Upload
others
View
4
Download
0
Embed Size (px)
Citation preview
CADTH Drug Portfolio
Information Session
THURSDAY, NOVEMBER 1, 2018
1:30 P.M. TO 4:00 P.M.
Health Technology Management
1. An increased emphasis on implementation support.
2. A shift in focus from assessment at the point of adoption to
evaluation across the life cycle of a drug or health
technology.
3. Strengthened engagement with stakeholders (patients,
clinicians, citizens, industry).
4. An approach that better supports access, appropriate use,
and affordability.
Pre-market
dialogue
Managed
entry
Post-market
surveillance
Managed
exit
Reassessment
Mission CADTH consistently
delivers credible scientific
evidence and management
strategies that enable the
appropriate use of health technologies.
Vision Canada has a world-class
system for assessing and
managing health
technologies to achieve
better outcomes and value for Canadians.
CADTH Values
• Excellence
o CADTH is trustworthy, delivers what it promises, and exceeds
expectations by focusing on impact to drive better health, better
patient experience, and better value for Canadians.
• Responsiveness
o CADTH understands and meets the needs of its customers in a
timely fashion.
• Collaboration
o CADTH creates and nurtures partnerships with those who produce,
acquire, deploy, and use health care technologies to promote their
appropriate use.
• Transparency
o CADTH makes timely and user-friendly information about its
programs, processes, and performance widely available, with a
special emphasis on engaging key stakeholders.
CADTH Strategic Plan 2018-2021
1. Close the gap between evidence,
policy, and practice.
2. Adopt a life-cycle approach to health
technology assessment.
3. Anticipate health system and
technology trends and develop agile
management strategies.
Strategic Issues
1. The pan-Canadian Health Organization
Implementation Steering Group
2. The Advisory Council on the Implementation
of National Pharmacare
3. The Health and Biosciences Economic
Strategy Table
Session Objectives
• CADTH is committed to ongoing, two-way
communication with stakeholders.
• Today is an opportunity to learn about:
o CADTH’s Pharmaceutical Reviews Portfolio
o recent program updates from CDR and pCODR
o other CADTH initiatives
o key topics of interest to you during the Open Forum.
CADTH Drug Portfolio Information Session
It is not an opportunity
to focus on PMPRB!
Overview of the Agenda TOPIC LEAD
Welcome and Strategic Overview Brian O’Rourke
Pharmaceutical Reviews Portfolio a) Overview b) Program Updates from CDR and pCODR
Brent Fraser Alexandra Chambers Trevor Richter
New Initiatives a) Consultations on Complex/Highly Specialized Therapies b) Implementation Advice — Cancer Drug Implementation
Advisory Committee (CDIAC) Transition c) CAR T-Cell Therapies d) Real-World Evidence
Heather Logan Tarry Ahuja
Scientific Advice Michelle Mujoomdar
Stakeholder Engagement Michelle Mujoomdar Harindra Wijeysundera Brian O’Rourke
Open Forum CADTH Team
Wrap Up Brian O’Rourke
Presenters
• Brian O’Rourke, President and CEO
• Brent Fraser, VP, Pharmaceutical Reviews
• Heather Logan, A/VP, Pharmaceutical Reviews
• Alexandra Chambers, Director, CADTH pan-Canadian
Oncology Drug Review
• Trevor Richter, Director, CADTH Common Drug Review
and Optimal Use
• Tarry Ahuja, Manager, Program Development, RWE Lead
• Michelle Mujoomdar, A/VP, Evidence Standards
Questions
• Questions of clarification after each agenda item
• Open Forum at the end of the session
• In-person:
o Please use a microphone for the benefit of on-line
participants
• On-line:
o Use the question feature on the webinar toolbar
Pharmaceutical Reviews Portfolio a) Overview
b) Program Updates From CDR and pCODR
CADTH Pharmaceutical Reviews
Transparency Initiatives • Enhancing transparency is an important objective of
CADTH’s 2018-2021 strategic plan.
• Regulatory authorities, including Health Canada, are
engaged in initiatives to disclose the clinical data used in
their review processes.
• CADTH is similarly committed to increasing transparency in
our drug review processes.
• CADTH is currently unable to disclose information deemed
confidential by drug companies, but we are planning to
initiate discussions with stakeholders with the goal of
ensuring that all clinical and economic data can be
disclosed as part of our review processes.
CADTH Pharmaceutical Reviews
Collaboration and Partnerships • CADTH continues to collaborate with key partners including:
o Canadian Blood Services
o Pan-Canadian Pharmaceutical Alliance
o Canadian Association of Provincial Cancer Agencies
o Regulatory Review of Drugs and Devices (R2D2).
• CADTH is pleased to be an important contributor to Health Canada’s plan to
improve the affordability, accessibility, and appropriate use of prescription drugs.
• These initiatives have brought the regulatory and HTA agencies together in an
effort to find efficiencies and improve the Canadian pharmaceutical review
processes.
• This new era of collaboration has fostered closer communication and alignment
between Health Canada, CADTH, and INESSS.
• There has been considerable progress in the past year and plans are in place to
continue this engagement.
Health Canada/HTA Alignment
• Aligned review process implemented by CADTH,
INESSS, and Health Canada in June 2018
• Pre-NOC submissions and information-sharing
agreement will reduce delays in access
• Plan to re-evaluate after one year to determine
how to expand and enhance the process
• Timing of pre-NOC submissions (180 days)
• CADTH Collaborative Workspace
• New biosimilar review process
• New CADTH Pharmaceutical Reviews Update newsletter
• New aligned review process with Health Canada
• Aligned duration of submission screening periods
• Alignment of resubmission criteria
• Continued cross-training and integration of infrastructure
CDR and pCODR Alignment (Past Year)
Pharmaceutical Reviews Portfolio a) Overview
b) Program Updates From CDR and pCODR
Updates From CADTH’s Common
Drug Review (CDR) and Optimal
Use of Drugs Programs
CDEC Update • Call for CDEC nominations was issued in 2018
• CADTH thanks all outgoing CDEC members:
o Anatoly Langer, Kerry Mansell, Silvia Alessi-Severini
Current CDEC members:
James Silvius (Chair) Peter Jamieson
Ahmed Bayoumi Allen Lefebvre
Bruce Carleton Heather Neville (New)
Alun Edwards Rakesh Patel (New)
Bob Gagne Emily Reynen (New)
Ran Goldman Yvonne Shevchuck
Allan Grill Adil Virani
Volume and Performance Metrics
0
10
20
30
40
50
2011 2012 2013 2014 2015 2016 2017 2018
Resubmission RFA Submission (YTD)
• 100% compliance with performance metric (i.e., ≤ 180 days)
CDR Pre-NOC Submissions
7% 12%
36% 38% 43%
62%
53% 57%
0%
10%
20%
30%
40%
50%
60%
70%
2011 2012 2013 2014 2015 2016 2017 2018
Currently > 50%
• Revised resubmission criteria November
• Launch of Collaborative Workspace
• New biosimilar review process February
• Update Guidance on Application Fees
• Expansion of pre-NOC process March
• Expansion of pre-submission meetings May
• Aligned reviews with Health Canada and INESSS June
• New CDR Procedures and Guidelines
• New Therapeutic Review Framework
• New Pharmaceutical Reviews Update
August
• CADTH/INESSS clinician engagement pilot September
• Request for clarification to be posted
• New template for reconsiderations October
CDR Program Updates (At a Glance)
• CDR now accepts submissions six months pre-NOC
• Information-sharing agreement with Health Canada
• Allows alignment of CADTH and Health Canada reviews
CDR Pre-NOC Submissions
Health Canada Review (Priority) CADTH Review Health Canada Review (AC NOC/c) CADTH Review Health Canada Review (Standard) CADTH Review
0 30 60 90 120 150 180 210 240 270 300
Days from Acceptance of Review by Health Canada
New Procedure Document
• Single document created to consolidate
CDR procedure and guidelines
• Will be routinely updated to reflect any
revisions
• New CADTH Pharmaceutical Reviews Update launched in August
2018
• Consolidated publication that will be used to communicate updates
regarding all of CADTH’s drug review processes
• Newsletter will be issued at the same time each month for greater
predictability (every second Thursday, as required)
• Replaces separate communications for patient groups, CDR, pCODR,
Therapeutic Reviews, Optimal Use of Drugs
• Older updates being removed and archived
o Transparent approach to avoid confusion regarding out-of-date materials
(e.g., web searching can lead to old templates)
Planned and Ongoing Initiatives
• Enhanced clinical engagement o Clinical expert panels
o Recruiting clinical specialists
• Drug program consultations o Strengthening reimbursement criteria
o Non-submissions by manufacturers
o Posting comments and responses
• Other initiatives o Improving transparency
o Seeking efficiencies
Clinical Panels (Review Phase)
• Part of broader plan for strengthening the process for
establishing reimbursement criteria in recommendations
• Panels will assist in characterizing the following: o Unmet therapeutic need
o Gaps in the evidence and opportunities for data collection
o Potential implementation challenges
o Clinical management of patients
o Potential place in therapy of the drug
• Plan to target drugs that undergo an accelerated review
by Health Canada (i.e., priority review or NOC/c at outset) o Promote alignment through the health system
CADTH-INESSS Pilot Project
• CADTH and INESSS launched a pilot for the joint
engagement of clinical specialists in September 2018
• Objectives of the pilot project are as follows: o Strengthen the current pharmaceutical review processes
with respect to engagement with clinical specialists.
o Pilot is targeting drugs that are likely to be associated with
complex evaluations and challenging deliberations.
o Promote efficiency within Canadian HTA processes by
reducing duplication of effort.
• Attendance at clinical panel meetings will be limited to
the clinical specialists, key expert review committee
members, and key CADTH and INESSS staff.
Clinical Panels (Implementation Phase)
• Part of CADTH’s strategic plan to provide enhanced
implementation support
• For certain drugs, the jurisdictions have indicated there is a
need for additional support from clinical panels to assist in
the development and refinement of reimbursement criteria
and conditions after the completion of CADTH’s review.
• CADTH is currently engaged in developing a process to
facilitate these panels
• Currently distinct from CDR process, but may be integrated
into the process as a formal “implementation phase”
Recruitment of Clinical Specialists
• CDR is planning to issue Calls for Clinical Specialists at
the same time we post the call for patient input
• We hope this initiative could assist CADTH in identifying
experts who could participate in our review processes
• Clinicians are selected based primarily on the following: o Expertise regarding the diagnosis and management of the
disease for which the drug is indicated
o Conflict of interest declaration
o Availability to commit to review timelines
• CADTH is currently investigating the logistical requirements
for this initiative and is planning to release further details in
winter 2019
Consultation: Reimbursement Criteria
• Drug plans requested greater detail and consistency in the
initiation, renewal, and discontinuation criteria included in
CDEC recommendations, particularly for “complex” drug
treatments
• Stakeholder feedback supports additional clinician
engagement
• CADTH has begun integrating clinical expert panels into the
CDR review process
o Details will be communicated through a CADTH Pharmaceutical
Reviews Update
• Options being considered regarding the other aspects (e.g.,
manufacturer submitted criteria)
Consultation: Non-Submissions
• Challenges when manufacturers do not file submissions, but the drug
plans are receiving requests for coverage
• Proposal included a formalized process for reaching out to manufacturers
regarding submissions for eligible products
• Also includes providing drug plans with a public statement regarding the
status of the product; e.g., a statement on the CADTH website to indicate
that CADTH is unable to recommend reimbursement because a
submission was not filed
• No major opposition to this proposal and CADTH will be moving ahead
with the proposed changes
• An implementation plan is being developed
o Details will be communicated through a CADTH Pharmaceutical Reviews
Update
Consultation: Posting comments
• CADTH responses to the manufacturer comments are shared with the
manufacturer, CDEC, and the drug plans, but not publicly posted
• Proposal aimed to publicly post feedback from manufacturer on draft
CDR review reports and the CADTH responses to feedback
• Feedback raised concerns regarding posting potentially erroneous
information and insufficient time for review
• CADTH agrees with maintaining status quo (posting final rather than
draft; not extending review timelines)
• Further dialogue required prior to implementation
Optimal Use Update
• CADTH has been working with jurisdictions to
revise the Optimal Use program
• Goal is to substantially increase the number of
optimal use projects conducted each year
• Progress to date:
o Restructuring of advisory group
o New OU products are being developed
o Optimal Use Information Network is being established
Reminders
• Please consider info-sharing initiative with Health Canada.
• Always use [email protected] for inquiries to ensure
tracking, triage, and record keeping.
• Consultants must copy an official contact for the
manufacturer on all email correspondence with CADTH.
• Ensure templates are obtained exclusively from the
CADTH website and are the latest versions posted.
• CADTH does not provide guidance on hypothetical
scenarios (e.g., “What if we had a product that was…”).
o Proactively fill out the Submission Eligibility Form when inquiring to
help speed up the process.
Updates From CADTH’s pan-
Canadian Oncology Drug Review
(pCODR) Program
pCODR Expert Review Committee
(pERC) Membership
• Maureen Trudeau (Chair)
• Catherine Moltzan (Vice-chair)
• Daryl Bell (New)
• Kelvin Chan
• Flay Charbonneau
• Matthew Cheung
• Winson Cheung
• Henry Conter (New)
• Avram Denburg
• Leela John
• Anil Joy
• Christine Kennedy
• Christian Kollmannsberger (New)
• Cameron Lane
• Valerie McDonald
• Marianne Taylor
• Dominika Wranik (New)
CADTH thanks the following outgoing members of pERC for their hard work
and dedication through the years: Carole McMahon, Craig Earle, Mike Doyle
Current pERC Members
New CADTH Product: CADTH
Technology Review
https://www.cadth.ca/metastatic-melanoma-gap-analysis
Key Metrics for pCODR Program
Total Number of Submissions Received by pCODR Since July 2011
June 30, 2018
0
5
10
15
20
25
30
35
3 8 11
6 11
9 9
7
1
5 5
8
9 12 13
14
2
1 1
1
1 2 3
1
1
Request for Advice
Resubmission
Expanded Scope
New Indication
New Drug
Note: CADTH’s Projected Production Capacity for pCODR: 20 to 25 submissions per year
pERC Final Recommendation pCODR has issued 115* notification
to implement as of September 30,
2018
10 (9%) recommend to reimburse
81 (71%) recommend to reimburse
with clinical criteria and/or
conditions
23 (20%) do not recommend to
reimburse
*Note: pERC issued notification for 1
Request for Advice
9%
71% 20%
Column1
PositiveRecommendation
ConditionalRecommendation
NegativeRecommendation
Patient and Clinician Input
0
20
40
60
80
100
120
140
Patient Input Clinician Input
125
56
4
6
# of pCODR Reviews
With Input Without Input
Please note: pCODR started collecting clinician input in 2016, while patient input has been part of the pCODR process since 2011.
CADTH and Canadian Cancer
Action Network (CCAN) HTA Project
Collaboration
Continued engagement with patient groups in HTA
processes for oncology drugs in Canada
Cancer Drug Pipeline Information for
Patient Groups
• Searchable cancer drug information pipeline database
http://www.ccanceraction.ca/pipeline/
Qualitative Studies: What is
meaningful patient engagement?
Part 2…
• Aim: To explore the experiences of patient advocacy
groups participating in the pCODR process with a view to
articulating the strengths, opportunities for improvement and
future development of patient engagement at pCODR
• Study Design: o Qualitative interview study
o All registered patient groups were invited (N=44)
o Participants: N=22 individuals
Characteristics of Participants/
Organization
Themes from the Patient Groups
1. Making submissions is a priority
2. Resourcing/logisitics
3. Locating relevant patients It’s not difficult per se to find the patients, but it’s really difficult to find the
patients who’ve used these drugs for that indication.
4. Perceived impact of patient input
It’s meaningful as a patient representative to be able to say to the Canadian
patients that we’re going to represent your voice, especially the voices of
people who’ve been on clinical trials. To be able to give them meaning for
having gone through that is meaningful in a personal way as a patient
representative.
Thank you to everyone who so generously shared their
perspectives for the study.
Expanding pCODR’s Clinician
Input/Feedback Process
Expanding Clinician Input/Feedback
• pCODR updated the clinician input process in 2018,
expanding the process to include:
o oncology physicians, pharmacists, and nurses; and
o a designated clinician input form for each drug for
upcoming drug reviews.
• This initiative allows for broader clinician participation in
providing and enhancing value-added information in the
discussion of drug-funding decisions in Canada.
A New Resource for Clinicians
Acknowledgement: This resource is made possible through a joint collaboration by CADTH (pCODR program), Innovative Medicines Canada (Joint Oncology Project Team), and Lymphoma Canada. https://cadth.ca/pcodr/clinician-input-and-feedback
Reassessments
Reassessments—Background
• Cancer Quality Council of Ontario’s 2015 Programmatic
Review recommended:
• A consistent process for disinvestment (or “reinvestment”) and
renegotiation of prices with buy-in from the public, patients and
clinicians should be explored (i.e., delisting drugs should be
considered in tandem with the prioritization of new drugs)
• CADTH’s Strategic Plan 2018-2021:
• Adopt a Life-Cycle Approach to Health Technology Assessment
• Recently, pERC has made some recommendations that
reference time-limited funding
Reassessments—What’s next?
• CADTH Reassessment Working Group • Streamlining and adapting current review mechanisms to accommodate
reassessment reviews
• CanREValue Project (CIHR-PHSI grant led by Kelvin Chan) • Exploring RWE in cancer drug funding decisions, and developing a
framework for provinces to generate and use RWE in a consistent and
integrated manner
• Multi-stakeholder initiative that will require a series of
consultations over the next year
New Initiatives a) Proposed Supplemental Process for
Highly Specialized/Complex Therapies
b) Implementation Advice — Canadian Drug
Implementation Advisory Committee
(CDIAC) Transition
c) The Assessment of CAR T-Cell Therapies
d) Real-World Evidence
Proposed Supplemental Process for Highly
Specialized / Complex Drugs
• In 2014, provincial/territorial health ministers established the
Expensive Drugs for Rare Diseases Working Group (EDRD WG)
to explore management of rare disease drug therapies.
• A proposal for a Supplemental Process has been drafted.
• External stakeholder consultation begins in November. Written
submission process will be confirmed following consultation.
• CADTH’s role: Facilitating the external consultation process and
working to continue to enhance existing review processes to
support our customers.
• Questions: [email protected]
New Initiatives a) Proposed Supplemental Process for Highly
Specialized/Complex Therapies
b) Implementation Advice — Cancer Drug
Implementation Advisory Committee
(CDIAC) Transition
c) The Assessment of CAR T-Cell Therapies
d) Real-World Evidence
Implementation Advice + CDIAC
A commitment to harmonize,
streamline, and transition
About CAPCA
BC Cancer Dr. Malcolm Moore
Alberta Health Services Nancy Guebert
Saskatchewan Cancer Agency Dr. Jon Tonita
CancerCare Manitoba Dr. Sri Navratnam
Cancer Care Ontario Dr. Michael Sherar
Direction québécoise de cancérologie Dr. Jean Latreille
New Brunswick Cancer Network Dr. Eshwar Kumar
Nova Scotia Health Dr. Drew Bethune
PEI Cancer Dr. Philip Champion
Eastern Health Cancer Care Program Elaine Warren
Our Partners CADTH/pCODR Pan-Canadian Pharmaceutical Alliance (pCPA) Canadian Partnership Against Cancer (CPAC)
The Canadian Association of Provincial Cancer Agencies (CAPCA) is an inter-provincial organization of provincially designated cancer programs.
Drug Funding Sustainability
CAPCA works with partners to improve access to cancer drugs with a meaningful patient benefit to ensure that the most effective drugs are available as quickly as possible.
Tremendous progress has been made in how cancer drugs are evaluated, priced, and used, though opportunities to continue to enhance the current system remain.
The Cancer Drug Implementation Advisory Committee (CDIAC) has been a critical mechanism in CAPCA’s collaborative work to create a more sustainable cancer system.
About CDIAC
Role Contribution Membership
• Help identify and provide advice on how new drugs can be integrated into existing treatment pathways.
• Advice provided to
provincial ministries of health and pCPA following approval by CAPCA Board of Directors.
• Achieves greater consistency in drug-funding decisions across Canada.
• Carefully considers
provincial and pan-Canadian affordability and budget impact to understand financial implications of various funding options.
• Chair a CAPCA Board member.
• Multi-disciplinary: provincial pharmacy leads, medical oncologists, ethicist, and health economist. Patient, family, and public representatives.
• Engages disease site-specific clinicians as needed.
• Quebec serves as an observer.
A Positive Impact
• Helping to enhance clinician input into how new drugs are implemented
• Supporting consistency in provincial oncology drug listing
• Without unnecessarily delaying patient access to new therapies
Strengthening the Work • To strengthen the work, CAPCA and CADTH are discussing how key
components of the work can be transitioned from CAPCA to CADTH.
• CAPCA will continue to partner with CADTH, provinces, and territories to ensure a sustainable, high quality cancer system that provides timely access to the most effective cancer drugs by:
• providing guidance regarding provincial representation
• endorsing implementation recommendations
• providing clarity regarding issues relating to health human resources, administration, and treatment delivery
• continuing as role of conduit to cancer programs and ministries of health
• providing support to cancer programs on pan-Canadian message development and information sharing.
Decisions to Date
No decisions have been made about how any new
implementation advisory responsibilities would be
operationalized at CADTH, however:
• We would not necessarily adopt CDIAC as is. We would consider a
variety of possible options.
• Although initially our focus would be on cancer drugs, the new
implementation advisory function could expand over time to include
non-cancer drugs.
Expert Panel
• Purpose: To develop an implementation-focused
recommendation deliberative framework and provide advice
about process and operations of this new area of work
• The Deliberative Framework Expert Panel includes:
o three practicing clinicians: two medical oncologists plus one
hematologist
All three clinicians have had experience as members of pERC.
All come from different provinces.
o three clinician researchers and/or academics:
health technology assessment
implementation science
decision support and stakeholder engagement in decision-making
o provincial pharmacy operations.
Timeline Oct 2018 Deliberative Framework Expert Panel
convened
Nov 1, 2018 Official announcement of agreement in principle
Dec 2018 to Feb 2019 Consultation with stakeholders
Mar 2019 Rollout of new, expanded implementation
advisory role at CADTH
Apr 1, 2019 New, expanded implementation advisory work
begins
Note: Between now and March 31, 2019, CDIAC will continue to operate as is, under
CAPCA.
New Initiatives a) Proposed Supplemental Process for Highly
Specialized/Complex Therapies
b) Implementation Advice – Canadian Drug
Implementation Advisory Committee
(CDIAC) Transition
c) The Assessment of CAR T-Cell Therapies
d) Real-World Evidence
Overview
• CAR T-cell therapy overview
• Medical devices portfolio
• Health Technology Expert Review Panel (HTERP)
• Assessment process for CAR T-cell therapies
CAR T-Cell Therapy — Overview
Medical Devices
• Medical devices
• Diagnostic tests
• Surgical procedures
• Dental procedures
• Medical procedures
• Clinical interventions
Optimal Use
• HTA with recommendations
• Policy and practice decisions
• Inform appropriate use
• Stakeholder engagement
• Expert involvement
• Recommendations developed by an expert committee
• Knowledge mobilization
Optimal Use Components
Clinical Context/ Need
Benefits and Harms Economic Impact
Patient Perspectives Ethics
Implementation
Legal
HTERP Deliberative Framework
Clinical Context/ Need
Benefits and Harms Economic Impact
Patient Perspectives Ethics
Implementation
Environmental Impact
Legal
Health Technology Expert Review
Panel (HTERP)
• Seven core members
o Chair
o Public member
o Ethicist
o Health economist
o Three health care practitioners
• Up to five expert members
Timeline
Step 1: Project Planning Activities
Step 2: Screen Information Submitted and Initiate Review
Step 3a: Collect Patient Group Information
Step 3b: Collect Registered Stakeholder Input
Step 4: Conduct Clinical, Economic and Implementation
Review and clarify information with submitter
Step 5: Targeted Patient and Stakeholder Group
Feedback
Step 6: Summarize and Review with HTERP
Step 7: Get input from Patient Groups and Stakeholders
Step 8: Summarize and Review With HTERP
Step 9: Prepare and Publicly Post Final Recommendation
Submitting Input
https://drugreviewsadmin.cadth.ca/Landing/Register/Register.
aspx?token=CART
New Initiatives a) Proposed Supplemental Process for Highly
Specialized/Complex Therapies
b) Implementation Advice – Canadian Drug
Implementation Advisory Committee
(CDIAC) Transition
c) The Assessment of CAR T-Cell Therapies
d) Real-World Evidence
What is Real-World
Evidence (RWE)?
What is RWE?
Real-world evidence (RWE) is evidence derived from the analysis and/or synthesis of real-world data (RWD).
RWD is an overarching term for data that are not collected in the context of conventional randomised controlled trials (RCTs).
What is RWE?
• Randomized controlled trials (RCTs) have been the gold standard
• Patients are quite homogenous • Data collected are closely controlled and monitored • High internal validity, low external validity
* adapted from www.nehi.net
What is RWE?
• Data comes from various non-RCT sources • Patients are quite heterogenous • Data collected from real-world practice settings • Low internal validity, high external validity
* adapted from www.nehi.net
CADTH and RWE
CADTH and RWE
• CADTH currently uses RWD/RWE in different applications
• Different sources of evidence will be used to inform different
policy/research questions
• Follow the hierarchy of evidence — RWE will not replace RCTs
• RWE will be used with the product life-cyle approach
• RWE will continue to be used and developed for both drugs and devices
RWE Summit
Defining “Decision-Grade” Real-World Evidence
and its Role in the Canadian Context:
A Design Sprint
October 21, 2018, in Toronto, Ontario
RWE Summit
• Multi-stakeholder RWE workshop held at CAPT meeting (Oct. 2018)
• Organizing partners included: CADTH, Health Canada, IHE, CAPT
• Participants included: Academia, researchers, clinicians, patient
representatives, payers, data providers, and industry
• Used a design sprint model to make the meeting actionable
RWE Summit
• The objectives of the workshop were:
o to identify the value and applications of RWE in supporting
pharmaceutical regulatory and reimbursement decision-making
o to identify the conditions upon which RWE will be considered of
sufficient quality to inform decision-making.
RWE Summit
• Participants worked on two case studies with discussion period
• Meeting summary currently being drafted (Nov/Dec 2018)
Next Steps
Next Steps
• CADTH is currently drafting an RWE Action Plan
• Consultation and collaboration with Health Canada to generate an
RWE Framework document in spring 2019
• Several touch points for consultation and stakeholder feedback during
the process
Scientific Advice
Early Scientific Advice at CADTH
• Launched in January 2015
• Advice on early drug development plans from a Health Technology Assessment perspective, with emphasis on the Canadian setting
• Voluntary, non-binding, fee for service, cost-recovery program
• Eligibility: Before initiation of pivotal trials (Phase II or Phase III)
International Landscape
Geographic Region
HTA Agency / Regulatory Body
Year
Europe EMA-EUnetHTA July 20172
UK NICE-MHRA 20103,4
Australia PBAC-TGA (pilot) May 20095
Geographic Region
Country / HTA Agency / Program
Year of Inception
Europe United Kingdom (NICE) Spain (Regional) Sweden (TLV-MPA) Germany (GBA) Italy (AIFA) France (HAS) Netherlands (ZINL, ZINL-CEB) EUnetHTA pilots European Commission
2009 2010 2009 2012 2011 2012 Tapestry: 2010 2011-15 2014
North America
Canada (CADTH) Green Park Collaborative (USA) MaRS EXCITE (Canada)
2015 2011 2011
HTA Scientific Advice Programs1 Early Parallel Scientific Advice
References
1. http://pharmaphorum.com/views-and-analysis/needs-early-advice/
2. http://www.ema.europa.eu/ema/index.jsp?curl=pages/regulation/g
eneral/general_content_001857.jsp&mid=WC0b01ac0580a11c96
3. https://www.nice.org.uk/about/what-we-do/scientific-advice/nice-
mhra-scientific-advice
4. Wonder. Expert Rev Pharmacoecon & Outcomes Res
2014;4:465-7.
5. Wonder et al. Value in Health 2013;16:1067-73.
Types of Advice Requested
Clinical Trial Design Economic Analysis
Target trial population and subgroups
Trial design and duration
Choice of trial comparators
Primary and secondary end points;
surrogate outcomes
Patient-reported outcomes
Health-related quality of life measures
Population(s)
Choice of comparators
Choice of economic model
Data to populate the model
Utility values
Time horizon and extrapolation hypotheses
Resource utilization data
CADTH Scientific Advice Timelines
Standard Timelines
*Flexibility and customized timelines available.
Patient Involvement in CADTH Scientific Advice Process
• Individual patient with the condition is invited to an interview:
o Patient journey from diagnosis, symptoms over time
o Treatment experiences, challenges
o Most significant health issues related to the condition that impact daily life
o What is hoped for in a new treatment
• Patient interview summary and key points from past patient group input provided to the company and incorporated into the advice
CADTH Scientific Advice Experience To Date
0
1
2
3
4
5
6
2015-2016 2016-2017 2017-2018 2018-2019
Among 14 SA procedures, 5 companies returned a second time
Fiscal Year *Completed or in progress
*
Nu
mb
er
of
SA p
roce
du
res
com
ple
ted
CADTH/NICE Parallel Scientific Advice
• Parallel procedure in progress with NICE Scientific Advice (UK)
• Joint Scientific Advice Meeting held with CADTH/NICE/company
• Preliminary process; further details will be available later in the year
• Interested companies can contact either CADTH or NICE:
Future of Scientific Advice in Canada
• Exploring CADTH / Health Canada Parallel Scientific Advice
• Opportunity for companies to discuss any divergent advice in real time between regulator and HTA in Canada
• Updates to be provided
Stakeholder Engagement a) Patients and patient groups
b) Clinicians and clinical societies
c) Industry
Stakeholder Engagement a) Patients and Patient Groups
b) Clinicians and Clinical Societies
c) Industry
Overview
Where we can go: “Listening Exercise” with patient groups, committee members, and CADTH directors
Where we are now: Mapped with CADTH Patient Engagement Framework
Guiding us there: Patient and Community Advisory Group
Why Engage Patients
HTAi Patient & Citizen Involvement Interest Group www. htai.org
• Relevance
• Fairness
• Equity
• Legitimacy
• Capacity building
Why Engage Patients
• Patients actively contribute their insights to the Common
Drug Review, pan-Canadian Oncology Drug Review,
Optimal Use and Scientific Advice programs.
• Rapid Response and Optimal Use reports may include an
analysis of published literature on patient perspectives.
Relevance Patients have knowledge, perspectives, and experiences that are unique and contribute to essential evidence for HTA.
HTAi Patient & Citizen Involvement Interest Group www. htai.org
Why Engage Patients
• Patients can comment on proposed process changes,
participate in evaluations of CADTH, and contribute to
strategic direction.
• Comment on draft pERC recommendations and Optimal
Use recommendations produced by CDEC and HTERP.
Fairness Patients have the same rights to contribute to the HTA process as other stakeholders and have access to processes that enable effective engagement.
HTAi Patient & Citizen Involvement Interest Group www. htai.org
Why Engage Patients
• Patients, families, and communities can offer insights on the
diversity of needs across Canada.
• CADTH deliberative frameworks include multiple
perspectives.
Equity Patient involvement contributes to equity by seeking to understand the diverse needs of patients with a particular health issue, balanced against the requirements of a health system that seeks to distribute resources fairly among all users.
HTAi Patient & Citizen Involvement Interest Group www. htai.org
Why Engage Patients
• Groups provide input to CDR and pCODR and identify
patients with specific expertise to contribute to Scientific
Advice, Optimal Use, Environmental and Horizon Scans.
• Recommendations publicly detail how patient perspectives
were considered to reach conclusions.
Legitimacy Patient involvement facilitates those affected by the HTA recommendations to participate in the HTA; contributing to the transparency, accountability, and credibility of the decision-making process.
HTAi Patient & Citizen Involvement Interest Group www. htai.org
Why Engage Patients
• Travel awards to CADTH symposium.
• Dedicated patient engagement team.
Capacity Building Patient involvement processes address barriers to involving patients in HTA and build capacity for patients and HTA organizations to work together.
HTAi Patient & Citizen Involvement Interest Group www. htai.org
Listening Exercise
Patient Community
Liaison Forum
9 CADTH directors
4 patient and public committee members from pERC, CDEC, and HTERP
21 patient groups involved with CADTH
How can we greater involve
patients, patient groups, and
communities in our work?
We Heard: Greater Involvement
• Meaningful, respectful engagement
• Need for greater diversity of voices
• Greater interaction with expert committees and CADTH
researchers
• Involvement in CADTH governance
• Input and engagement measured to demonstrate impact
• From Patient Community Liaison Forum — involvement in
process design
• Mixed views on capacity for involvement in re-assessment
We Heard: To Be Supported
• Travel awards to CADTH symposium much appreciated
• Clear guidance on what is helpful or seen as biased
• Awareness raising of CADTH and role for patient
perspectives in assessments
• Help preparing / refining patient input
Our Guide: Advisory Group
• Advise CADTH across all programs
• Patient and community — broader than disease-specific
• Lived experience highlighted as essential
• Selected by nominations to meet core competencies
• Agreement that diversity of perspective, accountability to
the community represented, and transparency (financial
and meeting competencies) are important
• Nominations open around end of year, running into early
2019
Stakeholder Engagement a) Patients and Patient Groups
b) Clinicians and Clinical Societies
c) Industry
Stakeholder Engagement a) Patients and Patient Groups
b) Clinicians and Clinical Societies
c) Industry
Open Forum
Wrap Up