May 19: Leave No One Behind Panelists Brad Alyward, Head Market Access & Health Policy, Indivior Catherine Boivin, Patient Advocate, CORD Shona Kinley,Director, Federal Policy & Government Affairs, Novartis Bennett Lee, Head, Value & Access, Sanofi Joan Paulin, Patient Advocate, PHA Canada Trevor Richter, Director of Access and Reimbursement, Gilead

1. SPRING 2022
WEBINAR
SERIES
Planning SMART

2. SPRING 2022
WEBINAR
SERIES
MAY 3
12 - 1 pm
Rare Disease
Patient Registries:
Key to Drug
Development and
Access
MAY 5
12 - 2 pm
Rare Disease Centres
of Excellence:
Linchpin to patients,
community care, and
collaboration
MAY 19
12 - 1 pm
Leave No One
Behind
APRIL 19
12 - 1 pm
Application of
RWE in Drug
Access Decision
Making
APRIL 26
12 - 2 pm
Lessons for a SMART
Rare Drug System

3. RARE DRUG CONFERENCE
JUNE 8 - 9, 2022
Ottawa Marriott Hotel
Building Canada’s SMART Rare
Disease and Rare Drug System

4. CONFERENCE
OVERVIEW
Over the past 20 months, the Canadian rare disease community, under the leadership of the
Canadian Organization for Rare Disorders, has come together in 36 webinars and forums to create a
collaborative vision of an optimal national Rare Disease Program supported by a comprehensive and
integrated Rare Disease healthcare system. Now, we are ready to propose an operational framework
to realize that vision. We are NOT proposing minor tweaks to address gaps in access or improve
timeliness. We must NOT start with a formulary of “common” or “priority “rare disease therapies, even
if these are fully funded for all patients.
CORD is calling on all stakeholders at this conference to arrive at a consensus on a complete re-
imagining of a system from the bottom up and top down that is “fit for purpose” of assuring all
Canadians have access to the best therapies as soon as they are available anywhere in the world.
We need to start with a patient-centred rare disease system that assures every person, in as short a
time as possible, gets an accurate diagnosis, sees a specialist, forms an individualized care,
treatment, and support plan, is enrolled in a patient registry, is referred to a patient support group,
gets timely monitored access to the best treatment and, most important, has the knowledge and
right to participate as a full partner in all health-related decisions. This should be readily available for
all Canadians, regardless of where they live, their insurance coverage or their ability to pay.
Building Canada’s SMART Rare
Disease and Rare Drug System
June 8 - 9, 2022
Ottawa Marriott Hotel (Virtual option available)

5. CONFERENCE
OVERVIEW
Why is Rare Disease a Public Health Issue?
There are more Canadians with a rare disease than with all cancers, cardiovascular disease, diagnosed diabetes. Altogether, 3 million
Canadians, about 1 in 12, have a rare disease. Moreover, while 80% of rare diseases are genetic, 50% have no known family history. So
everyone is potentially at risk. Two-thirds of those affected are children, 30% of whom will not live to their fifth birthday. Sadly, in
Canada, a child dies of a rare disease every 18 minutes.
In terms of economic impact, rare disease costs the Canadian economy about $111 billion per year in direct medical costs,
nonmedical cost, and productivity costs.
Why is NOW the time for Canada’s SMART Rare Drug Strategy?
Rare disease drugs are saving lives, preventing and reducing disability, allowing patients and families to live more “normal”
productive lives, and, for some conditions, providing a long-lasting treatment or “cure.” Rare disease research is leveraging genomic
breakthroughs, advanced medical technologies, application of data science and artificial intelligence to big data, patient registries,
and real-world data, multiple disease cell-and-gene therapy platforms, and remote patient engagement. So rare disease therapies
are at the forefront of innovative research, providing solutions with impacts beyond rare diseases, including viruses, cancers, and
cardiovascular diseases.
Thanks to the US and EU Orphan Drug Acts, about 600 new therapies have been approved in the past 40 years but these cover only
about 5% of up to 7,000 rare diseases. To date, Canada has contributed to only a small handful of rare disease drug discoveries.
However, that scenario can and should change. Canada’s burgeoning life sciences strategy has the scientific, clinical, and patient
capacity to contribute significantly to discovery, manufacture, and management of rare disease diagnosis and treatment.
Most would agree that our drug assessment procedures were never designed to accommodate innovative therapies for small
patient populations but we have continued to put them through the process and have mostly “cobbled” “one-off” reimbursement
agreements with ostensibly minimal impact on the overall drug budgets. The solutions were rarely evidence-based, equitable, or
sustainable. The question is not whether we have reached the breaking point with the “old” access pathways but “how can we do
better?”
Building Canada’s SMART Rare
Disease and Rare Drug System

6. MAY 19
12 - 1 PM
Case Example - PMD
• PMD is a genetic progressive neuromuscular disorder that affects the
muscles but also heart and other organs. Infants with the most severe
form will display cardiac symptoms as early as six weeks while those with
less severe forms will develop various symptoms at ages up to early
adulthood.
• TR-PMD is a therapy that can slow or stop disease progression but not
demonstrated to reverse or cure the disease. The regulator has approved
TR-PMD for patients of all ages but the HTA agency has recommended
reimbursement only for children who are diagnosed and started prior to
six years of age, with the rationale that children under 6 years old (1) were
in the initial clinical trials; (2) have the most severe form of the disease; (3)
will experience the greatest health benefits; (4) require relatively smaller
amounts of the drug which is a weight-based treatment.
• Drug access for older patients may be requested and available on a case-
by-case basis; approval varies widely by jurisdiction.
Leave No One Behind

7. MAY 19
12 - 1 PM
Case Example - DG
• DG is a genetic-based progressive respiratory condition affecting quality
of life and leading to early death. Until a few years ago, it was treated
primarily by antibiotics and symptom management.
• The first approved therapy was targeted for a specific genetic mutation
affecting only 5% of the patient population. New therapies and
combination therapies are effective for up to 90% of genotypes, with
individual results varied as to size and duration of benefits. Moreover,
there is limited long-term evidence on effectiveness at treating the
underlying cause of disease as well as impact on reducing symptoms,
slowing disease progression, and, in some cases, reversing damage.
• However, there is limited evidence as to which patients will respond, how
much benefit individual patients will experience, and for how long the
therapies will continue to work. While available through many private
drug plans, the co-pay can make the therapy unaffordable for some
individuals and families.
Leave No One Behind

8. MAY 19
12 - 1 PM
Case Example - SW
• SW is a drug that was approved about 12 years ago as the first
treatment for an ultra- rare blood disorder.
• Over time, there have been additional conditions approved, one
which affects a relatively large (non-rare) population with
several alternative therapies.
• The drug price was negotiated for the original rare condition.
The drug plans are requesting a re-negotiation based on prices
of alternative therapies for the large disease group.
Leave No One Behind

9. MAY 19
12 - 1 PM
Case Example - KA
• KA is a disease that affects about 300 individuals in Canada; the only
therapy is an old drug used for several conditions; it no longer has a
patent but there is only one manufacturer. The therapy is effective in
managing the symptoms but does not treat the underlying condition.
The biggest challenge is that it is difficult to administer requiring slow
infusion with a pump overnight.
• A new formulation is an oral therapy, taken once a day. In clinical trials,
the oral therapy was demonstrated “non-inferior” to the original therapy
and approved by the regulator.
• Most of the patients would prefer the oral therapy but the HTA agency
did not recommend reimbursement because (1) there is no greater
efficacy, only greater convenience; (2) the calculated “incremental cost
effectiveness ration (ICER) was considerably higher than the benchmark
“willingness to pay” (WTP) threshold of public drug plans for cost per
Quality Adjusted Life Year (>$200,000/QALT) and a budget impact over $1
million/per year for fewer than 1,000 patients.
Leave No One Behind

10. MAY 19
12 - 1 PM
Stories from the front lines: : impact of denied or delayed or never launched drug access
• Good: Rescuing Individuals falling between the cracks
• Better: Solutions to bridging systemic gaps
• Best: Designing fair rare care … from R&D to monitored access
Managed drug access: the new normal and not the exception
• Managed access criteria = individualized drug utilization = optimized humanized monitored
healthcare
• Managed access programs = value-based access = optimal societal cost utility/ROI
No Common QALY
• If the single common QALY is not relevant to rare disease therapies, what is the alternative, if
anything?
• QALYs are only as good as the data on which they are calculated. If clinical trial rare drug data are
uncertain, what’s the value of the calculated QALY?
• If the validity of the QALY relies on the validity of the quality-of-life scores for various disease states
and the QoL scales are not relevant for most rare diseases, what is the validity of the QALY? Does it
contribute to the valuation of the therapy?
• Qualitative patient reports of health-related experiences and outcomes, including personal vignettes,
are rich in data but difficult to transform into quantitative evidence that can be analyzed. Do these
contribute to valuation of the therapy?
One Rare Pathway … Multiple Funding Sources?
Should all Canadians with the same rare condition have the same access to care, treatment, and
support?
Leave No One Behind

11. PANELISTS/
DISCUSSION
Panelists
• Brad Alyward, Head Market Access & Health Policy, Indivior
• Catherine Boivin, Patient Advocate, CORD
• Shona Kinley, Director, Federal Policy & Government Affairs, Novartis
• Bennett Lee, Head, Value & Access, Sanofi
• Joan Paulin, Patient Advocate, PHA Canada
• Trevor Richter, Director of Access and Reimbursement, Gilead
Panel Discussion
• What does “Leave No One Behind” mean in these scenarios? Who should get access and
under what circumstances?
• Which principles, values and fairness rules should guide which drugs are available to
whom under what conditions?
• Can QALYs be made to work for rare disease?
• Should there be different Willingness to Pay (WTP) thresholds for rare?
• Should budget impact (BI) be a key criteria for making drugs available?
• Can multiple drug plans participate in a single drug allocation process?
• What does fair pricing look like?
Moderators
• Bill Dempster, 3Sixty Public Affairs
• Durhane Wong-Rieger, CORD

12. RARE DRUG CONFERENCE
JUNE 8 - 9, 2022
Ottawa Marriott Hotel
Building Canada’s SMART Rare
Disease and Rare Drug System

13. Driving Innovation Forward
10 Years since CAR-T was introduced
CORD webinar /May 19, 2022
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