Report on Proton Beam Therapy from the Institute for Clinical and Economic Review (ICER) Shows Most Uses Not Supported by Evidence

 — Report Prepared for Washington State Health Care Authority —

Boston, Mass., July 9, 2014 – The Institute for Clinical and Economic Review has produced a final evidence report on the comparative clinical effectiveness and comparative value of proton beam therapy (PBT) relative to conventional radiation treatments for 16 types of cancer and 3 benign tumors. The report, which highlighted the lack of evidence for most uses of PBT, was prepared for the Washington State Health Technology Assessment program’s Health Technology Clinical Committee and was an integral part of the ultimate coverage decision on the use of PBT for individuals covered in the state’s Medicaid, public employee and workers’ compensation programs. The Clinical Committee arrives at a decision following an open, transparent review of the evidence on safety, efficacy, and cost-effectiveness.

“Our independent and comprehensive review shows that while data exist to support the use of PBT for a few select cancers, there is inadequate evidence to support its use across a broad range of conditions,” said Steven D. Pearson, MD, MSc, President of ICER. “ICER is proud to support public, transparent processes in which the evidence on clinical effectiveness and value are considered together to make the best use of limited health care resources. This report demonstrates the critical role that evidence-based medicine has in supporting high quality, high value outcomes for patients, payers and physicians in Washington and across the nation.”

The American Cancer Society estimates that there are 14.5 million cancer survivors in America, with approximately 1.7 million new diagnoses expected in 2014. Half of all patients diagnosed with cancer will receive some form of radiation therapy as part of their treatment.

Proton beam therapy has attracted much clinical interest due to its physical properties, which have the potential to deliver more accurately targeted radiation as well as to reduce the amount of normal tissue exposed to radiation. Currently, there are 14 PBT centers across America, with 11 more planned or under construction. There is only sparse evidence comparing PBT to other forms of radiation as well as alternative treatments. To date, there has been only one randomized controlled trial comparing PBT to an alternative treatment, and few high-quality observational studies are available.

Despite these limitations, ICER’s review found that PBT was superior to surgical treatment of tumors of the eye, and appears to be a safer alternative to conventional radiation in brain and spinal tumors as well as pediatric cancers, although most of the evidence in the latter two conditions has been based on simulation modeling rather than actual clinical study.

There was insufficient evidence to determine whether PBT offers any improvement over alternative treatments for many other conditions, including bone, breast, gastrointestinal, head and neck cancers, lymphomas, and benign arteriovenous malformations.

For many other conditions, including prostate, liver and lung cancer, PBT provides comparable outcomes but at greatly increased cost. Payments for PBT are typically 2-3 times higher than for other forms of radiation treatment and may approach $50,000 per course of treatment in some situations. The higher costs and limited evidence of effectiveness have prompted many policymakers to carefully examine the circumstances under which PBT should be offered.

About ICER
The Institute for Clinical and Economic Review (ICER) is an independent non-profit health care research organization dedicated to improving the interpretation and application of evidence in the health care system. ICER directs two core programs: The California Technology Assessment Forum (CTAF), and the New England Comparative Effectiveness Public Advisory Council (CEPAC). For more information about ICER, please visit www.icer-review.org.

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