Vorinostat (Zolinza) is now available on the PBS for patients with relapsed or refractory cutaneous T-cell lymphoma, following a successful submission by patient advocacy group Rare Cancers Australia.

This is believed to be the first time a drug has been sponsored through the PBAC approval process by a community organisation rather than a pharmaceutical company.

Vorinostat was registered by the TGA in 2009 but an application to PBAC by MSD Australia and New Zealand was rejected in 2011.

Richard Vines, chairman of Rare Cancers Australia, told the limbic drug approvals for rare diseases were hampered by a lack of evidence related to the small numbers of patients.

“We need a more flexible system if we’re going to get drugs out to people with rare cancers. We are conscious of the fact that the current system provides taxpayer protection aimed at value for money, but it’s no good if it denies patients treatment just because they are so few in number.”

He said their recent success was ultimately due to all parties ‘working together in good faith’.

“We approached MSD as we needed their cooperation. We also approached the department. They were the ones who had to sit down and negotiate a price.”

Peter MacCallum Cancer Centre provided additional ‘real world evidence’ from their cutaneous lymphoma database, he said.

Associate Professor Chris McCormack, a specialist in dermato-oncology at Peter MacCallum, told the limbic improved access to vorinostat would improve the quality of life and prolong life for patients with extensive disease.

“It’s a very symptomatic disease which is itchy and painful and causes patients huge cosmetic concern and contributes to social isolation. A significant proportion of people will have a prolonged response to this therapy.”

Associate Professor McCormack said while the PBS usually found it difficult to cope with rare diseases, the latest round of wins also included adalimumab for the treatment of the inflammatory skin disease hidradentis suppurativa.