How patient groups can have more

say in the drug approval process

Today’s webinar will begin with a sound check and

introduction at 12:55 p.m. EST.

1

Patient Involvement in the Lifecycle of a Medicine

February 2015

What today is about

• UNDERSTAND the lifecycle of a medicine

• HOW are patients currently involved in the various

steps of this lifecycle?

• WHAT are the upcoming opportunities for patients

to help shape how drugs are approved and

reimbursed?

• HOW can the various review processes evolve to

better meet patients’ needs?

3

Drug Development

Health Canada Review

Patented Medicines Prices Review Board

Health Technology Assessment

Hospitals

Patient Access

Private Drug Plans

Provincial

Drug Plans

First – an overview

of the Canadian

drug review process

Manufacturer clinical trials:

quality, safety and efficacy

Regulatory review:

quality, safety and efficacy

“Pricing” review:

maximum “non-excessive”

price

Funding recommendations:

Comparative safety, efficacy

and cost effectiveness

FUNDING

DECISIONS

Patient criteria

/appropriate use

4

Research and Development

• Participating in clinical trials

• Public registries: ClinicalTrial.gov

and canadiancancertrials.ca

• Getting involved in research

• CIHR’s Strategy for Patient-Oriented Research (SPOR):

• patients provide input on identifying health research priorities and

participate in the design and undertaking of research projects

• Patient and Community Engagement Research (PACER) program:

• joint initiative between Alberta Health Services (AHS) and the Institute

for Public Health at the University of Calgary

• trains patients in research methods so that they can become patient-

engagement researchers and join AHS’ Strategic Clinical Networks in

order to determine how the health system can deliver high-quality,

patient-centred care

5

Health Canada Pilot Project

• Announced by Minister Ambrose in

August 2014

• Targets patient input for approval of orphan drugs

• simulate how input from patients will be gathered and

incorporated into the drug review process once the Orphan Drug

Framework is in effect

• 2 drugs: Hoffmann-La Roche’s drug Gazyva for Chronic

Lymphocytic Leukemia and Hyperion Therapeutics’ drug Ravicti

for Urea Cycle Disorders (UCDs)

• Questions posed to patients: • how the rare disease affects their ability to manage their day-to-day

lives

• what treatments are currently available

• what therapeutic benefits are most important to them

• their risk tolerance for any new treatments

6

Various HTA Processes

• National processes:

• For MOST meds: Canadian Agency for Drugs and Technologies

in Health’s (CADTH’s) Common Drug Review (CDR)

• For cancer meds: Pan-Canadian Oncology Drug Review

(pCODR) – now transferred to CADTH as of April 2014

• Provincial processes: • British Columbia: “Your Voice” program

• Ontario: Ontario Public Drug Programs

and ODPRN’s drug class reviews

• Quebec: l’INESSS 30 day feedback

• Hospitals and Private Payers: • Limited opportunities for patient input

7

National HTA processes

• All provinces participate in CDR and pCODR, except Quebec

8

• Federal drug plans also participate in CDR (not pCDOR)

CDR’s expert committee (CDEC)

makes one of four

recommendations:

pCODR’s expert committee

(pERC) makes one of three

recommendations:

i. List

ii. List with clinical criteria and/or

requirements

iii. Do not list at the submitted price

iv. Do not list

i. Recommend

ii. Consider with conditions

iii. Do not recommend

HTA Expert Committees

• Appointed experts who review the clinical and economic evidence

and patient input

• They are responsible for developing funding recommendations which

are passed on to participating plans

• A typical expert committee is comprised of…

• Pharmacologists / Pharmaco-epidemiologists

• Physicians

• Pharmacists

• Health economists

• Sometimes public/lay voice or patients

9

Provincial Reviews

10

Jurisdictions have their own expert review committees

• some consider the national review again and make a further

recommendation to province

Provinces consider CDR/pCODR recommendation and

account for local circumstances, priorities and budgets

Negotiations on price/criteria of drugs often take place:

• joint negotiations through the pan-Canadian Pharmaceutical Alliance (pCPA),

which includes all provinces except QC - QC recently announced its intention to

join but legislative changes are required first

• individual provincial negotiations

In most cases, the minister of health is the final decision-maker, but

the real decision is delegated to relevant government officials (such

as Ontario’s Executive Director of the Ontario Public Drug Programs)

CDR Patient Input Process (2010)

• Part of the CDR process and fits into review timeframes

• Seeks input via Canadian patient groups

• Guide, you tube videos, on-line submissions

• Template for most medications and template for subsequent subsequent entry biologics (SEBs)

• 35 days deadline for input and CADTH advises that focus should be on “expectations for the new drug and reporting any patient experiences with the drug in review”

• Letters of appreciation with feedback on the submission are sent

• Patient input summaries are included in clinical review reports posted online and patient groups can comment on the summaries before they are finalized

• Entire patient submissions are shared with the expert committee, participating plans and posted online

• Creation of CADTH Patient Community Liaison Forum

• Patient involvement in Scientific Advice Program

• Pilot project underway to obtain input from individual patients and caregivers in cases where there is no patient group (until Aug. 3, 2015)

• CADTH is piloting patient input in therapeutic reviews (until Nov. 30, 2015)

11

Expert Committee Deliberation Steps

1. One public member presents summary of patient group input -

stated values and preferences and issues of patients and/or their

caregivers related to the condition for which the medication is

indicated and its treatment

2. Two discussants present their overviews of the clinical and economic

evidence

3. Members provide input; and CDR staff, including clinical and economic

reviewers, and invited experts provide input as required

4. Deliberation on patient group input, clinical and economic evidence,

and formulate a recommendation and provide reasons for it.

5. Members choose one of four recommendation options

6. Secret ballot voting on the recommendation option and the reasons

for the recommendation; decided by majority of votes

12

13

CADTH 2014

Patient Group Participation at CDR

14

CADTH 2013

pCODR Patient Input Process

• Canadian patient groups are invited to provide input:

• Must be registered

• Mandate must pre-date decision to register

• Only one submission per registered group is permitted

• Must be funded by more than one funder and no funder may provide more than 50% of the group’s funding

• No individual patient submissions are permitted

• Patient groups have two opportunities to provide input:

• at time of review initiation (deadline: 10 business days)

• after an initial recommendation is published (deadline: 10 business days)

• Patients are members of the expert committee – they will summarize and present the patient input • this input is one of four considerations within pCODR’s deliberative framework

• pCODR is working with Canadian Cancer Action Network on a Patient HTA Navigator pilot project • navigator provides support to patient groups to optimize patient submissions

Patient Group Participation at

pCODR

pCODR Merger with CADTH

• pCODR transferred to CADTH in April 2014

• Steering committee has been established with representation

from drug plans and cancer agencies

• pCODR and CDR to remain independent programs

• CADTH now focusing on aligning both processes:

• adopting one set of common guiding principles for both processes

• aligning CDR and pCODR evaluation criteria

• standardizing the recommendation options

• building on efficiencies and best practices from both programs

• CADTH plans to consult stakeholders on these initiatives in

coming months

17

pCODR CADTH

Engagement Opportunities

• pCODR transfer to CADTH:

• Opportunity in coming months for stakeholders to provide input

on common guiding principle, evaluation criteria and

recommendation options to be established for both processes

• Opportunity to also highlight any best practices that should be incorporated or

improvements that should be in both processes

• CADTH’s evaluation of medical devices:

• Plans to refine its approach to health technology assessment of medical devices

throughout the product life cycle

• Potential opportunity to help shape patient involvement process in this context

• pCPA

• New pCPA office expected to be established in Spring 2015

• Roundtables to engage stakeholders about this new coordinating organization suppose

to be held early 2015 – no details on this has yet been released

• CADTH Symposium

• CADTH has started to offer financial support for patient groups wanting to attend the

annual CADTH symposiums

18

How to Increase Patient Voice in the

Lifecycle of Medicines • How and at what stage should patients be involved?

• What are the criteria for successful involvement of patients in

regulatory, HTA and reimbursement processes?

• What is the current impact of patient input to HTA and funding

decisions?

19

• How should patients’ perspectives be

evaluated and factored into the formulary

recommendations and, ultimately, coverage

decisions?

• What are some of the current

obstacles/barriers for successful patient

involvement?

• What are strategies/approaches to address

them?

20

DISCUSSION

21

ANNEXES

CDR – How Patient Input is Used

22

CADTH 2014

pCODR Patient Input Process

5.

Summarize

& Review

with pERC

3.2 Collect

Patient

Advocacy

Group

Input

7.3 Get

Feedback from

Patient

Advocacy

Group

8. Summarize

& Review with

pERC

6. Prepare &

Publicly Post

Initial

Recomm,

Post

Reviews

4.2.

Conduct

Economic

Review

4.1.

Conduct

Clinical

Review

9. Prepare &

Publicly Post

Final Recomm

& Post Input

Direct impact of patient

perspective

Indirect impact of patient

perspective

pCODR 2013

Canadian Cancer Survivor Network Contact Info

Canadian Cancer Survivor Network

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