Rare Disease Centres of Excellence Webinar May 5, 2022
Christopher McMaster/Etienne Richer, CIHR Institute of Genetics
Craig Campbell, Department of Pediatrics, Western University
Domenica Talarico, European Reference Networks
Matt Bolz-Johnson, WHO-RDI Global Rare Disease Network
Rare Disease Patient Registries:
Key to Drug Development and Access
Tuesday, May 3 @ 12:00 – 1:00 pm EDT
Slides:
USA National Institutes of Health RaDaR
NORD IAMRARE (Pam Gavin, VP)
Canada INFORM RARE (Beth Potter, CHEO; Pranesh Chakraborty, CHEO; Kim Angel, Can MPS Society; John Adams, CanPKU)
Universal Health Care: Perceptions, Values, and IssuesRenzo Guinto
From the workshop "Universal Health Care: The First Step to Global Health Equity" held last August 5-9, 2012 in Mumbai, India during the 61st General Assembly March Meeting of the International Federation of Medical Students' Associations (IFMSA). Brought to you by the IFMSA Global Health Equity Initiative (http://www.ifmsa.org/Activities/Initiatives/The-IFMSA-Global-Health-Equity-Initiative).
For more information about the workshop, visit http://www.scribd.com/doc/193822108/Universal-Health-Care-PreGA-Program
The global patient engagement solutions market size stood at around USD 11.8 billion in 2019 and is projected to reach USD xx billion by 2028, exhibiting a CAGR of xx% during the forecast period.
The market for patient engagement solutions is mainly driven by the increasing utilization of electronic health records for patient-centric care and the adoption of mobile health devices. According to a survey conducted by the NHS in 2019, approximately 92.0% of patients feel assured in self-managing treatment.
Market Drivers
Factors such as the growing burden of chronic diseases and a rise in the geriatric population have led to the adoption of patient engagement solutions worldwide. For instance, in 2017, according to CDC statistics, 1.3 million new cases of diabetes were recorded in the US alone. In response to the growing number of diabetic patients, companies are focusing on the development of patient-centric engagement solutions. For example, in June 2019, Allina Health launched a patient-centric engagement solution platform for improving the Diabetes Self-Management Education (DSME) program. This enables diabetic self-care clinical services, particularly for those located in remote locations. Moreover, programs such as these help in cost evaluation, operational effectiveness, and clinical outcomes.
Government support is expected to drive the adoption of patient engagement solutions in the coming years. For instance, in 2018, the FDA established the Patient and Caregiver Connection program. The program was aimed to foster engagement with patients, patient advocates, and caregivers throughout the evaluation and surveillance of medical devices.
Visit https://insights10.com/ for more healthcare industry insights.
Connect with us @ info@insights10.com
Frugal Innovation is about doing more with less. Entrepreneurs and innovators in emerging markets have to devise low cost strategies to either tap or circumvent institutional complexities and resource limitations to innovate, develop and deliver products and services to low income users with little purchasing power, often at mass scale and arguably in a sustainable manner. My DPhil research seeks to understand this institutionally complex, constraint-based, and low cost innovation which is designed in or for emerging market contexts.
Rare Disease Centres of Excellence Webinar May 5, 2022
Christopher McMaster/Etienne Richer, CIHR Institute of Genetics
Craig Campbell, Department of Pediatrics, Western University
Domenica Talarico, European Reference Networks
Matt Bolz-Johnson, WHO-RDI Global Rare Disease Network
Rare Disease Patient Registries:
Key to Drug Development and Access
Tuesday, May 3 @ 12:00 – 1:00 pm EDT
Slides:
USA National Institutes of Health RaDaR
NORD IAMRARE (Pam Gavin, VP)
Canada INFORM RARE (Beth Potter, CHEO; Pranesh Chakraborty, CHEO; Kim Angel, Can MPS Society; John Adams, CanPKU)
Universal Health Care: Perceptions, Values, and IssuesRenzo Guinto
From the workshop "Universal Health Care: The First Step to Global Health Equity" held last August 5-9, 2012 in Mumbai, India during the 61st General Assembly March Meeting of the International Federation of Medical Students' Associations (IFMSA). Brought to you by the IFMSA Global Health Equity Initiative (http://www.ifmsa.org/Activities/Initiatives/The-IFMSA-Global-Health-Equity-Initiative).
For more information about the workshop, visit http://www.scribd.com/doc/193822108/Universal-Health-Care-PreGA-Program
The global patient engagement solutions market size stood at around USD 11.8 billion in 2019 and is projected to reach USD xx billion by 2028, exhibiting a CAGR of xx% during the forecast period.
The market for patient engagement solutions is mainly driven by the increasing utilization of electronic health records for patient-centric care and the adoption of mobile health devices. According to a survey conducted by the NHS in 2019, approximately 92.0% of patients feel assured in self-managing treatment.
Market Drivers
Factors such as the growing burden of chronic diseases and a rise in the geriatric population have led to the adoption of patient engagement solutions worldwide. For instance, in 2017, according to CDC statistics, 1.3 million new cases of diabetes were recorded in the US alone. In response to the growing number of diabetic patients, companies are focusing on the development of patient-centric engagement solutions. For example, in June 2019, Allina Health launched a patient-centric engagement solution platform for improving the Diabetes Self-Management Education (DSME) program. This enables diabetic self-care clinical services, particularly for those located in remote locations. Moreover, programs such as these help in cost evaluation, operational effectiveness, and clinical outcomes.
Government support is expected to drive the adoption of patient engagement solutions in the coming years. For instance, in 2018, the FDA established the Patient and Caregiver Connection program. The program was aimed to foster engagement with patients, patient advocates, and caregivers throughout the evaluation and surveillance of medical devices.
Visit https://insights10.com/ for more healthcare industry insights.
Connect with us @ info@insights10.com
Frugal Innovation is about doing more with less. Entrepreneurs and innovators in emerging markets have to devise low cost strategies to either tap or circumvent institutional complexities and resource limitations to innovate, develop and deliver products and services to low income users with little purchasing power, often at mass scale and arguably in a sustainable manner. My DPhil research seeks to understand this institutionally complex, constraint-based, and low cost innovation which is designed in or for emerging market contexts.
Health technology assessment (HTA) is familiar as technique for gauging the value of specific medical technologies or approaches to care. As Adrian Towse points out, however, HTA has a much broader, ‘macro’ role in contributing to the efficiency of health care systems and supporting universal health coverage. This is particularly crucial in the face of increasing demands and limited budgets.
May 19: Leave No One Behind
Panelists
Brad Alyward, Head Market Access & Health Policy, Indivior
Catherine Boivin, Patient Advocate, CORD
Shona Kinley,Director, Federal Policy & Government Affairs, Novartis
Bennett Lee, Head, Value & Access, Sanofi
Joan Paulin, Patient Advocate, PHA Canada
Trevor Richter, Director of Access and Reimbursement, Gilead
MediMeals Investor Presentation - February 2017Cory Glazier
Clinical trials have proven irrevocably that heart disease and diabetes can be reversed through deliberate nutritional therapy with a shift to consuming a whole food, plant-based diet.
MediMeals is an evolutionary health service that makes it as easy for doctors to prescribe scientifically proven meal regimens as it has been to prescribe pharmaceuticals and surgery in the past.
We get nutritionally precise, delicious meals to our patients nationwide, on doctor's orders.
We have reduced the learning curve and time constraints to upgrading diet. Healing the body with food has never been as accessible.
July 22, 2021 : Aligning on Rare Drug Strategy, PMPRB, and Federal Election
Engage with our Expert Panel in a Townhall style meeting to add your voice on these important issues in preparation for upcoming federal election.
Panelist
Alexandra Chambers (Novartis)
Peter Brenders (BeiGene)
Angela Genge (The Neuro)
Fred Horne (3Sixty Public Affairs)
Leanne Ward (CHEO)
The Future of Personalizing Care Management & the Patient ExperienceRaphael Louis Vitón
Actionable segmentation model findings - by Raphael Louis Vitón & Dream team of industry experts, physicians and leaders from Blue Cross, GEHealthCare, RingLeaderVentures, Maddock Douglas, Dr.Daniel Friedland, etc working on improving health outcomes by Personalizing the Care Management business model for Better Outcomes & Better Economics (through patient empowerment)
Why Precision Medicine and Personalized Healthcare and Why Now? Links to Canada’s Rare Disease Strategy The Future is Now
1) Marc LePage, Genome Canada
2) Michael Duong, Roche
3) Danica Stanimirovic, National Research Council
4) Daniel Gaudet, University of Montreal
5) Christine Dalgleish, Patient Perspective
6) Jamie Bruce, Khure Health
Successfully Managing Chronic Health Conditions with Human-Centered Digital T...Cognizant
By adopting evidence-based digital therapeutics, healthcare organizations can alleviate the mounting costs of caring for patients with chronic conditions — and enter the fourth wave of digital healthcare.
Effective Patient Stratification: Four Solutions to Common HurdlesHealth Catalyst
Accurate patient stratification, the first step of any effective population health strategy, identifies patients who will benefit most from a population health intervention. Successful patient stratification is critical when laying the foundation for any population health initiative, yet many health systems struggle with this step.
Care teams can apply four solutions to overcome common patient stratification hurdles, target the most impactable patients, and carry out population health initiatives:
Consider both the physical and the mental.
Prove and measure return on investment.
Complete data sets.
Transparent, customizable technology.
The mission of the program is to sensitize the elderly about how they could get access to their medicine. The primary goal is to ensure that older adults are living well by getting access to their medicines when they want them depending on their condition
This e-book focuses on Health Management Solutions the value it adds alongside other systems that are already in place throughout the care lifecycle...
Rock Report: Personalization in Consumer Health by @Rock_HealthRock Health
Overview of personalization in healthcare, including opportunities, barriers and case studies related to a market estimated to reach $450B+ by 2015. Purchase the report here: https://gumroad.com/l/XxcA
1.Write an essay discussing the various causes and solutions for aBenitoSumpter862
1.Write an essay discussing the various causes and solutions for a patient’s inability to pay for medications (prescriptions).
Your response should be at least 200 words in length
2. “Front desk syndrome” is a particularly troublesome aspect of patients visiting their healthcare provider. Write an essay describing this phenomena and methods to prevent or decrease its occurrence.
Your response should be at least 200 words in length.
Inmate Manipulation and Boundaries
Nicole Jones, Dionne Russell, Francisco Villegas, Lupe Silva
University of Phoenix
CPSS/430
Dr. Debra McCoy
May 16, 2022
What to Expect from This Presentation
Summary of manipulation scenario
Development of a plan for how to maintain boundaries in this situation
Possible obstacles in setting and maintaining these boundaries
Identifying ethical and legal consequences to not maintaining boundaries in this situation
Manipulation Scenario
Patricia Wilkes works as a correctional officer at Corcoran California state prison. Corcoran is a high security institution that houses some of the most violent male offenders. Patricia has worked at the institution for four years and has encountered two attacks against her from two separate inmates over a year span. Patricia is freshly returned back to work after being off for a week due to her injuries from her latest attack. The attack involved her being pushed down by an inmate, causing a sprain arm. Feeling vulnerable Patricia became friends with an inmate who witnessed the attack and promised to issue protection if she sneaks in contraband for him. She is conflicted but, because she is afraid; she agrees to do it.
How to Maintain Boundaries in this Situation
How to Maintain Boundaries in this Situation (continued)
How to Maintain Boundaries in this Situation (continued)
Possible Obstacles in Setting and Maintaining these Boundaries
Possible Obstacles in Setting and Maintaining these Boundaries (continued)
Possible Obstacles in Setting and Maintaining these Boundaries (continued)
Ethical and Legal Consequences to not Maintaining Boundaries in this Situation
Ethical and Legal Consequences to not Maintaining Boundaries in this Situation (continued)
Ethical and Legal Consequences to not Maintaining Boundaries in this Situation (continued)
Questions
References
HCA 4303, Comparative Health Systems 1
Course Learning Outcomes for Unit I
Upon completion of this unit, students should be able to:
1. Identify and explain how demographics, such as age and race, impact true access to quality care.
Reading Assignment
Chapter 1: Introduction
Chapter 2: Disparities in Health Care: Race and Age Matters
Unit Lesson
Many Americans find it difficult to even comprehend being denied access to medical care. When we are sick,
we want to see a physician, promptly receive a diagnosis, and procure the necessary treatment that will
restore us to our previous level of health. T ...
Health technology assessment (HTA) is familiar as technique for gauging the value of specific medical technologies or approaches to care. As Adrian Towse points out, however, HTA has a much broader, ‘macro’ role in contributing to the efficiency of health care systems and supporting universal health coverage. This is particularly crucial in the face of increasing demands and limited budgets.
May 19: Leave No One Behind
Panelists
Brad Alyward, Head Market Access & Health Policy, Indivior
Catherine Boivin, Patient Advocate, CORD
Shona Kinley,Director, Federal Policy & Government Affairs, Novartis
Bennett Lee, Head, Value & Access, Sanofi
Joan Paulin, Patient Advocate, PHA Canada
Trevor Richter, Director of Access and Reimbursement, Gilead
MediMeals Investor Presentation - February 2017Cory Glazier
Clinical trials have proven irrevocably that heart disease and diabetes can be reversed through deliberate nutritional therapy with a shift to consuming a whole food, plant-based diet.
MediMeals is an evolutionary health service that makes it as easy for doctors to prescribe scientifically proven meal regimens as it has been to prescribe pharmaceuticals and surgery in the past.
We get nutritionally precise, delicious meals to our patients nationwide, on doctor's orders.
We have reduced the learning curve and time constraints to upgrading diet. Healing the body with food has never been as accessible.
July 22, 2021 : Aligning on Rare Drug Strategy, PMPRB, and Federal Election
Engage with our Expert Panel in a Townhall style meeting to add your voice on these important issues in preparation for upcoming federal election.
Panelist
Alexandra Chambers (Novartis)
Peter Brenders (BeiGene)
Angela Genge (The Neuro)
Fred Horne (3Sixty Public Affairs)
Leanne Ward (CHEO)
The Future of Personalizing Care Management & the Patient ExperienceRaphael Louis Vitón
Actionable segmentation model findings - by Raphael Louis Vitón & Dream team of industry experts, physicians and leaders from Blue Cross, GEHealthCare, RingLeaderVentures, Maddock Douglas, Dr.Daniel Friedland, etc working on improving health outcomes by Personalizing the Care Management business model for Better Outcomes & Better Economics (through patient empowerment)
Why Precision Medicine and Personalized Healthcare and Why Now? Links to Canada’s Rare Disease Strategy The Future is Now
1) Marc LePage, Genome Canada
2) Michael Duong, Roche
3) Danica Stanimirovic, National Research Council
4) Daniel Gaudet, University of Montreal
5) Christine Dalgleish, Patient Perspective
6) Jamie Bruce, Khure Health
Successfully Managing Chronic Health Conditions with Human-Centered Digital T...Cognizant
By adopting evidence-based digital therapeutics, healthcare organizations can alleviate the mounting costs of caring for patients with chronic conditions — and enter the fourth wave of digital healthcare.
Effective Patient Stratification: Four Solutions to Common HurdlesHealth Catalyst
Accurate patient stratification, the first step of any effective population health strategy, identifies patients who will benefit most from a population health intervention. Successful patient stratification is critical when laying the foundation for any population health initiative, yet many health systems struggle with this step.
Care teams can apply four solutions to overcome common patient stratification hurdles, target the most impactable patients, and carry out population health initiatives:
Consider both the physical and the mental.
Prove and measure return on investment.
Complete data sets.
Transparent, customizable technology.
The mission of the program is to sensitize the elderly about how they could get access to their medicine. The primary goal is to ensure that older adults are living well by getting access to their medicines when they want them depending on their condition
This e-book focuses on Health Management Solutions the value it adds alongside other systems that are already in place throughout the care lifecycle...
Rock Report: Personalization in Consumer Health by @Rock_HealthRock Health
Overview of personalization in healthcare, including opportunities, barriers and case studies related to a market estimated to reach $450B+ by 2015. Purchase the report here: https://gumroad.com/l/XxcA
1.Write an essay discussing the various causes and solutions for aBenitoSumpter862
1.Write an essay discussing the various causes and solutions for a patient’s inability to pay for medications (prescriptions).
Your response should be at least 200 words in length
2. “Front desk syndrome” is a particularly troublesome aspect of patients visiting their healthcare provider. Write an essay describing this phenomena and methods to prevent or decrease its occurrence.
Your response should be at least 200 words in length.
Inmate Manipulation and Boundaries
Nicole Jones, Dionne Russell, Francisco Villegas, Lupe Silva
University of Phoenix
CPSS/430
Dr. Debra McCoy
May 16, 2022
What to Expect from This Presentation
Summary of manipulation scenario
Development of a plan for how to maintain boundaries in this situation
Possible obstacles in setting and maintaining these boundaries
Identifying ethical and legal consequences to not maintaining boundaries in this situation
Manipulation Scenario
Patricia Wilkes works as a correctional officer at Corcoran California state prison. Corcoran is a high security institution that houses some of the most violent male offenders. Patricia has worked at the institution for four years and has encountered two attacks against her from two separate inmates over a year span. Patricia is freshly returned back to work after being off for a week due to her injuries from her latest attack. The attack involved her being pushed down by an inmate, causing a sprain arm. Feeling vulnerable Patricia became friends with an inmate who witnessed the attack and promised to issue protection if she sneaks in contraband for him. She is conflicted but, because she is afraid; she agrees to do it.
How to Maintain Boundaries in this Situation
How to Maintain Boundaries in this Situation (continued)
How to Maintain Boundaries in this Situation (continued)
Possible Obstacles in Setting and Maintaining these Boundaries
Possible Obstacles in Setting and Maintaining these Boundaries (continued)
Possible Obstacles in Setting and Maintaining these Boundaries (continued)
Ethical and Legal Consequences to not Maintaining Boundaries in this Situation
Ethical and Legal Consequences to not Maintaining Boundaries in this Situation (continued)
Ethical and Legal Consequences to not Maintaining Boundaries in this Situation (continued)
Questions
References
HCA 4303, Comparative Health Systems 1
Course Learning Outcomes for Unit I
Upon completion of this unit, students should be able to:
1. Identify and explain how demographics, such as age and race, impact true access to quality care.
Reading Assignment
Chapter 1: Introduction
Chapter 2: Disparities in Health Care: Race and Age Matters
Unit Lesson
Many Americans find it difficult to even comprehend being denied access to medical care. When we are sick,
we want to see a physician, promptly receive a diagnosis, and procure the necessary treatment that will
restore us to our previous level of health. T ...
1.Write an essay discussing the various causes and solutions for aSantosConleyha
1.Write an essay discussing the various causes and solutions for a patient’s inability to pay for medications (prescriptions).
Your response should be at least 200 words in length
2. “Front desk syndrome” is a particularly troublesome aspect of patients visiting their healthcare provider. Write an essay describing this phenomena and methods to prevent or decrease its occurrence.
Your response should be at least 200 words in length.
Inmate Manipulation and Boundaries
Nicole Jones, Dionne Russell, Francisco Villegas, Lupe Silva
University of Phoenix
CPSS/430
Dr. Debra McCoy
May 16, 2022
What to Expect from This Presentation
Summary of manipulation scenario
Development of a plan for how to maintain boundaries in this situation
Possible obstacles in setting and maintaining these boundaries
Identifying ethical and legal consequences to not maintaining boundaries in this situation
Manipulation Scenario
Patricia Wilkes works as a correctional officer at Corcoran California state prison. Corcoran is a high security institution that houses some of the most violent male offenders. Patricia has worked at the institution for four years and has encountered two attacks against her from two separate inmates over a year span. Patricia is freshly returned back to work after being off for a week due to her injuries from her latest attack. The attack involved her being pushed down by an inmate, causing a sprain arm. Feeling vulnerable Patricia became friends with an inmate who witnessed the attack and promised to issue protection if she sneaks in contraband for him. She is conflicted but, because she is afraid; she agrees to do it.
How to Maintain Boundaries in this Situation
How to Maintain Boundaries in this Situation (continued)
How to Maintain Boundaries in this Situation (continued)
Possible Obstacles in Setting and Maintaining these Boundaries
Possible Obstacles in Setting and Maintaining these Boundaries (continued)
Possible Obstacles in Setting and Maintaining these Boundaries (continued)
Ethical and Legal Consequences to not Maintaining Boundaries in this Situation
Ethical and Legal Consequences to not Maintaining Boundaries in this Situation (continued)
Ethical and Legal Consequences to not Maintaining Boundaries in this Situation (continued)
Questions
References
HCA 4303, Comparative Health Systems 1
Course Learning Outcomes for Unit I
Upon completion of this unit, students should be able to:
1. Identify and explain how demographics, such as age and race, impact true access to quality care.
Reading Assignment
Chapter 1: Introduction
Chapter 2: Disparities in Health Care: Race and Age Matters
Unit Lesson
Many Americans find it difficult to even comprehend being denied access to medical care. When we are sick,
we want to see a physician, promptly receive a diagnosis, and procure the necessary treatment that will
restore us to our previous level of health. T ...
mHealth Israel_GEARING COMMUNICATIONS TO RAISE CAPITAL AND ATTRACT CUSTOMERS_...Levi Shapiro
Presentation by Gil Bashe, Managing Director, Healthcare Practice, Finn Partners: "GEARING COMMUNICATIONS TO RAISE CAPITAL AND ATTRACT CUSTOMERS- FROM PLAN TO PARTNERS TO PATIENTS". Includes tips to avoid failure by embracing complexity, description of the Health Ecosystem Landscape, developing a plan to impact care, cost and outcomes, overview of the US Payer market, and top digital health influencers.
On this webinar, we’ll hear from experts on the issue and invite an open conversation with stakeholders. We need discussion, shared questions and answers and a review of case studies, which is why we are hosting this session.
Panelist:
Neil Palmer, Principal Consultant, WN Palmer & Co. and former PMPRB staff
Michael Dietrich, Executive Director, Policy, Innovative Medicines Canada
Laurene Redding, Global Head, Strategic Pricing (ex-China), BeiGene
Durhane Wong-Rieger, President & CEO, CORD
Moderator: Bill Dempster, CEO, 3Sixty Public Affairs
CORD Rare Drug Conference: June 8-9, 2022
Registries and Real-World Data
INFORM RARE: Beth Potter, Alexandra Wyatt, Pranesh Chakraborty,
Monica Lamoureux, John Adams, Kim Angel
CORD Rare Drug Conference: June 8-9, 2022
Registries and Real-World Data
INFORM RARE: Beth Potter, Alexandra Wyatt, Pranesh Chakraborty,
Monica Lamoureux, John Adams, Kim Angel Opportunities and Challenges for Data Management
CORD Rare Drug Conference June 8-9, 2022
Global, International, and National Rare Disease Networks
Rare Disease Research Network and National Children’s Hospital - Marshall
Summar, Rare Disease Institute
CORD Rare Drug Conference: June 8-9, 2022
Global, International, and National Rare Disease Networks
WHO-RDI Global Rare Disease Network - Matt Bolz-Johnson, EURORDIS
CORD Rare Drug Conference: June 8-9, 2022
Global, International, and National Rare Disease Networks
Canadian Network of Rare Disease Centres of Excellence - Paula Robeson, Children’s Healthcare Canada
CORD Rare Drug Conference: June 8 - 9, 2022
The Ottawa Pediatric Bone Health Research Group and The Canadian Consortium for Children’s Bone Health/Canadian Alliance for Rare Disorders of the Skeleton - Leanne Ward, CHEO
CORD Rare Drug Conference: June 8-9, 2022
What is status of Canadian access for RD drugs?
• Canada access and Rest of World - Alexandra Chambers, Novartis
• Canada access to essential rare disease drugs - Nigel Rawson
More from Canadian Organization for Rare Disorders (20)
Flu Vaccine Alert in Bangalore Karnatakaaddon Scans
As flu season approaches, health officials in Bangalore, Karnataka, are urging residents to get their flu vaccinations. The seasonal flu, while common, can lead to severe health complications, particularly for vulnerable populations such as young children, the elderly, and those with underlying health conditions.
Dr. Vidisha Kumari, a leading epidemiologist in Bangalore, emphasizes the importance of getting vaccinated. "The flu vaccine is our best defense against the influenza virus. It not only protects individuals but also helps prevent the spread of the virus in our communities," he says.
This year, the flu season is expected to coincide with a potential increase in other respiratory illnesses. The Karnataka Health Department has launched an awareness campaign highlighting the significance of flu vaccinations. They have set up multiple vaccination centers across Bangalore, making it convenient for residents to receive their shots.
To encourage widespread vaccination, the government is also collaborating with local schools, workplaces, and community centers to facilitate vaccination drives. Special attention is being given to ensuring that the vaccine is accessible to all, including marginalized communities who may have limited access to healthcare.
Residents are reminded that the flu vaccine is safe and effective. Common side effects are mild and may include soreness at the injection site, mild fever, or muscle aches. These side effects are generally short-lived and far less severe than the flu itself.
Healthcare providers are also stressing the importance of continuing COVID-19 precautions. Wearing masks, practicing good hand hygiene, and maintaining social distancing are still crucial, especially in crowded places.
Protect yourself and your loved ones by getting vaccinated. Together, we can help keep Bangalore healthy and safe this flu season. For more information on vaccination centers and schedules, residents can visit the Karnataka Health Department’s official website or follow their social media pages.
Stay informed, stay safe, and get your flu shot today!
These simplified slides by Dr. Sidra Arshad present an overview of the non-respiratory functions of the respiratory tract.
Learning objectives:
1. Enlist the non-respiratory functions of the respiratory tract
2. Briefly explain how these functions are carried out
3. Discuss the significance of dead space
4. Differentiate between minute ventilation and alveolar ventilation
5. Describe the cough and sneeze reflexes
Study Resources:
1. Chapter 39, Guyton and Hall Textbook of Medical Physiology, 14th edition
2. Chapter 34, Ganong’s Review of Medical Physiology, 26th edition
3. Chapter 17, Human Physiology by Lauralee Sherwood, 9th edition
4. Non-respiratory functions of the lungs https://academic.oup.com/bjaed/article/13/3/98/278874
NVBDCP.pptx Nation vector borne disease control programSapna Thakur
NVBDCP was launched in 2003-2004 . Vector-Borne Disease: Disease that results from an infection transmitted to humans and other animals by blood-feeding arthropods, such as mosquitoes, ticks, and fleas. Examples of vector-borne diseases include Dengue fever, West Nile Virus, Lyme disease, and malaria.
Basavarajeeyam is an important text for ayurvedic physician belonging to andhra pradehs. It is a popular compendium in various parts of our country as well as in andhra pradesh. The content of the text was presented in sanskrit and telugu language (Bilingual). One of the most famous book in ayurvedic pharmaceutics and therapeutics. This book contains 25 chapters called as prakaranas. Many rasaoushadis were explained, pioneer of dhatu druti, nadi pareeksha, mutra pareeksha etc. Belongs to the period of 15-16 century. New diseases like upadamsha, phiranga rogas are explained.
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.
Lung Cancer: Artificial Intelligence, Synergetics, Complex System Analysis, S...Oleg Kshivets
RESULTS: Overall life span (LS) was 2252.1±1742.5 days and cumulative 5-year survival (5YS) reached 73.2%, 10 years – 64.8%, 20 years – 42.5%. 513 LCP lived more than 5 years (LS=3124.6±1525.6 days), 148 LCP – more than 10 years (LS=5054.4±1504.1 days).199 LCP died because of LC (LS=562.7±374.5 days). 5YS of LCP after bi/lobectomies was significantly superior in comparison with LCP after pneumonectomies (78.1% vs.63.7%, P=0.00001 by log-rank test). AT significantly improved 5YS (66.3% vs. 34.8%) (P=0.00000 by log-rank test) only for LCP with N1-2. Cox modeling displayed that 5YS of LCP significantly depended on: phase transition (PT) early-invasive LC in terms of synergetics, PT N0—N12, cell ratio factors (ratio between cancer cells- CC and blood cells subpopulations), G1-3, histology, glucose, AT, blood cell circuit, prothrombin index, heparin tolerance, recalcification time (P=0.000-0.038). Neural networks, genetic algorithm selection and bootstrap simulation revealed relationships between 5YS and PT early-invasive LC (rank=1), PT N0—N12 (rank=2), thrombocytes/CC (3), erythrocytes/CC (4), eosinophils/CC (5), healthy cells/CC (6), lymphocytes/CC (7), segmented neutrophils/CC (8), stick neutrophils/CC (9), monocytes/CC (10); leucocytes/CC (11). Correct prediction of 5YS was 100% by neural networks computing (area under ROC curve=1.0; error=0.0).
CONCLUSIONS: 5YS of LCP after radical procedures significantly depended on: 1) PT early-invasive cancer; 2) PT N0--N12; 3) cell ratio factors; 4) blood cell circuit; 5) biochemical factors; 6) hemostasis system; 7) AT; 8) LC characteristics; 9) LC cell dynamics; 10) surgery type: lobectomy/pneumonectomy; 11) anthropometric data. Optimal diagnosis and treatment strategies for LC are: 1) screening and early detection of LC; 2) availability of experienced thoracic surgeons because of complexity of radical procedures; 3) aggressive en block surgery and adequate lymph node dissection for completeness; 4) precise prediction; 5) adjuvant chemoimmunoradiotherapy for LCP with unfavorable prognosis.
CDSCO and Phamacovigilance {Regulatory body in India}NEHA GUPTA
The Central Drugs Standard Control Organization (CDSCO) is India's national regulatory body for pharmaceuticals and medical devices. Operating under the Directorate General of Health Services, Ministry of Health & Family Welfare, Government of India, the CDSCO is responsible for approving new drugs, conducting clinical trials, setting standards for drugs, controlling the quality of imported drugs, and coordinating the activities of State Drug Control Organizations by providing expert advice.
Pharmacovigilance, on the other hand, is the science and activities related to the detection, assessment, understanding, and prevention of adverse effects or any other drug-related problems. The primary aim of pharmacovigilance is to ensure the safety and efficacy of medicines, thereby protecting public health.
In India, pharmacovigilance activities are monitored by the Pharmacovigilance Programme of India (PvPI), which works closely with CDSCO to collect, analyze, and act upon data regarding adverse drug reactions (ADRs). Together, they play a critical role in ensuring that the benefits of drugs outweigh their risks, maintaining high standards of patient safety, and promoting the rational use of medicines.
Title: Sense of Taste
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 structure and function of taste buds.
Describe the relationship between the taste threshold and taste index of common substances.
Explain the chemical basis and signal transduction of taste perception for each type of primary taste sensation.
Recognize different abnormalities of taste perception and their causes.
Key Topics:
Significance of Taste Sensation:
Differentiation between pleasant and harmful food
Influence on behavior
Selection of food based on metabolic needs
Receptors of Taste:
Taste buds on the tongue
Influence of sense of smell, texture of food, and pain stimulation (e.g., by pepper)
Primary and Secondary Taste Sensations:
Primary taste sensations: Sweet, Sour, Salty, Bitter, Umami
Chemical basis and signal transduction mechanisms for each taste
Taste Threshold and Index:
Taste threshold values for Sweet (sucrose), Salty (NaCl), Sour (HCl), and Bitter (Quinine)
Taste index relationship: Inversely proportional to taste threshold
Taste Blindness:
Inability to taste certain substances, particularly thiourea compounds
Example: Phenylthiocarbamide
Structure and Function of Taste Buds:
Composition: Epithelial cells, Sustentacular/Supporting cells, Taste cells, Basal cells
Features: Taste pores, Taste hairs/microvilli, and Taste nerve fibers
Location of Taste Buds:
Found in papillae of the tongue (Fungiform, Circumvallate, Foliate)
Also present on the palate, tonsillar pillars, epiglottis, and proximal esophagus
Mechanism of Taste Stimulation:
Interaction of taste substances with receptors on microvilli
Signal transduction pathways for Umami, Sweet, Bitter, Sour, and Salty tastes
Taste Sensitivity and Adaptation:
Decrease in sensitivity with age
Rapid adaptation of taste sensation
Role of Saliva in Taste:
Dissolution of tastants to reach receptors
Washing away the stimulus
Taste Preferences and Aversions:
Mechanisms behind taste preference and aversion
Influence of receptors and neural pathways
Impact of Sensory Nerve Damage:
Degeneration of taste buds if the sensory nerve fiber is cut
Abnormalities of Taste Detection:
Conditions: Ageusia, Hypogeusia, Dysgeusia (parageusia)
Causes: Nerve damage, neurological disorders, infections, poor oral hygiene, adverse drug effects, deficiencies, aging, tobacco use, altered neurotransmitter levels
Neurotransmitters and Taste Threshold:
Effects of serotonin (5-HT) and norepinephrine (NE) on taste sensitivity
Supertasters:
25% of the population with heightened sensitivity to taste, especially bitterness
Increased number of fungiform papillae
TEST BANK for Operations Management, 14th Edition by William J. Stevenson, Ve...kevinkariuki227
TEST BANK for Operations Management, 14th Edition by William J. Stevenson, Verified Chapters 1 - 19, Complete Newest Version.pdf
TEST BANK for Operations Management, 14th Edition by William J. Stevenson, Verified Chapters 1 - 19, Complete Newest Version.pdf
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
2. WHERE ARE WE NOW
AND WHERE DO WE
NEED TO GO?
It is mid-2022. As we move closer to the launch of Canada’s Rare Drug Strategy, CORD is
stepping up consultations with five to six webinars in April and May and a two-day “in person”
conference on June 8-9 in Ottawa. Our intended outcome is NOT a plan to allocate the $1
billion start-up funding nor the $500 million annual commitment. We are NOT proposing
minor tweaks to address gaps in the system nor even incremental improvements. We are
calling on all stakeholders to arrive at “as near a consensus as possible” on a complete re-
imagining of a system from the bottom up and top down that is “fit for purpose” of assuring
all Canadians have access to the best therapies as soon as they are available anywhere in the
world.
This means reimaginig and executing a patient-centred rare disease system that assures
every person, in as short a time as possible, gets an accurate diagnosis, sees a specialist, forms
an individualized care, treatment, and support plan, is enrolled in a patient registry and
referred to a patient support group, gets timely monitored access to the best treatment and,
most important, has the knowledge and right to participate as a full partner in all health-
related decisions. This is available for all Canadians, regardless of where they live, their
insurance coverage or their ability to pay.
3. WHY IS RARE DISEASE
A PUBLIC HEALTH
ISSUE?
There are more Canadians with a rare disease than with all cancers,
cardiovascular disease, diagnosed diabetes. Altogether, 3 million
Canadians, about 1 in 12, have a rare disease. Moreover, while 80% of rare
diseases are genetic, 50% have no known family history. So everyone is
potentially at risk. Two-thirds of those affected are children, 30% of whom
will not live to their fifth birthday. Sadly, in Canada, a child dies of a rare
disease every 18 minutes.
In terms of economic impact, rare disease costs the Canadian economy
about $111 billion per year in direct medical costs, nonmedical cost, and
productivity costs.
4. WHY IS NOW THE TIME
FOR CANADA’S SMART
RARE DRUG STRATEGY?
Rare disease drugs are saving lives, preventing and reducing disability, allowing patients and families to live more “normal”
productive lives, and, for some conditions, providing a long-lasting treatment or “cure.” Rare disease research is leveraging
genomic breakthroughs, advanced medical technologies, application of data science and artificial intelligence to big data, patient
registries, and real-world data, multiple disease cell-and-gene therapy platforms, and remote patient engagement. So rare disease
therapies are at the forefront of innovative research, providing solutions with impacts beyond rare diseases, including viruses,
cancers, and cardiovascular diseases.
Thanks to the US and EU Orphan Drug Acts, about 600 new therapies have been approved in the past 40 years but these cover
only about 5% of up to 7,000 rare diseases. To date, Canada has contributed to only a small handful of rare disease drug discoveries.
However, that scenario can and should change. Canada’s burgeoning life sciences strategy has the scientific, clinical, and patient
capacity to contribute significantly to discovery, manufacture, and management of rare disease diagnosis and treatment.
Consider two axioms here: “Your very success is what is going to destroy you” and the “tragedy of the commons” whereby pursuit
of individual good can result in disaster for the common good. In other words, early reimbursement solutions for rare disease drugs
therapies cannot be repeated infinitely when the number of new therapies expand exponentially. Most would agree that our drug
assessment procedures were never designed to accommodate innovative therapies for small patient populations but we have
continued to put them through the process and have mostly “cobbled” “on-off” reimbursement agreements with ostensibly
minimal impact on the overall drug budgets. The solutions were rarely evidence-based, equitable, or sustainable. The question is
not whether we have reached the breaking point with the “old” access pathways but “how can we do better?”
5. WHAT ARE THE HALLMARKS
OF A SMART RARE DRUG
STRATEGY?
The opportunity to introduce a new Rare Disease Drug
System is to build it with SMART principles. SMART
systems share three attributes.
• engaged patients;
• digital capture, linkage and timely sharing of relevant data;
• timely production of research evidence;
• appropriate decision supports;
• aligned governance, financial and delivery arrangements;
• a culture of rapid learning and improvement; and
• competencies for rapid learning and improvement.
1. SMART HEALTH SYSTEMS ARE
DESIGNED TO BE RAPID LEARNING
SYSTEMS, AS INDICATED BY:
6. • Specific: Well defined, clear, and unambiguous
• Measurable: With specific criteria that measure your
progress toward the accomplishment of the goal
• Achievable: Attainable and not impossible to achieve
• Realistic: Within reach, realistic, and relevant to your life
• Timely: With a clearly defined timeline, including a starting
date and a target date. The purpose is to create urgency.
2. SMART HEALTH SYSTEMS
ENGAGE IN AGILE PLANNING
WHICH MEANS CONTINUOUSLY
UPDATING SMART GOALS, WHICH
ARE DEFINED AS:
7. 3. SMART HEALTH SYSTEMS INTEGRATE
SMART TECHNOLOGY, REFERRING TO
SELF-MONITORING ANALYSIS AND
REPORTING TECHNOLOGY
Two trends driving healthcare are, on the demand side, growing recognition of health services
as a basic human right and an investment for a healthy society and, on the supply side,
increasingly complex and impactful solutions that are highly specialized and costly. The
conundrum is compounded by increasing competition for resources both within healthcare
and with other sectors as economies recover from the pandemic and beleaguered healthcare
providers exit their professions.
The way forward is the digital transformation of healthcare, and technology companies are
being called up to develop solutions that improve healthcare performance and outcomes.
Examples span individual devices to system management including:
• digitally connected remote healthcare services to expert centres and even more remote
patients;
• sharing of patient health data across a variety of systems for multiple purposes;
• employing AI to rollout vaccines, conduct virtual screening, simulate drug adverse effects,
and speed up diagnosis using algorithms
• Proactive healthcare wearables that can collect signals in real time
• Smart hospital management: workloads and patient flow
8. Initial Response to: Canadian Government’s
Discussion Guide for National Strategy on Drugs
for Rare Diseases
CORD High-Level Critique
Our reactions to What We Heard: We feel dismayed, bewildered, and betrayed.
• Know you heard us because your recapitulation of your WWH summary was mostly what we said back then
• Potential Draft Framework: sets up the slippery slope toward no meaningful action
• Language throughout is highly speculative, theoretical, and deliberately vague with no commitment to actions, goals
or outcomes, and timelines
1. Potential common vision is underwhelming, not worthy of the boldacious 2019 government commitment backed with $1
billion initial funding. We cannot galvanize the Canadian rare disease community to collective action with a vision of
“improved access” and “better health outcomes.”
2. These eight principles are meaningless. Principles are “fundamental truths” that underlie actions, whereas these are
presented as a list of words with no explanation as to how these will be fundamental to the design, implementation and
evaluation of the Rare Disease Drug Framework.
3. Invest in 4 Strategic Pillars
• These are not strategic nor pillars nor even “four” different sects of activities but necessarily highly related toward one
(unstated) goal
• Many of the activities are couched in the form of “advisory” activities, such as “explore feasibility”, “engage with”,
“create a plan for future”, “support”, “invest in”, “build relationships with” with no reference as to “the entity” that is
doing the advising (government, advisory committee, new drug agency) and to whom the advice is provide for
execution (designated or accountable body or coalitions).
• There are no linkages of activities to the guiding principles or the desired outcomes of these activities.
9. Initial Response to: Canadian Government’s
Discussion Guide for National Strategy on Drugs
for Rare Diseases cont…
Pillar 1: Improve access to rare disease treatments and make it consistent across Canada
• Improved and consistent access could be a “death spiral” unless there is a clear commitment to an overarching
principle: All rare disease patients shall get the fasted access possible to the best drug for their specific condition and
personal profile in the shortest time possible (through clinical trials, special access, and other early access paths)
comparable to the best countries). Otherwise:
• Improved access without requirement of best could mean any therapy that is better than nothing or access for all rare
disease patients to the lowest common denominator of willingness to fund
• Without commitment to rapid access, coordination across decision makers could lead to intolerable delays anchored
by the least willing funder
Pillar 2: Optimize, collect and use data along drug system continuum and across lifecycle must be
based on:
• Principle of “adaptive learning”, that is, recognize the realities of our non-existent national data platform and system
(have we learned nothing from InfoWay)?
• Learn from discrete programs (existing and new) which rely on existing data systems and platforms that are already
collecting health data and using them to monitor treatment and outcomes, in specialty clinical networks, in cancer
networks, in private services using and service public systems.
10. Initial Response to: Canadian Government’s
Discussion Guide for National Strategy on Drugs
for Rare Diseases cont…
Pillar 3: Support optimal patient outcomes and sustainability of health system by spending on
drugs that bring “value for money.”
• This pillar is just big giveaway or back door to health technology assessment (HTA), that is, cost-effectiveness and cost-
utility assessments (cost per Quality-Adjusted Life Year calculations) with comparative and competitive ICERS
(incremental cost-effectiveness ratios). The premise of “value for money” can ONLY work in achieving the best
outcomes for patients and society are the driving principles and IF the failure of tradition HTA methodologies (timing,
evidence requirements, and benchmark ICERs) to achieve optimal and even reasonable assessments for rare disease
therapies.
• Additional concern for Pillars 1, 2, and 3: these are all the same domain and all directed toward shared or consensual
HTA for DRDs and providing access to those that meet whatever criteria or threshold would be deemed “value for
money.” These do not represent an innovative step forward.
Pillar 4: Strengthen alignment of research and innovative systems with DRD access objectives
• This in and of itself is not very illuminating. However, the proposal to build rare disease research capacity to create data
collection and sharing systems that can contribute to clinical trials (and other goals) is worthwhile, even if vague as to
implementation.
11. Initial Response to: Canadian Government’s
Discussion Guide for National Strategy on Drugs
for Rare Diseases cont…
Iterative Implementation
Drug Coverage
Why as first phase federal support, with partners and payers, of coverage for “select drugs of common concern.” Not most
pressing concern from patients.
Duplicates what already exists. More reasonable to gaps: provide mandatory coverage to all families, similar to the Quebec
model.
Greatest obstacle to consensus is which drugs are to selected by whom and serving whose common concern? By
definition, rare disease drugs are not common concerns.
Top priority is access to those (often new and advanced) therapies that have the greatest impact on saving lives,
improving disease outcomes, or transforming lives.
Need accelerated pathways that would provide access at the very earliest possibility, during clinical trials, pre-NOC,
immediately upon NOC through managed access programs that would provide patient access with provisions for
monitoring and real-world data collection, with reimbursement potentially conditional on outcomes.
An initial “common concerns” drug list does not address the patient needs, the system gaps and definitely not long-term
“value for money” and sustainability of the Rare Drug program and the health system.
12. Initial Response to: Canadian Government’s
Discussion Guide for National Strategy on Drugs
for Rare Diseases cont…
Governance
Committees for specific therapies include experts from affected rare disease community, including patients and families,
clinicians, and other care providers.
Independent Rare Disease Drug Program = all stakeholders at all levels in multiple roles, including patients, not only on advisory
committees but also assessment, monitoring, and decision-making bodies at every level, including the top governing board.
All stakeholders should receive the necessary training and support to share expertise in a collaborative environment.
Transparency should include open access to all materials and to the assessment, deliberation,
and decision-making processes.
Optimal patient outcomes
“Value for money” is just another way of describing HTA; traditional methodologies not working for rare disease drugs in Canada
or elsewhere)
Streamlining process and efforts for DRD will NOT resolve the fact that the traditional model is not “fit for purpose”, which is true
worldwide.
Risk-sharing and equitable sharing of costs need a single platform for assessment and development of “managed access”
agreements including CT evidence, extension to non-trial patients, and collecting outcomes in real-world to monitor safety and
efficacy.
Risk-sharing of the costs and innovative payment models are on-going processes where the costs paid (over time) may vary as
evidence on outcomes and number of patients evolve over time in real-world usage.
Establish Canadian Network of Rare Disease Centres of Excellence with specialty networks embedded across the Centres. These
Centres of Excellence are also the vehicles for data collection and analysis, which will allow for aligning research and therapy
management.
14. SPRING 2022
WEBINARS
MAY 3
12 - 1 pm
Rare Disease
Patient Registries:
Key to Drug
Development and
Access
MAY 5
12 - 2 pm
Rare Disease Centres
of Excellence:
Linchpin to patients,
community care,
and collaboration
MAY 19
12 - 1 pm
Leave No One
Behind
MAY 26 *POSTPONED
Economic Case for
Rare and Innovative
Research
* Now will be held as a
session during the June 8-9
conference
APRIL 19
12 - 1 pm
Application of
RWE in Drug
Access Decision
Making
APRIL 26
12 - 1 pm
Lessons for a SMART
Rare Drug System
15. MAY 5
12 - 2 PM
The government of Canada has committed $1 billion to set up Canada’s Rare Disease Drug
Strategy. The rare disease community, under the leadership of the Canadian Organization
for Rare Disorders, has called for the implementation of a SMART Rare Drug Strategy as
part of Canada’s SMART Rare Disease System that will address the challenges of access
today and carry us into the future to provide FAST access to medicines that will also
stimulate investment in innovation that will deliver best health outcomes, research and
development, and sustainable public health, economic growth, and societal benefits.
As continuation to two years of public consultation, CORD has launched a six-session
consultation on a SMART Rare Disease Drug Strategy and System. CORD in collaboration
with Institute of Genetics, CIHR, and MICYRN (Maternal, Infant, Child, Youth Research
Network) are hosting Webinar 4 on Rare Disease Centres of Excellence to learn about COEs
in other jurisdictions, rare disease research and treatment networks across Canada, and
rare disease specialty network. An expert multi-stakeholder panel will consider how
learnings from other countries can be applied in Canada (or not) and deliberate on a plan
of action to implement toward establishment of a Canadian Network for Rare Disease
Centres of Excellence, especially as a foundation for the Rare Drug Strategy.
Rare Disease Centres of Excellence:
Linchpin to patients, community care,
and collaboration
16. AGENDA
• Learning from Ex-Canada
⚬ European Reference Networks (Sara Talarico/Domenica Taruscio)
⚬ WHO-RDI Global Rare Disease Network (Matt Bolz-Johnson)
⚬ NORD Centres of Excellence (Marybeth McAfee)
• Canada’s Rare Research Networks
⚬ Institute of Genetics, CIHR (Christopher McMaster/Étienne Richer)
⚬ Maternal, Infant, Child, Youth Research Network (Thierry Lacaze-Masmonteil)
⚬ Genome Canada (Ivana Cecic)
• Excellence in Canada
⚬ All for One/Genomics 4RD, CHEO (Kym Boycott)
⚬ Metabolic Disorders Research/Clinical Program, Manitoba (Cheryl Rockman-Greenberg)
⚬ Ottawa Pediatric Bone Health Group and Canadian Working Group (Leanne Ward)
⚬ Pediatric Neurology Research Program, Western University (Craig Campbell)
• Faculty Discussion
• Visioning, SMART Thinking, Next Steps
17. PRESENTERS
An opening panel will present exemplars of existing, emerging, and proposed models and best
practices in coordinated rare disease network from across the globe.
• Sara Talarico/Domenica Taruscio, European Reference Networks
• Matt Bolz-Johnson, WHO-RDI Global Rare Disease Network
• Marybeth McAfee, NORD Centres of Excellence
• Christopher McMaster/Étienne Richer, CIHR Institute of Genetics
• Thierry Lacaze-Masmonteil, Maternal, Infant, Child, Youth Research Network
• Ivana Cecic, Genome Canada
• Kym Boycott, Children’s Hospital of Eastern Ontario
• Cheryl Rockman-Greenberg, Max Rady College of Medicine, University of Manitoba
• Leanne Ward, Children’s Hospital of Eastern Ontario
• Craig Campbell, Department of Pediatrics, Western University
18. DISTINGUISHED
FACULTY
·A panel of distinguished faculty from across Canada are invited to respond to presenters and
consider the implementation of Canada’s Rare Disease Network
• Stuart Turvey, BC Children's Hospital
• Peter Kannu, Stollery Children Hospital, Edmonton
• Francois Bernier, Alberta Children's Hospital
• Micheil Innes, Alberta Children's Hospital
• Beth Potter, CHEO
• Jason Berman, CHEO
• Ronald Cohn, The Hospital for Sick Children, Toronto
• James Dowling, The Hospital for Sick Children, Toronto
• Jacques Michaud, Sainte Justine
• Hugh McMillan, McGill - Montreal Children's Hospital
• Nicolas Chrestian, CHU Laval - Quebec
Moderators
• Bill Dempster, 3Sixty Public Affairs
• Durhane Wong-Rieger, CORD
19. CONFERENCE
JUNE 8 - 9, 2022
Ottawa Marriott Hotel
Building Canada’s SMART Rare
Disease and Rare Drug System
20. National Organization for Rare Disorders | rarediseases.org
NORD RARE DISEASE CENTERS
OF EXCELLENCE PROGRAM
Marybeth McAfee, MA, GC
Associate Director, NORD RD CoE Program
21. NORD, a 501(c)(3) organization, is a patient
advocacy organization dedicated to individuals
with rare diseases and the organizations that
serve them. NORD, along with its more than
330 patient organization members, is committed
to the identification, treatment, and cure of rare
disorders through programs of education,
advocacy, research, and patient services.
NORD® MISSION STATEMENT
rarediseases.org 2
22. 3
rarediseases.org
NORD Rare Disease Centers of Excellence
On Nov 4th 2021, NORD announced its designation of 31 medical institutions across
the United States with exceptional programs for patients with rare diseases as NORD
Rare Disease Centers of Excellence (NORD RD CoE’s).
23. 4
rarediseases.org
Vision of the NORD RD CoE Program
All persons living with a rare disease, regardless of disease, socioeconomic level, or
demographics, have access to timely diagnosis, quality, compassionate clinical care, research
opportunities, and supportive resources.
• Policy
• Professional Education
• Research
• Care
25. 6
rarediseases.org
Application Overview – Multiple Choice Questions
• Slightly over 100 Multiple Choice
Questions
• Qualitatively assessing each site’s
“Onsite” or “Local” access to specific
staffing, facilities, or services
• Quantitative collection of “clinical
fulltime equivalents” (cFTE’s) in key
specialties very often involved in
rare disease diagnosis and care.
• Examples:
• Liver transplant service
• Pediatric heart surgery
• Disease patient “navigators” and/or
case managers
• Specialty laboratories for esoteric
rare disease diagnostic or
monitoring tests, etc.
• # of Genetic Counselors, Clinical
Geneticists, Biochemical or
Molecular Geneticists, Pediatric
Neurologists
26. 7
rarediseases.org
Application Overview – Essay Questions & LOS
10 Essay Questions
• 750-word limit
• Answers to focus on rare disease
• Rubric for scoring was included
Letters of Support
• Ranged from 1-4
• Examples:
• Multidisciplinary clinics
• Types of rare disease certified
clinical training programs
• Rare disease patient resources
• Programs to reach underserved
• Pediatric to adult transition
• Patient satisfaction scores
27. 8
rarediseases.org
Applications Process
Invitations
• Invitations were sent to the 40 plus
Institutions
• Directed to head of clinical genetics
programs
• Had ACGME certified clinical training
programs
Technical Assessment Call
• Program information – more than a
sticker
• Application information
• Explanation of rubrics
• Q&A
28. 9
rarediseases.org
Analyzing the Applications
Multiple choice questions:
• Pulled from application system
• Did little to differentiate between applicants
Essay questions:
• Scored using rubric by 3 different NORD staff
• Each staff had 8 or so applicants essay answers to review
• Letters of Support were read but were not scored or included in scoring rubric
29. 10
rarediseases.org
31 NORD RD CoE
• University of Alabama at Birmingham Medicine/Children’s of Alabama
• Children’s Hospital of Orange County / UC Irvine
• University of California, San Francisco & UCSF Benioff Children's Hospitals
• University of Colorado Anschutz Medical Campus/Children’s Hospital Colorado
• University of Miami Miller School of Medicine
• Emory Division of Medical Genetics
• Indiana University School of Medicine
• University of Iowa Health Care
• Johns Hopkins Medicine/Kennedy Krieger Institute
• Mass General Hospital/Mass General Hospital for Children/Brigham and Women’s
Hospital/Boston Children’s Hospital
• Mayo Clinic
• M Health Fairview-University of Minnesota Masonic Children's Hospital
• Washington University/BJC Healthcare
• UNMC Munroe-Meyer Institute, Omaha Children's Hospital and Nebraska Medicine
• Columbia University Irving Medical Center
• Department of Genetics and Genomic Sciences, Icahn School of Medicine at Mount
Sinai
• Duke University Medical Center
• UNC Children's - North Carolina Children's Hospital
• Cincinnati Children's Hospital Medical Center
• Nationwide Children's Hospital/Ohio State University
• OU Health/University of Oklahoma Health Sciences Center
• Penn Medicine/Children’s Hospital of Philadelphia
• UPMC Center for Rare Disease Therapy
• Vanderbilt University Medical Center
• Baylor College of Medicine/Baylor St. Luke’s Medical Center/Texas Children’s Hospital
• McGovern Medical School/UTHeath
• UT Southwestern Medical Center
• University of Utah Medical Genetics
• Rare Disease Institute at Children’s National Hospital
• Children's Wisconsin/Medical College of Wisconsin
• University of Wisconsin Center for Rare Diseases
31. 12
rarediseases.org
Revising the Application
Multiple Choice Questions
• Retain most of the questions
• Consolidate some of specialist questions
• Remove redundancies
• Increase clearness of questions
Essay Question
• Analyzing reviewers’ comments (2021 cycle)
• Reconsider 750-word limit
• Refine rubric
• Modify weighting
32. 13
rarediseases.org
A Few Thoughts to Consider
• Try to envision situations where worthy
applicants will apply that won’t meet the
designation criteria
• Independent children’s hospital
• Independent specialty hospitals
• Come up with strategies to mitigate loss of
quality applicants
• Support working with a hospital that treats
adults to put in a joint application
• Support with larger university hospital to put
in a joint application
• Know ahead of time what you
want to analyze to distinguish
your vision of a rare disease
center of excellence
• Be cautious making
announcements that may commit
you to a challenging timeline
• Consider supporting co-director
structure rather than single
director at each Center