PP75 Using Real World Evidence To Support The Reimbursement Of Proton Therapy Across A Broad Range Of Rare Cancers

Abstract

Introduction

The Australian Bragg Centre for Proton Therapy and Research in Adelaide will be Australia’s first center with the capacity to deliver proton beam therapy (PBT). PBT uses energy from protons to target cancer cells while minimizing damage to surrounding healthy tissue, including vital organs. Compared to X-ray (photon) radiation therapy (PRT), PBT reduces the risk of serious and long-term complications.

To improve access to PBT, the South Australian Health and Medical Research Institute (SAHMRI) submitted an application to the Medical Services Advisory Committee (MSAC) including a cost utility analysis (CUA) comparing PBT with PRT to treat specific pediatric/adolescent and young adult (AYA) and rare adult tumors.

Methods

A systematic review identified 28 comparative, mostly real-world studies to support the conclusion that PBT has superior safety and non-inferior efficacy to PRT in the requested indications. The key challenge for the CUA was to quantify the cost and quality of life implications of the superior safety profile across a wide range of indications with a limited comparative evidence base. A simple lifetime decision analytic model was developed which modeled the rates, costs and utilities associated with relevant toxicities. The complications of radiotherapy are often chronic and included secondary malignancies, visual impairments, endocrine dysfunction, dysphagia, hearing loss and intellectual disability. Some of these toxicities are only applicable to patients with cranial cancers. Therefore, the event rates applied in the evaluation were adjusted to account for the proportion of patients within each population estimated to have extracranial cancers.

Results

When results in the adult and pediatric/AYA populations were weighted across the expected utilization of PBT (34% adults, 66% pediatric/AYA) in each population, PBT was dominant relative to PRT.

Conclusions

In November 2020, MSAC recommended funding PBT in specific populations at high risk of long-term side effects from PRT. To address uncertainties around the evidence base, MSAC further requested the following:

• • All patients receive comparative photon/proton plans to determine eligibility

• • A national registry is established for patients treated with PBT.