This document discusses access challenges for orphan drugs and specialized therapies in Canada. It proposes alternative models like managed access programs and adaptive listings to generate real-world evidence for these treatments. Managed access programs in other countries tie reimbursement to ongoing data collection on safety, effectiveness and appropriate use. Challenges include defining patient eligibility, setting stopping criteria, and assigning stakeholder roles. The document also describes private insurers' initiatives in Canada like chronic disease management programs and references to CADTH reviews. It argues collaborative solutions are needed to provide timely, equitable access to promising rare disease therapies in a sustainable way.
Testing Telehealth Solutions for Post Acute CareVSee
Telehealth Failures & Secrets to Success Conference 2017 by VSee
Speaker: Tomi Ryba & Margaret Wilmer
Senior Director of Integrated Care of El Camino Hospital
More info at: vsee.com/conference
Identifying High Performing Sites and Engaging PatientsMedpace
One of the biggest challenges facing any clinical trial is how to identify the sites with the greatest potential to engage and retain patients. Applying decades of experience to the topic, Medpace experts will share considerations, lessons-learned and best practices for developing patient recruitment strategies to put you on the path for optimal success.
Presentation in the framework of the International Conference "10th anniversary of the Spanish Network of Health Technology Assessment Agencies. Towads patient and public engagement in HTA" Zaragoza 27-28 April 2017
Final MSN-CNL Capstone Poster PresentationAshleyKendall6
My master's clinical nurse leader capstone quality improvement project's purpose was to decrease the number of intentional poisoning patient admissions on the transitional care unit (TCU) at SSM Health Cardinal Glennon Children's Hospital. Shown here is the project poster for presentation I created in May 2021.
Expediting Colonoscopy for Patients with + Faecal Occult Blood Test in a Publ...Cancer Institute NSW
Colon cancer is the commonest cancer in Australia. The Federal Gov. has recently accelerated the rollout of the National Bowel Cancer Screening Program to 2nd yearly after age 50 by 2018. We anticipate up to 1000 extra colonoscopies on the public system at NSLHD.
Enhancing the patient experience in a new purpose-build MDT meeting room with...Cancer Institute NSW
St Vincent’s Head and Neck Clinic is a well-established, multidisciplinary clinic which has provided a co-ordinated team approach to the head and neck patient’s complex needs for over three decades. With the development of a new, purpose-built cancer facility, a clinical redesign project was undertaken, with the aim to further enhancing the patient experience and improving the quality of care for patients attending the weekly Multidisciplinary Head and Neck Clinic.
Optimising the Model of Care for Patient Management at The Tweed Cancer Care ...Cancer Institute NSW
The commonly understood model of shift to shift nursing handover does not apply to most ambulatory day treatment units. Nonetheless, ‘handover’ of patient clinical information remains quintessential to safe clinical practice. Of considerable interest is how EMR may aid the transfer of patient clinical information in these circumstances and address the question: does this facilitate improved patient care?
Testing Telehealth Solutions for Post Acute CareVSee
Telehealth Failures & Secrets to Success Conference 2017 by VSee
Speaker: Tomi Ryba & Margaret Wilmer
Senior Director of Integrated Care of El Camino Hospital
More info at: vsee.com/conference
Identifying High Performing Sites and Engaging PatientsMedpace
One of the biggest challenges facing any clinical trial is how to identify the sites with the greatest potential to engage and retain patients. Applying decades of experience to the topic, Medpace experts will share considerations, lessons-learned and best practices for developing patient recruitment strategies to put you on the path for optimal success.
Presentation in the framework of the International Conference "10th anniversary of the Spanish Network of Health Technology Assessment Agencies. Towads patient and public engagement in HTA" Zaragoza 27-28 April 2017
Final MSN-CNL Capstone Poster PresentationAshleyKendall6
My master's clinical nurse leader capstone quality improvement project's purpose was to decrease the number of intentional poisoning patient admissions on the transitional care unit (TCU) at SSM Health Cardinal Glennon Children's Hospital. Shown here is the project poster for presentation I created in May 2021.
Expediting Colonoscopy for Patients with + Faecal Occult Blood Test in a Publ...Cancer Institute NSW
Colon cancer is the commonest cancer in Australia. The Federal Gov. has recently accelerated the rollout of the National Bowel Cancer Screening Program to 2nd yearly after age 50 by 2018. We anticipate up to 1000 extra colonoscopies on the public system at NSLHD.
Enhancing the patient experience in a new purpose-build MDT meeting room with...Cancer Institute NSW
St Vincent’s Head and Neck Clinic is a well-established, multidisciplinary clinic which has provided a co-ordinated team approach to the head and neck patient’s complex needs for over three decades. With the development of a new, purpose-built cancer facility, a clinical redesign project was undertaken, with the aim to further enhancing the patient experience and improving the quality of care for patients attending the weekly Multidisciplinary Head and Neck Clinic.
Optimising the Model of Care for Patient Management at The Tweed Cancer Care ...Cancer Institute NSW
The commonly understood model of shift to shift nursing handover does not apply to most ambulatory day treatment units. Nonetheless, ‘handover’ of patient clinical information remains quintessential to safe clinical practice. Of considerable interest is how EMR may aid the transfer of patient clinical information in these circumstances and address the question: does this facilitate improved patient care?
The Design of Accountable Care OrganizationsCJ Fulton
Pillars for Accountable Care
PCMH versus ACOs
Core competencies
Six core structural components of successful ACO deployment
Pioneer ACO burn and learn lessons
Barriers & root cause analysis
Patient attribution
Five modes of Accountable Care
Early value-based adopters
Value discovery assessment
Modified Triple Aim
GPRO
Breakdown by 33 Measures
Patient-centered pharmacovigilance represents a pivotal shift in the landscape of healthcare, emphasizing the active involvement of patients in the monitoring and reporting of adverse drug reactions. Unlike traditional pharmacovigilance, which primarily relies on healthcare professionals to identify and document adverse events, this approach recognizes patients as critical stakeholders in ensuring medication safety. By empowering patients to share their experiences, concerns, and observations regarding medication effects, whether positive or negative, healthcare systems can gain a comprehensive understanding of drug safety and efficacy in real-world settings. Patient-centered pharmacovigilance fosters a collaborative partnership between patients, healthcare providers, and regulatory agencies, promoting transparency, accountability, and ultimately, better patient outcomes. Through increased patient engagement and the utilization of patient-reported data, this approach enables healthcare systems to identify potential safety issues earlier, tailor treatment strategies to individual needs, and enhance overall drug safety surveillance efforts.
Real-World Data and Real-World Evidence Webinar
Panelists
Tara Cowling, Medlior
Laurie Lambert, CADTH
Craig Campbell, London Health Sciences
Sandra Anderson, Innomar Strategies
Brad Alyward, Canadian Organization for Rare Disorders
Durhane Wong-Rieger, Canadian Organization for Rare Disorders
Financial Protection and Improved Access to Health Care: A Spotlight on Pharm...HFG Project
Presented at “Financial Protection and Improved Access to Health Care: Peer-to-Peer Learning Workshop Finding Solutions to Common Challenges” in Accra, Ghana, February 2016. To learn more, visit: https://www.hfgproject.org/ghana-uhc-workshop
Comprehensive and person centred approach to addressing Polypharmacy in adult...Health Innovation Wessex
The Health Innovation Network Polypharmacy programme is working with healthcare professionals to address problematic polypharmacy by supporting easier identification of patients at potential risk from harm from multiple medications.
Our evidence-based polypharmacy Action Learning Sets (ALS) are being rolled out across England to support GPs, pharmacists and other healthcare professionals who undertake prescribing or medication reviews to understand the complex issues around stopping inappropriate medicines safely.
To drive and accelerate changes in practice, delegates complete a quality improvement project to address problematic polypharmacy in their workplace. This poster summary, Comprehensive and person centred approach to addressing Polypharmacy in adult care home residents, can be viewed here.
For more information about the polypharmacy programme, please visit https://thehealthinnovationnetwork.co.uk/programmes/medicines/polypharmacy/
Examples of Transformative Technologies Driving Precision Medicine and Personalized Healthcare
1) Marc LePage, Genome Canada
2) Daniel Gaudet, Ecogene-21
3) Kristy Dickinson, Chronically Simple
4) Jian Wang, Health Canada
5) Michael May, CCRM
6) Maxwell Morgan, M4Kids (Pediatric Cancer Therapies)
Pharmacovigilance in Special Populations: Considerations for Pediatric and Ge...ClinosolIndia
Pharmacovigilance is the science and activities related to the detection, assessment, understanding, and prevention of adverse effects or any other drug-related problems. When it comes to special populations, such as pediatric and geriatric patients, there are specific considerations that need to be taken into account in pharmacovigilance efforts. Here are some key points regarding pharmacovigilance in these population
What’s Next in US Payor Communications: The Impact of FDA's Proposed Guidance...Nathan White, CPC
The recent enactment of the 21st Century Cures Act has profound immediate and long-term implications for development and communication of HEOR/RWE in the US, particularly in relation to communications with payors about healthcare economic information (HCEI). In January, the FDA released draft guidance for public comment to outline its thinking around communication to payors of HCEI, but there are still unanswered questions to be addressed in the final guidance. Industry will need to quickly establish new policies and procedures to maintain compliance with the new regulations, especially in relation to OPDP submission requirements – a steep transition from a space that has largely been unregulated.
This presentation explains the main features of medicines which will be developed and authorised via the adaptive pathways. It provides a definition of real world evidence and the caveats associated with the use and analysis of real world evidence in drug development.
Please share this video with anyone who may be interested!
Watch all our webinars: https://www.youtube.com/playlist?list=PL4dDQscmFYu_ezxuxnAE61hx4JlqAKXpR
In this webinar:
● Takeaways from a roundtable held on June 1st about patient-centred pharmacare in Canada
● Reports from patient groups and other subject matter experts
● A cohesive vision and set of values for national pharmacare in Canada
View the video: https://youtu.be/HMy_gsTDkfI
Follow our social media accounts:
Twitter - https://twitter.com/survivornetca
Facebook - https://www.facebook.com/CanadianSurvivorNet
Pinterest - https://www.pinterest.com/survivornetwork
YouTube - https://www.youtube.com/user/Survivornetca
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
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Many ways to support street children.pptxSERUDS INDIA
By raising awareness, providing support, advocating for change, and offering assistance to children in need, individuals can play a crucial role in improving the lives of street children and helping them realize their full potential
Donate Us
https://serudsindia.org/how-individuals-can-support-street-children-in-india/
#donatefororphan, #donateforhomelesschildren, #childeducation, #ngochildeducation, #donateforeducation, #donationforchildeducation, #sponsorforpoorchild, #sponsororphanage #sponsororphanchild, #donation, #education, #charity, #educationforchild, #seruds, #kurnool, #joyhome
Presentation by Jared Jageler, David Adler, Noelia Duchovny, and Evan Herrnstadt, analysts in CBO’s Microeconomic Studies and Health Analysis Divisions, at the Association of Environmental and Resource Economists Summer Conference.
A process server is a authorized person for delivering legal documents, such as summons, complaints, subpoenas, and other court papers, to peoples involved in legal proceedings.
Jennifer Schaus and Associates hosts a complimentary webinar series on The FAR in 2024. Join the webinars on Wednesdays and Fridays at noon, eastern.
Recordings are on YouTube and the company website.
https://www.youtube.com/@jenniferschaus/videos
What is the point of small housing associations.pptxPaul Smith
Given the small scale of housing associations and their relative high cost per home what is the point of them and how do we justify their continued existance
Russian anarchist and anti-war movement in the third year of full-scale warAntti Rautiainen
Anarchist group ANA Regensburg hosted my online-presentation on 16th of May 2024, in which I discussed tactics of anti-war activism in Russia, and reasons why the anti-war movement has not been able to make an impact to change the course of events yet. Cases of anarchists repressed for anti-war activities are presented, as well as strategies of support for political prisoners, and modest successes in supporting their struggles.
Thumbnail picture is by MediaZona, you may read their report on anti-war arson attacks in Russia here: https://en.zona.media/article/2022/10/13/burn-map
Links:
Autonomous Action
http://Avtonom.org
Anarchist Black Cross Moscow
http://Avtonom.org/abc
Solidarity Zone
https://t.me/solidarity_zone
Memorial
https://memopzk.org/, https://t.me/pzk_memorial
OVD-Info
https://en.ovdinfo.org/antiwar-ovd-info-guide
RosUznik
https://rosuznik.org/
Uznik Online
http://uznikonline.tilda.ws/
Russian Reader
https://therussianreader.com/
ABC Irkutsk
https://abc38.noblogs.org/
Send mail to prisoners from abroad:
http://Prisonmail.online
YouTube: https://youtu.be/c5nSOdU48O8
Spotify: https://podcasters.spotify.com/pod/show/libertarianlifecoach/episodes/Russian-anarchist-and-anti-war-movement-in-the-third-year-of-full-scale-war-e2k8ai4
Understanding the Challenges of Street ChildrenSERUDS INDIA
By raising awareness, providing support, advocating for change, and offering assistance to children in need, individuals can play a crucial role in improving the lives of street children and helping them realize their full potential
Donate Us
https://serudsindia.org/how-individuals-can-support-street-children-in-india/
#donatefororphan, #donateforhomelesschildren, #childeducation, #ngochildeducation, #donateforeducation, #donationforchildeducation, #sponsorforpoorchild, #sponsororphanage #sponsororphanchild, #donation, #education, #charity, #educationforchild, #seruds, #kurnool, #joyhome
2. Agenda
§ Review of Payer Framework
§ Review of Orphan Drug Characteristics- why are they different?
§ Consideration of Current Access Challenges
§ Consideration of alternative models for access:
- Managed Access
- Adaptive Listings
3. Increased Management of Drug ProgramsPrivate
Payer
Value DATA, and Customization will be KeyManufacturer
Cost-Containment Environment – Price DrivenPublic PayerPublic
Payer
The Payer Framework in Canada
4. Orphan Drugs
3/16/16 CONFIDENTIAL4
The “Why” and The “How”
SPECIALTY DRUGS ORPHAN DRUGS
Small Population <500 ~100-300 Patients
Burden of Illness / Population
Impact
Definitive studies Non-definitive studies
New Born Screening Not Required Required
Delayed Diagnosis / Definitive
Diagnosis
Definitive Diagnosis Delayed Diagnosis
Genetic Testing / Component Not Applicable
~80% of Rare Disease Population has a
genetic component
Reimbursement Challenges Established Reimbursement Framework
No clear path, despite other established
markets (FDA, EU)
Financial Assistance Yes Yes, with conditions
Health Outcomes Studies Not Required Required
Integrated / Closed Distribution
Recommended
*Required for Health Outcomes
Measurements and Adherence
5. Specialty Reimbursement through Private Payers
Timelines
• Up to 100
days to gain
access
• Variation
Private vs.
Public
Medical
Policy
• Defined criteria for
use (e.g. clinical
effectiveness,
selected
specialties, 3°
centers, etc.)
• Prior Auth.
• Published
PLA
• Additional conditions
to limit exposure for a
specific payer (e.g.
volume-based price,
capped usage, etc.)
• Prior Auth.
• Terms are usually
confidential
Case
Management
• Reimbursement
decision based on
individual patient
assessment
• Requires prior auth.
• Subject to periodic
review
3/16/16 CONFIDENTIAL5
Cost Containment Is Key
PPNs; Capped Mark-Ups; Pooling...
6. Medavie Blue Cross
• Managing Chronic
Disease Program: 56%
of workforce, ≈
• Self-care approach for
chronic disease;
targeted health services
delivered by specialized
HCPs
• Diabetes Management
Program (pharma
partnership)
Manulife
§ Designed to delay listing
& reimbursement until
CADTH has reviewed
§ Manulife will review
based on its own
schedule
Sun Life
Provincial Integration
Program
Great West Life
• Monitoring a patient for
a specific period of time
to ensure the best
health outcome
Sun Life
Manulife
GreenShield
Medavie
Private Sector Policy Initiatives
3/16/16 CONFIDENTIAL6
Green Shield
Cooperators
Medavie
Sun Life
Manulife
INSURERS
REFERENCING
CADTH/CDR
HEALTH CASE
MANAGEMENT
PPNs
MANAGING CHRONIC
DISEASE
8. Italy
§ Drug-monitoring to asses and track patient
eligibility, evaluate utilization in clinical
practice, collect epidemiological data
(safety data and post marketing info)
§ Meant to guarantee appropriate use of
medicines according to its therapeutic
indication
§ Providing important information on the
tolerability of a new drug and prescribing
appropriateness.
3/16/16 CONFIDENTIAL8
Example of Adaptive Listing Model: Start, Stop Criteria to be negotiated with Manufacturer
9. Challenges with Managed Access Programs
Defining patient
eligibility
• Collecting data
on outcomes
• Who will be
responsible for the
patient registries?
• How will
confidentiality be
maintained?)
Setting stopping
criteria
• Is it possible to
achieve a
consensus on
stopping criteria
when rare diseases
are so
heterogeneous?
• How will decisions
be made for those
who cannot speak
for themselves?
Assigning
stakeholder roles
and
responsibilities
• What roles will
Health Canada,
payers, and
pharmaceutical
companies have?
• How will patient
involvement be
organized?
MAPs must work
for both patients
and decision-
makers.
• To avoid some of
the issues such as
backlash when the
evidence does not
support continued
funding
• There is need to
incorporate
feedback from all
stakeholders,
including patients.
MAPs should
consider all
support
• Support for patients
but not just the
drug alone
3/16/16 CONFIDENTIAL9
10. 3/16/16 CONFIDENTIAL10
The HealthForward Model in Canada
3/16/16 CONFIDENTIAL10
Collaborate with the
patient and their
healthcare team
during all phases of
the patient journey
from diagnosis to
treatment and
beyond
Collecting
meaningful insights
through well-defined
feedback
mechanisms and
action planning
Exceptional patient
and healthcare
professional quality
of care equates to
better overall
adherence
11. Cost-Effectiveness of the HealthForward Model is under
Evaluation
3/16/16 CONFIDENTIAL11
GWL-insured patient
cohort
HCM patient cohort
Non-HCM patient
cohort
Full reimbursement
for six months
Full reimbursement
without limited time
Treatment
responders
Treatment non-
responders
Continuous
reimbursement
Reimbursement
discontinued
Continuous
reimbursement
Reimbursement
discontinued
Ongoing treatment
Treatment discontinued
Treatment adherence
associated with HCM
Ongoing treatment
Treatment discontinued
Treatment adherence
associated without HCM
Accepted abstract at coming 21st ISPOR international conference:
INDEPENDENT FACTORS AFFECTING PATIENT COMPLIANCE TO PRIVATE INSURER-
FUNDED HEALTH CASE MANAGEMENT IN CANADA
13. PSP Evidence Generation to support market access
of orphan drugs
Closing the gap between regulatory needs and payer needs
14. 3/16/16 CONFIDENTIAL14
Considerations for Orphan Program Solutions
Cover certain drugs by therapeutic class based on:
Adaptive Listing Scheme following a framework based on:
• Agreed upon criteria per Orphan category;
• General framework for post marketing surveillance
requirements
Central Registry/PSP
model
• Testing requirements vary – will not be able to
standardize consistent approach
Genetic Testing
requirements
• Link to Payer validated data requirements
• Consider Central Data Collection
• Monitoring tool to provide ability to cover drugs based on
evaluation of outcomes- proactively agreed upon data
points
Data Collection criteria
based on approved
guidelines
3/16/16 CONFIDENTIAL
15. How can We Support the Rare Disease Strategy?
Private
Payer
Manufacturer
Public PayerPublic
Payer
Provide timely, equitable and evidence-informed
care:
Right patient, Right drug, Right time
Providing sustainable access to promising
therapies
Consider offering a unique model based on outcomes
for Rare Diseases
Generate real-world evidence to meet market
access needs
Disease burden studies to demonstrate unmet medical
needs and support advocacy of rare disease