1. Challenges and opportunities in New Zealand’s pharmaceutical scene Kevin Sheehy – Chief Executive, Medicines New Zealand Dalton Kelly – Chief Executive, New Zealand Cancer Society Assoc. Prof. Ken Whyte – University of Auckland Medical School

2. Changes & opportunities in NZ Pharmaceutical Scene: An individual clinician’s view. Ken Whyte Respiratory, Transplant & Sleep Physician

3. Demographic time bomb Christensen K et al, Lancet 2009;374:1196

4. Increasing demands: kidney dialysis projections

5. Pharmac & NZ successes  Controlling pharmaceutical spending  For majority of conditions has delivered adequate therapies & benefits at reduced cost  Developed a “commercial” model that both sides (funder & Pharmaceutical Industry) understand  Clinicians griped but could live with system for most therapies – system struggled with genuinely “new” therapies and high cost treatments.  Pharmac overall has “Brownie points” in the bank with the health system (providers/funders/users)

6. Pharmac & NZ weaknesses?  Treasury small – Health budget finite  Minimal resource for health economic analysis:  Poor costing data for NZ health & disability costs  Total lack of University health economic departments to train & offer analytical resource for Pharmac/Industry/Government/Patient groups  Pharmac very limited resource ( MOH similar)  Failure to debate the ethics of health funding and delivery.

7. Ethical approach – not clear?  Utilitarianism  the greatest good for the greatest number  The worst off have no priority  Maxmin Principle (Rawls – Justice as Fairness)  to the greatest benefit of the least advantaged, open to all under conditions of fair equality of opportunity In health care (+/- social care) – should a life saving or altering therapy, (even if very expensive) take priority over treating a lesser problem in a larger number? Variety of tools tried to measure but crude tools in reality & struggle to assess life altering/saving therapies. Precedent, equity & transparency – if one high cost treatment funded ($/QUALY) then others of same benefit should be funded

8. The Clinician’s dilemma  Advocate for individual patient  In rare or very disabling conditions often no other advocates for the patient group  Awareness of resources and societal demands  Pharmac’s response:  Exceptional circumstances  Now named patient approach (NPPA) from 1/3/12  In past no clear societal decision in health funding re Utilitarianism versus more “graded” approach

9. Looming threats  Break down of the Pharmaceutical model:  In short term many therapies will come off patent so $$$ looks good  Large pharmaceutical companies portfolios shrinking  Small niche companies (often funded by venture $$) - targeted & often “individual” therapies (small volume & great cost)  Shrinking room for Pharmac to trade over a portfolio to allow new therapies affordable entry in NZ  Dramatically life altering & saving replacement therapies  This leads to an increasingly complex and probably insoluble dilemma around allocation of resources.  Orphan diseases*: rare, yet many (estimated 5000-7000) & both randomised trials and cost benefit analysis not feasible as such small numbers – yet up to 2+% of population suffer when all added together! * life-threatening or chronically debilitating diseases which are of such low prevalence that special combined efforts are needed to address them

10. Replacement therapies?  Pompe’s disease (progressive muscle weakness leading to disability & death):  algluosidase alfa drug, Lumizyme© - “adult”  $250,000 per year for initial improvement & slowing of progression  Societal costs of care altered by therapy?  Cystic Fibrosis – disabling, fatal 25-40yrs with very poor quality of life & huge cost:  For some sufferers – prospect of replacement therapy from infancy with dramatically altering life quality & expectancy:  Therapy for life - ?1-75 yrs  Cost versus benefit – normal versus disabled life.

11. Go forth and multiply?

12. Medical Devices?  Triumvirate of material scientists/smart engineers/health scientists  near limitless “new” developments  Rapid redundancy – limited time for trials  Traditional RCT model difficult:  Placebo difficult  Time frames to assess QALY often long  Cost benefit analysis for in-patient devices (theatre, wards etc) differ for individual services & different hospitals. “Publicly” funded utility trials rather than manufacturer funded?

13. Pharmac & NZ future?  Pharmac model needs to be pro-active & change before “melt-down” & loses credibility?  Pharmac taking on Medical devices maybe a step too far?  Pharmac has always been under-resourced (proud & lean is not always productive!)  NZ Society has to debate the ethics & make decisions – Pharmac can only delay