This document summarizes a presentation given by Maureen Smith on patient involvement in drug coverage reviews in Ontario. It outlines how patient groups can submit evidence to be considered by the Committee to Evaluate Drugs, including registering as a patient group, using the submission template, and meeting submission deadlines. It also provides suggestions for making submissions more impactful, such as prioritizing the most important impacts of a disease and treatment outcomes. The goal is to systematically incorporate the patient perspective into drug review and funding decisions in Ontario.

1. Patient Input: What is asked for and
what is helpful in a patient
submission
Cathy Evanochko
Co-Chair
Tuberous Sclerosis Canada
Sclérose Tubéreuse;
CORD, Board member
Maureen Smith
Patient Member
Committee to Evaluate Drugs
Ontario Ministry of Health &
Long-Term Care
March 7, 2015

2.  Tuberous sclerosis complex (TSC)
 tumours develop in multiple organ systems including
brain, heart, kidneys, lungs, skin, eyes
 complications can include: epilepsy, kidney failure, heart
arrhythmia, lung failure, cognitive disability and mental
illness issues, facial disfiguration
 Treatment for symptoms
 seizures – medication, surgery
 tumours – surgery
 nothing
How did we get here?
May 7, 2015

3.  drug re-purpose – Everolimus (RAD001) - an inhibitor
of mammalian target of rapamycin (mTOR)
 tumour shrinkage
 clinical trials
 Cincinnati, here we come
May 7, 2015
New adventure

4.  FDA approval for treating kidney AML – August, 2012
 clinical trial ended August, 2013
 Health Canada approval for treating kidney AML – October,
2012
 Patient input – Canadian Agency for Drugs and Technologies
in Health (CADTH) – Common Drug Review (CDR) negative
recommendation – September, 2013
May 7, 2015
Now what?

5.  We had 6 weeks from notification to submit input
 patient survey
 information specific to renal AMLs
 compilation of survey results, what is meaningful?
 challenges
 few individuals with any knowledge of the drug
 progression of disease – wide range of affectations –
not everyone has kidney tumours
 responses limited to responding to questions – online
process
May 7, 2015
Patient submission

6.  and then we waited…
May 7, 2015
April - September

7.  CDEC FINAL RECOMMENDATION
 EVEROLIMUS
 (Afinitor — Novartis Pharmaceuticals Canada)
 Indication: Renal Angiomyolipoma Associated with Tuberous Sclerosis Complex
 Recommendation:
 The Canadian Drug Expert Committee (CDEC) recommends that everolimus not be
listed.
 Reason for the Recommendation:
 In one double-blind, randomized controlled trial (RCT), everolimus was shown to
reduce the size of angiomyolipomas (AML) in 42% of treated patients. However, it has
not been definitively established that a reduction in AML size is correlated with a
reduction in bleeding complications, avoidance of surgery, or long-term preservation of
renal function.
 Of Note:
 CDEC considered subpopulations of patients for whom everolimus could be
recommended (e.g., those who are experiencing AML growth and who are not
candidates for surgery); however, there was no evidence for making such
recommendations.
May 7, 2015
Who knew?

8.  Patient input
 CADTH – SEGA
 Ontario, BC – AML, SEGA
 Ontario EAP recommendation March, 2014 - access criteria
– November 7, 2014
 Special access – Alberta, Manitoba, Quebec
 What next?
May 7, 2015
The journey continues

9. Patient Involvement in Drug
Coverage Review
Ontario Public Drug Programs
Patient Evidence Submissions
Maureen Smith
Patient Member
Committee to Evaluate Drugs
Ontario Ministry of Health and Long-Term Care
March 7, 2015

10. Ontario Drug Benefit (ODB) Program
 Approximately 3,400 drugs listed in the Drug Benefit
Formulary/Comparative Drug Index (Formulary), and an additional
850 through the Exceptional Access Program.
 Provides drug benefits for Ontarians who are:
 65 years of age or older;
 Residents of long-term care homes and homes for special care;
 Recipients of professional home care services;
 Recipients of social assistance, including Ontario Works and
Ontario Disability Support Program;
 Recipients of the Trillium Drug Program
Ontario Public Drug Programs
(OPDP)

11. Ontario’s Drug Review & Funding Process
Health Canada issues market authorization
Final funding decision made by Executive Officer
Common Drug Review (CDR)
CDEC recommendation to drug plans
Manufacturer submits clinical & economic evidence
Ontario’s CED conducts review in the Ontario context.
CED provides recommendation to Executive Officer.
New Drugs / Drugs with new indications (non-cancer)
CDEC = Canadian Drug Expert Committee
CED = Committee to Evaluate Drugs
pCODR
OthersCancer drugs
pCODR = pan-Canadian Oncology Drug Review

12.  An expert advisory group that makes recommendations to the
Executive Officer on drug funding and related issues.
 Membership includes:
 Physicians
 Pharmacists
 Health economists
 2 patient members (since June 2007)
 Key considerations for CED drug reviews:
 Clinical efficacy and safety of the drug product relative to
available alternatives
 Cost-effectiveness (i.e. evidence of value for money) of the
drug product relative to alternative treatments
 Patient impact
 Impact on other health care services
Committee to Evaluate Drugs (CED)

13.  Makes final drug funding decisions taking
into consideration:
 CED recommendation
 Advice from other advisory bodies, e.g. Citizens’
Council
 Patient and societal impact, public interest
 Product listing agreements with manufacturers
 Drug program budgets
 Other factors, e.g. government priorities
Executive Officer

14. Objective:
 To put in place a formal framework to systematically
incorporate patient evidence into the drug review and funding
process.
Rationale:
 Patients and caregivers can provide valuable information and
insight about the impact of a disease and new and existing
drug treatments.
 Patients and caregivers can identify areas of most importance
to patients in the particular disease.
 This information can help set the context for the evaluation of
clinical and economic data.
Patient Evidence Submission

15.  Patient evidence submissions are collated by the
Ministry and are provided to all members of the CED.
 Patient evidence submissions to CADTH and pCODR are
also provided to the CED.
 A CED patient member presents a summary of the
patient evidence submissions during drug funding
deliberations.
 Patient input is taken into consideration by the CED in
its recommendation to the Executive Officer.
How patient evidence submissions
are provided to the CED

16.  The CED meets face-to-face on a monthly basis
 The Committee follows a framework for each drug review
 Overview of the disease/condition treated with the product under review
 Presentation of societal values/patient perspective
 Patient members provides a summary of the patient submissions –
included in the report is information on the patient groups, the impact
of the disease/condition, the treatment outcomes that matter most to
patients and information from patients who have used the drug
 Clinical review
 Cost-effectiveness review
 Deliberations on patient perspective, clinical data, and economic data
 Recommendation to the Executive Officer
How does the CED use the patient
submissions along with the other
evidence?

17.  Patient evidence submissions are accepted from registered patient
groups only. Individual patients are encouraged to contact an
organized patient group if they wish to make a submission.
 Each patient group must submit a complete registration form, either
prior to or at the time of submission.
 A drug review schedule is posted on the Ministry’s website outlining
all new drugs undergoing funding review. Patient groups are
encouraged to check the drug schedule regularly and to provide their
patient evidence submission on the Ministry’s template by the posted
deadline.
 The deadline is based on the anticipated CED meeting date. A
minimum of 1 month is provided from time of posting on the drug
review schedule to the submission deadline.
 Balance: providing adequate time for groups to compile
submissions while not delaying drug review and access
http://www.health.gov.on.ca/english/providers/program/drugs/patien
t_evidence/drugreview_schedule.htm
How to make a submission

18.  The patient evidence submission template form and
the “how to guide” are posted on the Ministry’s
website.
http://www.health.gov.on.ca/english/providers/program/drugs/patient_evidence.html
 Submission template:
 Author information
 Conflict of interest declaration
 Impact of the disease/condition
 Outcomes that matter most to patients
 Information from patients who have used this drug
How to make a submission (cont’d)

19.  These suggestions are entirely my own and are not
intended to represent the views or advice of anyone
at the Ministry of Health and Long Term Care, of any
other member of CED, or of the Committee as a
whole.
“Learnings”

20. Information not required:
 Scientific evidence (e.g. clinical trial data) regarding the efficacy and safety of the
drug product and its comparators. The CED already has this information and it will be
considered in the clinical section of the presentation and deliberations.
If submitting to CADTH/pCODR and Ontario CED
 Look at your submission from an Ontario lens.
 Highlight what is different in the Ontario submission as compared to a submission to
a national reimbursement agency
 Is there a higher incidence in this province?
 Are there unmet needs in Ontario that differ from other jurisdictions?
 We expect that there may be some evidence which apply to both submissions.
“Learnings” to make submissions
more useful and impactful

21.  Patient input can be provided by various means and all are considered and
important
 Surveys, , interviews, focus groups, patient stories, etc.
 Best advice is be transparent and describe how you gathered the
information
 How many telephone interviews?
 How many patients/caregivers completed the survey, attended the focus group,
etc.
 Include the date
 State the objective of your information gathering –i.e. general information on
living with this condition, specific information from patients who have taken this
drug, etc.
 Don’t worry if the numbers are small, especially for rare diseases
“Learnings” – Information Gathering

22.  Your input can be more meaningful if you present the impacts
by prioritizing them from most important impact on patients
or most difficult adverse events to tolerate
 For example, the top three symptoms that impact the day to day
lives of these patients
 What is the effect of the symptom on your quality of life?
(does it prevent you from working full-time, can you prepare
meals for your family, etc.)
 Anecdotal statements from patients and/or caregivers can be
very insightful
 If the same theme is repeated in anecdotal statements, choose
one that is representative and state it is pervasive
“Learnings” – Impact of
Disease/Condition

23.  Be as specific as possible in this section
 Most patients want choices but this is a generic statement
 What are some of the practical issues and how do they tie in to the
impact of the condition? (subcutaneous injection vs. intramuscular, etc.)
 Given that the majority of treatments are not breakthrough, what
value does this drug add?
 What will this drug do differently? (Is is the first to address a
particular symptom, long-acting vs. short acting, etc.)
 Is there an unmet need with current therapy?
 What are the most important aspects of the condition that
you wish to see addressed by treatments?
“Learnings” – Treatment Outcomes

24.  Often occurs that you can’t provide information for this
section – just say so
 Can include patients’ input on the expectations for this drug
 If numbers are limited – say so and state number of patients
 Don’t base it on research from pharmaceutical companies or
from doctors – keep it focused on the patient
 Include both positive and negative experiences
 Include side effects of this drug and what patients are willing
to accept, what is unacceptable
 How does the drug compare to current therapy – ease of
administration, side effects, etc.
“Learnings” – Information from
patients who have taken this drug

25. Meaningful patient information
 The most important aspects of the illness that patients would like
the drug therapy to address.
 The shortcomings of existing therapies (that the new drug may or
may not be able to address).
 Other practical aspects of the illness that should be taken into
consideration (e.g. associated costs of living with the disease).
 If you feel that something very important was not specifically
asked, you may include it
Prioritize
 By prioritizing the most important aspects of the illness and
treatment outcomes, patient evidence can help set the context for
weighing the clinical and economic data and understanding the
therapeutic gaps that may exist.
“Learnings” in a nutshell

26. Contact Information:
OPDP Patient Submission
5700 Yonge Street, 3rd Floor
Toronto, Ontario M2M 4K5
Fax: 416-327-8123
Email: PatientSubmission.OPDP@ontario.ca
Patient evidence submission:
http://www.health.gov.on.ca/english/providers/program/drugs/patient_evidence/guidelines.html
Ontario Public Drug Programs:
http://www.health.gov.on.ca/english/providers/program/drugs/drugs_program_mn.html
Questions & Comments