The first meeting of the PTAC Rare Disorders Subcommittee was held on 20 June 2014.  The minutes of the meeting are now available on thew PHARMAC website. The Subcommittee recommended eight prerequisites for the draft RFP which were reviewed by PTAC on 4 July 2014.

Disorder related: 

1. There is a rare but clinically defined long-term disorder that is identifiable with reasonable diagnostic precision. 

2. Epidemiological and other studies provide evidence acceptable to PHARMAC that the disorder causes a significant reduction in either absolute or relative age-specific life expectancy or quality of life, for those suffering from the disorder. 

Treatment related: 

3. The medicine is regarded as a proven therapeutic modality for an identifiable patient population i.e. the medicine has been approved by Medsafe or an international regulatory authority for the identified indication. 

4. There is evidence acceptable to PHARMAC that the medicine is likely to be clinically effective for the identified patient population4. 

5. The patient’s absolute or relative age-specific life expectancy or quality of life could be substantially improved as a direct consequence of the treatment6. 

Alternatives related: 

6. The medicine is not registered for the treatment of another, non-rare disorder, or if it is, the cumulative prevalence still falls within the definition of rare7. 

7. There is no suitable comparable alternative treatment on the Pharmaceutical Schedule. 

8. There is no suitable funded alternative non-drug therapeutic modality for the rare disorder.

For more details, go to: http://www.pharmac.health.nz/assets/ptac-rare-disorders-subcommittee-minutes-2014-06.pdf