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 requests@cadth.ca 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: heatherl@cadth.ca

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

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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:

• scientificadvice@cadth.ca

• scientificadvice@nice.org.uk

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