HTA training - Philip Watt, CF Ireland - July 26th 2016

Patient Group Perspective (Ireland)

Philip WattCEO Cystic Fibrosis Ireland IPPOSI Workshop 26 July 2016

Summary of Presentation

1. Brief Case study CF Drug• Focus on CFI’s role

2. Some general conclusions of wider interest3. Some early thoughts on proposed new system

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

ImpactOnce submitted the NCPE

have 90 days (stop-clock

process) to make

recommendation

NCPE appraisal of the company submission

NCPE appraisal sent to company for comment

NCPE appraisal submitted to the HSE

“Fair Prices & Real Value”

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

Impact of KalydecoPrincipal 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

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

Outcomes of Appraisal Process

1. First appraisal Soft ‘No’ - but commitment to negotiate further with the Company

2. Price agreed after further negotiation process

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”.

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 clinicians2. We were concerned to demonstrate our

independence and expertise• Acting to our own agenda and not industry• Bringing added value• Interaction with all stakeholders

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

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’

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

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?

Thank you

• [email protected]