The Belgian Health Care Knowledge (KCE) released a report of HTA for proton beam therapy in adults

In January 2019, the Belgian Health Care Knowledge (KCE) published a report of health technology assessment that was dedicated to the evaluation of proton beam therapy in adults.

This report aims at reviewing the existing clinical data to support the use of proton beam therapy in specific indications.

The final goal of this research is to ensure proton beam therapy is used as objectively and as effectively as possible, saving it for those patients likely to benefit the most.

The report describes evidence concerning efficacy, safety, and cost-effectiveness of the proton beam therapy in adults for the following indications not yet reimbursed in Belgium:

• Low-grade glioma (LGG)
• Primary sinonasal tumors and recurrences of head & neck tumors
• Breast cancer in women
• Pancreatic cancer
• Hepatocellular cancer (HCC)
• Locally recurrent rectal cancer

A systematic review (SR) of the scientific literature was carried out in the main medical databases. In addition, HTA reports were looked for in the HTA database as well as at agencies’ sites. Reference lists of any relevant articles were checked to identify additional relevant studies/reports.

Results:

Overview of selected studies:

• In total, 11 systematic reviews / HTA reports were included. Six studies compared proton beam therapy with photon therapy, while two studies had the wrong comparator but sufficient patients in the proton beam therapy group (and thus were included as a single-arm study). Finally, 22 single-arm studies included at least 50 patients and reported on the relevant outcomes

Effectiveness results (key points):

• The available evidence on the effectiveness of proton treatment for the selected indications is limited to non-randomized comparative studies with methodological limitations and/or small sample sizes. The conclusions below, therefore, have a high degree of uncertainty:
  • There is evidence of a very low level (1 study, 32 patients) that proton treatment is associated with worse survival than photon radiotherapy in patients with primary intramedullary spinal cord gliomas. The data on recurrence is too imprecise to draw a firm conclusion
  • There is evidence of a very low level (1 study, 98 patients) that proton treatment is associated with worse physician-rated cosmetic results at five years than photon radiotherapy in patients with stage I breast cancer. No significant difference was found for patient-rated cosmetic results. The data on local failure rate is too imprecise to draw a firm conclusion
  • There is evidence of very low level (1 study, 25 patients) that proton treatment and hyperfractionated acceleration radiotherapy with concomitant S-1 do not differ significantly in their effect on survival and disease control in patients with locally advanced and unresectable pancreatic cancer, although the estimates are imprecise. The data on local progression is too imprecise to draw a firm conclusion
  • The data on the effect of proton treatment vs. photon radiotherapy on local recurrence rate in patients with recurrent hepatocellular cancer is too imprecise to draw a firm conclusion
• In the absence of clinical studies comparing proton treatment with photon-based radiotherapy, no conclusions can be drawn on the effectiveness of proton treatment for primary sinonasal cancer, recurrent head and neck cancer, and locally recurrent rectal cancer

Safety results:

• The available evidence on the safety of proton treatment for the selected indications is limited to non-randomized comparative studies with methodological limitations and/or small sample size and single-arm studies. The conclusions below, therefore, have a high degree of uncertainty:
  • The data on the effect of proton treatment vs. photon radiotherapy on radiation necrosis and pseudoprogression in patients with primary intramedullary spinal cord gliomas is too imprecise to draw a firm conclusion
  • There is evidence of very low level (1 study, 98 patients) that proton treatment is associated with more dermatologic toxicity (skin color changes, patchy atrophy, telangiectasia) than photon radiotherapy in patients with stage I breast cancer. The data on rib fractures and fat necrosis is too imprecise to draw a firm conclusion
  • The data on the effect of proton treatment vs. hyperfractionated acceleration radiotherapy with concomitant S-1 on acute grade 3 leukopenia, thrombocytopenia and ulcer in patients with locally advanced and unresectable pancreatic cancer is too imprecise to draw a firm conclusion
  • The data on the effect of proton treatment vs. photon radiotherapy on toxicity in patients with recurrent hepatocellular cancer is too scarce to draw a firm conclusion

Conclusions:

To allow a recommendation for or against the reimbursement of proton therapy for these indications, more information is needed in addition to this systematic review. Cost-effectiveness, organizational and ethical issues, which are part of a classical HTA approach, should ideally be put in the balance as well. However, in the absence of reliable data on the effectiveness of a treatment, which is the case for proton treatment for the selection of indications in this report, cost-effectiveness analysis is not adequate. In addition, one may consider it unethical to treat patients with an experimental treatment just based on assumptions of its advantages

See full-text report in English here. You can change to FR and NL in the top left corner.

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