Hitting the Target: Developing High-quality Evidence for Proton Beam Therapy Through Randomised Controlled Trials.

Area of Science:

• Radiation Oncology
• Clinical Trials
• Medical Physics

Background:

• The UK's National Health Service strategy for proton beam therapy (PBT) presents an opportunity for high-quality evidence generation.
• Randomised controlled trials (RCTs) are crucial for evaluating new cancer treatments like PBT.

Purpose of the Study:

• To summarize three ongoing UK multicentre phase III PBT RCTs (TORPEdO, PARABLE, APPROACH).
• To detail key design characteristics and outcome assessments for future PBT trial development.
• To address uncertainties regarding the clinical benefits of PBT, focusing on toxicity reduction and patient-reported outcomes.

Main Methods:

• Comparison of PBT with photon radiotherapy in three distinct cancer types: oropharyngeal squamous cell carcinoma, breast cancer, and oligodendroglioma.
• Multidisciplinary team design involving clinicians, trialists, scientists, patient advocates, and international collaborators.
• Inclusion of translational research components to investigate toxicity mechanisms.

Main Results:

• The three RCTs (TORPEdO, PARABLE, APPROACH) are actively recruiting and comparing PBT against conventional radiotherapy.
• Consistent focus across studies on reducing long-term radiotherapy-related toxicities.
• Evaluation of patient-reported outcomes and health-related quality of life is central to assessing PBT's benefits.

Conclusions:

• The UK is establishing an internationally significant PBT research portfolio through these RCTs.
• Combining RCT data with a national PBT outcomes registry will create a valuable resource for future research.
• This comprehensive approach will facilitate the design and delivery of subsequent PBT trials.