. Research on the
use of medications, especially psychotropic medications, has lagged behind.
OBJECTIVES: This systematic review sought to investigate psychotropic medication related findings and outcomes in those diagnosed with Fetal Alcohol Spectrum Disorder (FASD).
METHODS: Comprehensive searches were conducted in seven major databases (Medline/
PubMed, Scopus, Web of Knowledge, Embase, PsycINFO, Cochrane Library, and
PsycARTICLES) up to February 2017. Key search terms with synonyms were mapped on these databases. There were no timeline restrictions and no grey literature searches. Two reviewers
independently assessed 25 studies that met the inclusion criteria. Most studies were reviews of treatment and retrospective case series.
RESULTS: Two crossover randomized trials were reported, and the findings were not amenable to meta-analysis. Several conditions (depression, agitation, seizures, and outburst) combined with the most frequent presentation, ADHD, to represent the rationale for prescribing psychotropic medications. Second-generation antipsychotics were found to improve social skills, but the paucity of data limited the extent of clinical guidance necessary for the field.
CONCLUSIONS: The systematic review showed that there are some clinical evidence displaying
the validity of psychopharmacological interventions in people with FASD, which varies across the spectrum of disease severity, age, and gender. There is a need for more clinical evidencebased studies in addition to clinical expert opinions to substantiate an optimal ground for individualized management of FASD.
The study protocol for this review was registered in PROSPERO with registration number
CRD42016045703
NAPCRG Pearls: What Is New? The top nine research studies that will impact clinical practice for family physicians as presented by Drs. David Kaplan and David White at Family Medicine Forum in Quebec City, QC Nov 2014
5 years of “Rare” Progress Research: Cheryl Rockman-Greenberg, Max Rady College of Medicine, University of Manitoba
Rare Disease Day Conference 2020 March 9-10
IRCM’s Centre on Rare and Genetic Diseases in Adults
Sophie Bernard, M.D., PhD.
Head of the Rare Disease Clinic, IRCM Director, Genetic Dyslipidemias Clinic, IRCM Assistant Professor, Department of Medicine, Montreal University
Rare Disease Day Conference 2020 March 9-10
The utility of psychotropic drugs on patients with fetal alcohol spectrum dis...BARRY STANLEY 2 fasd
ABSTRACT
BACKGROUND: Treatment of the complications arising from Prenatal Alcohol Exposure (PAE) has largely been focused on psychosocial and environmental approaches. Research on the
use of medications, especially psychotropic medications, has lagged behind.
OBJECTIVES: This systematic review sought to investigate psychotropic medication related findings and outcomes in those diagnosed with Fetal Alcohol Spectrum Disorder (FASD).
METHODS: Comprehensive searches were conducted in seven major databases (Medline/
PubMed, Scopus, Web of Knowledge, Embase, PsycINFO, Cochrane Library, and
PsycARTICLES) up to February 2017. Key search terms with synonyms were mapped on these databases. There were no timeline restrictions and no grey literature searches. Two reviewers
independently assessed 25 studies that met the inclusion criteria. Most studies were reviews of treatment and retrospective case series.
RESULTS: Two crossover randomized trials were reported, and the findings were not amenable to meta-analysis. Several conditions (depression, agitation, seizures, and outburst) combined with the most frequent presentation, ADHD, to represent the rationale for prescribing psychotropic medications. Second-generation antipsychotics were found to improve social skills, but the paucity of data limited the extent of clinical guidance necessary for the field.
CONCLUSIONS: The systematic review showed that there are some clinical evidence displaying
the validity of psychopharmacological interventions in people with FASD, which varies across the spectrum of disease severity, age, and gender. There is a need for more clinical evidencebased studies in addition to clinical expert opinions to substantiate an optimal ground for individualized management of FASD.
The study protocol for this review was registered in PROSPERO with registration number
CRD42016045703
NAPCRG Pearls: What Is New? The top nine research studies that will impact clinical practice for family physicians as presented by Drs. David Kaplan and David White at Family Medicine Forum in Quebec City, QC Nov 2014
LGS Foundation 2016 Conference - Saturday AfternoonLGS Foundation
Topics Include: Expecting to Transition - Reframing the Journey presented by Amy Miller, MSN, MA, PCNS-BC, ACHPN, Individual Education Program (IEPs) presented by Dena Hook, Telling Your Story presented by Patricia Moore and Dietary Therapies: Creative Strategies presented by Stephanie Criteser, RD, CSP and Natalie Cummings, RD
Scott Letendre, MD, of the UC San Diego HIV Neurobehavioral Research Program, presents "Overview of HIV & Aging" for AIDS Clinical Rounds at UC San Diego
Journal Club presentation on Outbreak Investigation Study Kunal Modak
The following presentation is based on: Concurrent Multiple Outbreaks of Varicella, Rubeola,
German Measles Outbreak in Unvaccinated Children of
Co-Educational Mount Carmel Senior Secondary School,
Thakurdwara Palampur of Northern Himachal, India
KCR’s Piotr Piotrowski, Magdalena Czarnecka and Anna Baran talk about placebos, a key component of many clinical trials, and the ethics behind, in the European Pharmaceutical Contractor (EPC) magazine, Autumn 2017.
Author: Lucille Sanzero Eller, PhD, RN
Associate Professor
Rutgers, The State University of New Jersey College of Nursing
A Local Performance Site of the NY/NJ AETC
See: http://AIDSETC.org
February 9, 2018
In the past several years, the United States has struggled to respond to viral outbreaks, such as Ebola and Zika. There is now an awareness of the need to rapidly develop vaccines and treatments for epidemics that can quickly spread from country to country. But questions remain as how to best conduct clinical trials and development of vaccines in the context of an epidemic or outbreak.
At this panel discussion, two health policy experts examined the appropriate conduct of clinical trials during public health emergencies.
Learn more at: http://petrieflom.law.harvard.edu/events/details/clinical-trials-during-public-health-emergencies
Pharmacovigilance presentation Workshop - Adam Kwan, B.Sc, Amjad Atrash, B.Sc Pharmacy, Lia Alderete, B.Sc MLT Valentyna Burbelo, M.Sc. Pharmaceutical Sciences, Professor Peivand Pirouzi
Do House Officers Learn from their Mistakes?
LGS Foundation 2016 Conference - Saturday AfternoonLGS Foundation
Topics Include: Expecting to Transition - Reframing the Journey presented by Amy Miller, MSN, MA, PCNS-BC, ACHPN, Individual Education Program (IEPs) presented by Dena Hook, Telling Your Story presented by Patricia Moore and Dietary Therapies: Creative Strategies presented by Stephanie Criteser, RD, CSP and Natalie Cummings, RD
Scott Letendre, MD, of the UC San Diego HIV Neurobehavioral Research Program, presents "Overview of HIV & Aging" for AIDS Clinical Rounds at UC San Diego
Journal Club presentation on Outbreak Investigation Study Kunal Modak
The following presentation is based on: Concurrent Multiple Outbreaks of Varicella, Rubeola,
German Measles Outbreak in Unvaccinated Children of
Co-Educational Mount Carmel Senior Secondary School,
Thakurdwara Palampur of Northern Himachal, India
KCR’s Piotr Piotrowski, Magdalena Czarnecka and Anna Baran talk about placebos, a key component of many clinical trials, and the ethics behind, in the European Pharmaceutical Contractor (EPC) magazine, Autumn 2017.
Author: Lucille Sanzero Eller, PhD, RN
Associate Professor
Rutgers, The State University of New Jersey College of Nursing
A Local Performance Site of the NY/NJ AETC
See: http://AIDSETC.org
February 9, 2018
In the past several years, the United States has struggled to respond to viral outbreaks, such as Ebola and Zika. There is now an awareness of the need to rapidly develop vaccines and treatments for epidemics that can quickly spread from country to country. But questions remain as how to best conduct clinical trials and development of vaccines in the context of an epidemic or outbreak.
At this panel discussion, two health policy experts examined the appropriate conduct of clinical trials during public health emergencies.
Learn more at: http://petrieflom.law.harvard.edu/events/details/clinical-trials-during-public-health-emergencies
Pharmacovigilance presentation Workshop - Adam Kwan, B.Sc, Amjad Atrash, B.Sc Pharmacy, Lia Alderete, B.Sc MLT Valentyna Burbelo, M.Sc. Pharmaceutical Sciences, Professor Peivand Pirouzi
Do House Officers Learn from their Mistakes?
MJFF Strategy for Accelerating Development of Parkinson’s TherapiesLaxmi Wordham
Research Roundtable hosted by Michael J. Fox Foundation in Chicago, Illinois on June 27, 2011 on the topic of Strategies for Accelerating Development of Parkinson’s Therapies.
NIH Research grants (R series) are an important funding mechanism for independent investigators as these awards offer the opportunity to head up major research projects.
In this presentation, Dr. Ian de Boer will leverage his experience from winning six different R awards to provide R series grant writing strategies. He explores: which R award is best for you; readiness and qualifications for independent support; formulating focused and solid research strategies; how to avoid common mistakes; tips for early investigators; and NIH expectations and grant requirements.
Slide presentation for the June 4, 2014 joint PCORI/ National Institute on Aging (NIA) of the National Institutes of Health webinar. This webinar announced the selection of the research team that will carry out a major, five-year, $30 million patient-centered study of the effectiveness of individually tailored care plans to help older individuals avoid falls and related injuries.
Co-design, Co-produce, Co-deliver: Collaboration is the only viable path to s...Simon R. Stones
In this presentation, delivered to the Translate external advisory board at their bi-annual meeting, the importance of patient and public involvement in research is highlighted, as well as simple strategies that researchers, healthcare professionals and private organisations can take to involve people in all aspects of research, from the bench to the bedside.
How evidence affects clinical practice in egyptWafaa Benjamin
Evidence based medicine is the gold standard for clinical care.
It implies the integration of best research evidence with clinical expertise and patient values.
There is still a wide gap between availability of evidence and its incorporation into routine practice in our country.
Barriers to implementation could be personal, social, institutional, financial and legal barriers.
True practice of evidence based care can only occur where evidence based decisions coincide with patients’ beliefs and clinicians’ preferences.
Continuing medical education programs should be set with integrating evidence based medicine teaching and learning within clinical training.
The importance of presence of local national guidelines which need to take into account variation in expertise, resources and patient preferences across our geographical and cultural contexts .
Customisation of a guideline to meet the local needs of a target patient population is critical to successful implementation.
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Avoiding Common Pitfalls in Cell and Gene Therapy TrialsMedpace
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Considerations for the Next Wave of COVID-19 DevelopmentMedpace
What did we learn from the first wave of COVID-specific studies that can be applied to those studies that will follow? Medpace has gathered some of its medical and operational team experts who have been on the front-line in designing and conducting trials for SARS-CoV-2 to answer some frequently asked questions, trends they see emerging and considerations for future development.
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n this free webinar, Medpace partners with Michelle Eagle of ATOM International, a provider of CE training for clinical trials across the world, to discuss approaches and steps that can be taken to ensure quality and integrity.
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In this webinar devoted to radiopharmaceuticals, the featured speakers will explore the scientific, operational and regulatory considerations for radiopharmaceuticals. With a focus on oncology, they will discuss the current regulatory landscape and how this impacts overall development programs. The speakers will explore the challenges of conducting radiopharmaceutical trials, offering insights into trial start-up, site selection and operational aspects to seamlessly execute these studies as part of clinical development plans.
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In this webinar, Medpace experts discuss key clinical, operational and laboratory considerations, lessons-learned, and best practices for accelerating the global development of safe and effective targeted therapeutics, using acute myeloid leukemia (AML) to highlight the complexities.
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In this webinar, we explore the regulatory implications of the RACE for Children Act and what this law means for your development program, particularly with navigating the change in requirements for pediatric oncology trials. Furthermore, we explore the challenges of executing oncology trials in pediatric populations and offer insight into design and operational aspects to seamlessly execute these studies as part of your clinical development plan
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Latin America: Challenges & Opportunities in Clinical ResearchMedpace
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This presentation discusses:
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Title: Sense of Smell
Presenter: Dr. Faiza, Assistant Professor of Physiology
Qualifications:
MBBS (Best Graduate, AIMC Lahore)
FCPS Physiology
ICMT, CHPE, DHPE (STMU)
MPH (GC University, Faisalabad)
MBA (Virtual University of Pakistan)
Learning Objectives:
Describe the primary categories of smells and the concept of odor blindness.
Explain the structure and location of the olfactory membrane and mucosa, including the types and roles of cells involved in olfaction.
Describe the pathway and mechanisms of olfactory signal transmission from the olfactory receptors to the brain.
Illustrate the biochemical cascade triggered by odorant binding to olfactory receptors, including the role of G-proteins and second messengers in generating an action potential.
Identify different types of olfactory disorders such as anosmia, hyposmia, hyperosmia, and dysosmia, including their potential causes.
Key Topics:
Olfactory Genes:
3% of the human genome accounts for olfactory genes.
400 genes for odorant receptors.
Olfactory Membrane:
Located in the superior part of the nasal cavity.
Medially: Folds downward along the superior septum.
Laterally: Folds over the superior turbinate and upper surface of the middle turbinate.
Total surface area: 5-10 square centimeters.
Olfactory Mucosa:
Olfactory Cells: Bipolar nerve cells derived from the CNS (100 million), with 4-25 olfactory cilia per cell.
Sustentacular Cells: Produce mucus and maintain ionic and molecular environment.
Basal Cells: Replace worn-out olfactory cells with an average lifespan of 1-2 months.
Bowman’s Gland: Secretes mucus.
Stimulation of Olfactory Cells:
Odorant dissolves in mucus and attaches to receptors on olfactory cilia.
Involves a cascade effect through G-proteins and second messengers, leading to depolarization and action potential generation in the olfactory nerve.
Quality of a Good Odorant:
Small (3-20 Carbon atoms), volatile, water-soluble, and lipid-soluble.
Facilitated by odorant-binding proteins in mucus.
Membrane Potential and Action Potential:
Resting membrane potential: -55mV.
Action potential frequency in the olfactory nerve increases with odorant strength.
Adaptation Towards the Sense of Smell:
Rapid adaptation within the first second, with further slow adaptation.
Psychological adaptation greater than receptor adaptation, involving feedback inhibition from the central nervous system.
Primary Sensations of Smell:
Camphoraceous, Musky, Floral, Pepperminty, Ethereal, Pungent, Putrid.
Odor Detection Threshold:
Examples: Hydrogen sulfide (0.0005 ppm), Methyl-mercaptan (0.002 ppm).
Some toxic substances are odorless at lethal concentrations.
Characteristics of Smell:
Odor blindness for single substances due to lack of appropriate receptor protein.
Behavioral and emotional influences of smell.
Transmission of Olfactory Signals:
From olfactory cells to glomeruli in the olfactory bulb, involving lateral inhibition.
Primitive, less old, and new olfactory systems with different path
ARTIFICIAL INTELLIGENCE IN HEALTHCARE.pdfAnujkumaranit
Artificial intelligence (AI) refers to the simulation of human intelligence processes by machines, especially computer systems. It encompasses tasks such as learning, reasoning, problem-solving, perception, and language understanding. AI technologies are revolutionizing various fields, from healthcare to finance, by enabling machines to perform tasks that typically require human intelligence.
Explore natural remedies for syphilis treatment in Singapore. Discover alternative therapies, herbal remedies, and lifestyle changes that may complement conventional treatments. Learn about holistic approaches to managing syphilis symptoms and supporting overall health.
- Video recording of this lecture in English language: https://youtu.be/lK81BzxMqdo
- Video recording of this lecture in Arabic language: https://youtu.be/Ve4P0COk9OI
- Link to download the book free: https://nephrotube.blogspot.com/p/nephrotube-nephrology-books.html
- Link to NephroTube website: www.NephroTube.com
- Link to NephroTube social media accounts: https://nephrotube.blogspot.com/p/join-nephrotube-on-social-media.html
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These lecture slides, by Dr Sidra Arshad, offer a quick overview of physiological basis of a normal electrocardiogram.
Learning objectives:
1. Define an electrocardiogram (ECG) and electrocardiography
2. Describe how dipoles generated by the heart produce the waveforms of the ECG
3. Describe the components of a normal electrocardiogram of a typical bipolar leads (limb II)
4. Differentiate between intervals and segments
5. Enlist some common indications for obtaining an ECG
Study Resources:
1. Chapter 11, Guyton and Hall Textbook of Medical Physiology, 14th edition
2. Chapter 9, Human Physiology - From Cells to Systems, Lauralee Sherwood, 9th edition
3. Chapter 29, Ganong’s Review of Medical Physiology, 26th edition
4. Electrocardiogram, StatPearls - https://www.ncbi.nlm.nih.gov/books/NBK549803/
5. ECG in Medical Practice by ABM Abdullah, 4th edition
6. ECG Basics, http://www.nataliescasebook.com/tag/e-c-g-basics
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Tom Selleck Health: A Comprehensive Look at the Iconic Actor’s Wellness Journeygreendigital
Tom Selleck, an enduring figure in Hollywood. has captivated audiences for decades with his rugged charm, iconic moustache. and memorable roles in television and film. From his breakout role as Thomas Magnum in Magnum P.I. to his current portrayal of Frank Reagan in Blue Bloods. Selleck's career has spanned over 50 years. But beyond his professional achievements. fans have often been curious about Tom Selleck Health. especially as he has aged in the public eye.
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Introduction
Many have been interested in Tom Selleck health. not only because of his enduring presence on screen but also because of the challenges. and lifestyle choices he has faced and made over the years. This article delves into the various aspects of Tom Selleck health. exploring his fitness regimen, diet, mental health. and the challenges he has encountered as he ages. We'll look at how he maintains his well-being. the health issues he has faced, and his approach to ageing .
Early Life and Career
Childhood and Athletic Beginnings
Tom Selleck was born on January 29, 1945, in Detroit, Michigan, and grew up in Sherman Oaks, California. From an early age, he was involved in sports, particularly basketball. which played a significant role in his physical development. His athletic pursuits continued into college. where he attended the University of Southern California (USC) on a basketball scholarship. This early involvement in sports laid a strong foundation for his physical health and disciplined lifestyle.
Transition to Acting
Selleck's transition from an athlete to an actor came with its physical demands. His first significant role in "Magnum P.I." required him to perform various stunts and maintain a fit appearance. This role, which he played from 1980 to 1988. necessitated a rigorous fitness routine to meet the show's demands. setting the stage for his long-term commitment to health and wellness.
Fitness Regimen
Workout Routine
Tom Selleck health and fitness regimen has evolved. adapting to his changing roles and age. During his "Magnum, P.I." days. Selleck's workouts were intense and focused on building and maintaining muscle mass. His routine included weightlifting, cardiovascular exercises. and specific training for the stunts he performed on the show.
Selleck adjusted his fitness routine as he aged to suit his body's needs. Today, his workouts focus on maintaining flexibility, strength, and cardiovascular health. He incorporates low-impact exercises such as swimming, walking, and light weightlifting. This balanced approach helps him stay fit without putting undue strain on his joints and muscles.
Importance of Flexibility and Mobility
In recent years, Selleck has emphasized the importance of flexibility and mobility in his fitness regimen. Understanding the natural decline in muscle mass and joint flexibility with age. he includes stretching and yoga in his routine. These practices help prevent injuries, improve posture, and maintain mobilit
Ethanol (CH3CH2OH), or beverage alcohol, is a two-carbon alcohol
that is rapidly distributed in the body and brain. Ethanol alters many
neurochemical systems and has rewarding and addictive properties. It
is the oldest recreational drug and likely contributes to more morbidity,
mortality, and public health costs than all illicit drugs combined. The
5th edition of the Diagnostic and Statistical Manual of Mental Disorders
(DSM-5) integrates alcohol abuse and alcohol dependence into a single
disorder called alcohol use disorder (AUD), with mild, moderate,
and severe subclassifications (American Psychiatric Association, 2013).
In the DSM-5, all types of substance abuse and dependence have been
combined into a single substance use disorder (SUD) on a continuum
from mild to severe. A diagnosis of AUD requires that at least two of
the 11 DSM-5 behaviors be present within a 12-month period (mild
AUD: 2–3 criteria; moderate AUD: 4–5 criteria; severe AUD: 6–11 criteria).
The four main behavioral effects of AUD are impaired control over
drinking, negative social consequences, risky use, and altered physiological
effects (tolerance, withdrawal). This chapter presents an overview
of the prevalence and harmful consequences of AUD in the U.S.,
the systemic nature of the disease, neurocircuitry and stages of AUD,
comorbidities, fetal alcohol spectrum disorders, genetic risk factors, and
pharmacotherapies for AUD.
Webinar: Getting Treatment Options to Rare Disease Patients Faster: Putting Patients First in Clinical Trials
1. CLICK TO EDIT
TITLE
Click to edit subtitle
[Insert Sponsor logo or delete]
Clinical Operations – ask the Design Assistant for assistance inserting
2. Optimizing Patient Recruitment
to Accelerate Research Today –
and Trends for Tomorrow
John Lynn Jefferies, MD, MPH,
FACC, FAHA, FHFSA
Director, Advanced Heart Failure
and Cardiomyopathy Programs,
Cincinnati Children’s Hospital
Medical Center
3. o Studies for drug and
device approval by
the Food and Drug
Administration (FDA)
rely on patient data
for safety and
efficacy
Optimizing Patient Recruitment
Therapeutically-Focused | Scientifically-Driven3
o Patient recruitment is critical for clinical research
o Many adult trials involve thousands of participants
4. o Rare disease studies have an advantage that
smaller numbers of patients are required by the
FDA for approval
Patient Recruitment: Rare Disease
Therapeutically-Focused | Scientifically-Driven4
o However, rare disease
patients are typically
spread out all over the
globe making access
to them for trials
challenging
5. o Many rare diseases
have epicenters of
care
o Institutions that have
a reputation for
being national or
international leaders
in the care of the
specific condition
Rare Disease Awareness
Therapeutically-Focused | Scientifically-Driven5
o However, many patients that may want to
participate in research are not aware of clinical
studies
6. o In the past, patients not seen at centers
performing the research would not have access to
participation
Increasing Awareness with Technology
Therapeutically-Focused | Scientifically-Driven6
o Developments in
technology and
changes in trial
designs now allow for
larger numbers of
patients and families
to be made aware of
clinical investigations
7. o Use of the internet and other mechanisms of
communication have greatly enhanced awareness
of studies
Increasing Awareness with Technology
Therapeutically-Focused | Scientifically-Driven7
Facebook and
other social media
Hand held
technologies
Patient advocacy
groups
8. o Patient self-referral has also added to trial
participation
Participants Take Control
Therapeutically-Focused | Scientifically-Driven8
o Many families will find
out about clinical
trials through various
mechanisms and go
to the centers doing
the research to
participate
9. o Prior research in rare diseases has typically been
done at single institutions
o This is challenging as it may be hard for families
to get to that center
Increasing Access
Therapeutically-Focused | Scientifically-Driven9
o Recent years have
seen the development
of multicenter trials
and collaborative
groups which facilitate
more options for
patients and families
10. o The future of trials and recruitment is very open
and encouraging
The Future of Recruitment
Therapeutically-Focused | Scientifically-Driven10
o As technologies continue to
improve and become more
available, the awareness of
clinical studies will be more
widespread allowing families
to prioritize which studies they
would like to participate in
moving forward
11. Bespoke Trial Design
and Patient Centricity
Michelle Petersen, MS
Associate Director, Clinical
Trial Management
12. o Rare disease requires strategic trial design as the
number of subjects to enroll are limited
Studies require patient input to ensure the design is
appropriate and includes what is meaningful to the patient
Collect samples at different time points in different patients
Crossover trial design is one way to increase data
collection in a limited patient population but only works in
ideal circumstances
Bespoke Trial Design
Therapeutically-Focused | Scientifically-Driven12
13. Therapeutically-Focused | Scientifically-Driven
o Hub and spoke trial design
for a hub site for complex
visits and spoke sites for
minor procedures or
dosing
Commit to opening
spoke sites in extension
studies
o Home healthcare
compliant visits and home
delivery of IP
o Increase time between
visits as study duration
increases
o Visit window flexibility
o Request site to extend
hours of service and offer
weekend appointments
o Travel/reimbursement
support
Concierge level service
to address challenges of
study participation and
remove financial burden
Support family (i.e.
considering childcare or
schooling of young
patients)
Retention Key: Burden Reduction
13
Complex endpoints can reduce the number of centers able to
participate which often increases subject burden
To Reduce This Impact Consider:
14. Therapeutically-Focused | Scientifically-Driven
o Get patient input of trial design, informed consent
process and what would improve the trial
o Avenues for patient insight:
Advocacy groups
Patient panels
International and regional patient meetings
Patient Input
14
o Keep updating the
community on progress
Webinar
Press releases
Sending results overview
in layman’s terms
16. o The increased use of CROs in rare disease
clinical research has enhanced participation
o CROs have numerous touchpoints across the
globe at leading institutions
Repeated collaboration can lead to enhanced
startup
o CROs also work with leaders in the field of the
particular rare disease to gain additional insight
into trial design and patient/family perspectives
CRO and Site Collaboration
Therapeutically-Focused | Scientifically-Driven16
17. A Family’s Story
Beth Woelfel Harvey
Executive Director, JB’s Keys to DMD
Mother of child with Duchenne
Muscular Dystrophy
18. JB Harvey
Diagnosed in 2009 at 16 months with DMD
JB one month before diagnosis
JB age 2JB walking on the beach age 3
19. Therapeutically-Focused | Scientifically-Driven
o No standards of care
o 3 clinical trials
o 3 companies
developing drugs
o Recently updated
standards of care
o Over 30 current
trials
o Over 40 companies
developing drugs
2009 vs. 2018
19
2009 2018
20. Therapeutically-Focused | Scientifically-Driven
o Trial #1
1 visit Age 2
DXA scan and function testing
o Trial #2
10 visits over 2 years, Age 6-9
Imaging and functional testing
o Trial #3
8 visits over 2 years from Age
7-9
Thigh MRI, labs, functional
testing, Echo, PFT, eye exam
o Trial #4
Over 20 visits in first year Age
9-10
Thigh MRI, Cardiac MRI, Liver
MRI, X-rays, labs, Echo,
Functional Testing, DXA
JB’s Clinical Trial Experience
20
Trial #3, 2015
Trial #4, 2016
21. Therapeutically-Focused | Scientifically-Driven
For children to participate in clinical trials they
must have stable mental health and be happy!
Mental Health
21
JB after skiing 20 runs
JB after his musical
performance
JB sailing
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GL-GN-15-SA | Version 1 (General PowerPoint Template)
PICTURES ARE SUBJECT TO COPYRIGHT. Do not insert images from the internet.
Review Medpace BP GL-GN-15 for more information.
PICTURES ARE SUBJECT TO COPYRIGHT. Do not insert images from the internet.
Review Medpace BP GL-GN-15 for more information.
PICTURES ARE SUBJECT TO COPYRIGHT. Do not insert images from the internet.
Review Medpace BP GL-GN-15 for more information.