The patient group perspective in Ireland

1. Patient Group Perspective
(Ireland)
Philip Watt
CEO Cystic Fibrosis Ireland
IPPOSI Workshop
26 July 2016

2. Summary of Presentation
1. Brief Case study CF Drug
• Focus on CFI’s role
2. Some general conclusions of wider interest
3. Some early thoughts on proposed new system

3. Existing Pharmacoeconomic
Assessment (summary)
For high cost products and /or those with significant budget
impact – Following rapid review
Product Recommended
for Full HTA
Formal Notification to
Company of Rapid
Review by CPU
Pre-submission
consultation between
NCPE & Company Reps
Formal submission of evidence
on cost-effectiveness & budget
Impact
NCPE appraisal of the
company submission
NCPE appraisal sent to
company for comment
NCPE appraisal
submitted to the HSE
“Fair Prices & Real Value”

4. Case Study: Kalydeco (Ivacaftor)
Ground breaking CF Drug
Results of Phase 3 Trials:
Increase in lung function by
10.6%
Increase in body weight by
2.8kg
54% decrease in risk of first
pulmonary exacerbation

5. Impact of Kalydeco
Principal Investigator, Professor Stuart Elborn
• ‘While there has been great progress in cystic fibrosis treatment during the last few
decades, we are still only treating the symptoms and complications of the
disease…..KALYDECO is a fundamentally different approach to the way we treat cystic
fibrosis because it targets the underlying cause of the disease’
• More Impact in Ireland than anywhere else in the world
• Impacts on those with G551D Gene Alteration
• 3% Worldwide 11% CF population in Ireland
• Up to one quarter of CF population attending Cork Hospital
• Forbes ‘Drug of the Year’ 2012

6. Kalydeco – not ‘cost-effective’
At a submitted cost of €234,000 per patient per year

7. Outcomes of Appraisal Process
1. First appraisal Soft ‘No’ - but
commitment to negotiate further with the
Company
2. Price agreed after further negotiation
process

8. Retrospective Feedback from
PatientsThe strength of the patient voice cannot be
underestimated!
“Physically- lung function has improved, weight has increased, Oxygen requirements reduced significantly,
Physically stronger, came off the lung transplant list Emotionally- a lot more hopeful for the future, huge positive
effect on my family, have become non-CF focused, happier Personally- Plan activities e.g. holidays, family
activities; can spontaneously do things, have commenced a degree course”
“CF no longer impacts significantly on my day-to-day life - I don't
think about it as often & feel as though I am leading a pretty normal
life now! Looking forward to my future personally and professionally
and feel much more positive about life in general. Stress levels have
definitely decreased & my entire family & friends are also much
happier and less stressed about CF”.
“Feel much more energetic
which has changed my life
completely. I now have a
diploma in social. I can do 2-3
12 hour shifts a week. I Also
support myself by living
independently which is
something I never expected to
be able to do”.
“General standard of life had improved. I don't
have to take my health into consideration over
every decision I make. I Lead a normal life and
go about my business as if I don't have cf. I am
happier over all”.

9. CFI’s Advocacy as a patient
group
Our approach to advocate for Kalydeco was governed by
following:
1. We knew this was an excellent drug
• Clinical Trials Data
• Testimonies from trial patients
• Independent discussion with clinicians
2. We were concerned to demonstrate our independence
and expertise
• Acting to our own agenda and not industry
• Bringing added value
• Interaction with all stakeholders

10. Key learning from this
experience
“Fair Prices & Real Value”
• The Health Technology Assessment is a relatively transparent process in Ireland
• However its is unlikely that any high tech drug for rare diseases would be approved at first
instance under the present system
• The NCPE respond very positively to communication from the CF community during these
assessments (“Clinicians involved in the care of patients with CF played a very constructive
role at all times during the Ivacaftor HTA process” Michael Barry)
• Pharmaceutical companies need to set fair prices for therapies
• HTA agencies need to capture the patient perspective
• Specific tools and processes need to be developed
• Challenges for patient groups
• Understand appraisal process
• Engage with NCPE using evidence and unique insights
• Advocacy should be used to challenge industry as well as state bodies as appropriate

11. Health economics & its’ limitations
for rare disease assessments
• Various limitations with the QALY – limited consideration for wider, long-
term savings to be made (joining the workforce, prevention of other
treatments being needed, etc.).
• Presumption that health economics can accurately value the quality of a
person’s life
• Lack of understanding of implications of living with a particular illness –
such as QoL, levels of suffering, etc.
• National Rare Disease Plan: Recommendation 30
• The utilitarian view – “the greatest good for the greatest number”
• Better to fund a million people who need aspirin than 5,000 who
might have a rare disease
• Rarer diseases will always lose out with this mind-set
• Danger of ‘rationing approach’

12. Proposed New system part of
overall reform
New four-year agreement with Irish Pharmaceutical Healthcare Association (IPHA) will
reduce the price the HSE pays for medicines from the 1st of August
Improved pricing mechanism will keep prices of medicines in Ireland on a downward
trajectory and will save an estimated €600 million in total over four years plus additional
€150m from non-IPHA companies
Savings and new assessment process to support investment by the HSE in new and
innovative medicines for Irish patients including cabinet decisions on expensive therapies

13. Initial Response
Patient group perspective
Positive
• Welcome proposed savings of €750m
• Commitment to use savings for new and innovative drugs
by Minister Harris
Challenges
• Will all savings promised be reinvested in new therapies?
• No specific reference to inclusion of patient groups in
assessment process yet building on progress to date
• Proof of pudding in the eating – how many new hi tech
drugs will be approved over next 4 years?

14. Thank you
• pwatt@cfireland.ie