Develop a pre-treatment strategy of risk assessment and benefits

Introduction: Rectal cancer is one of the most commonly diagnosed cancer in the world. In the UK, every year more than 16,000 new cases of rectal cancer are diagnosed. A third of diagnosed patients are deemed as ‘high risk’ with a higher probability of recurrence. The standard practice of treatment for a high-risk rectal cancer patient is Neoadjuvant chemoradiotherapy (nCRT) followed by resection surgery. This treatment pathway is associated with perioperative mortality, postoperative morbidity as well as long-term morbidity (bowel dysfunction, sexual dysfunction), the possibility of a permanent stoma and lower quality of life in general.

In the past decade, observational studies have found that in up to 30% of patients the tumour completely disappears i.e. patients have a clinical Complete Response (cCR) after nCRT. These patients with nCRT can avoid surgery and opt for active monitoring i.e. watch and wait strategy. This new cancer treatment strategy is known as organ preservation.

Similarly, studies (such as OnCoRe) have shown that this novel strategy; watch and wait is safe and does not compromise oncological and long-term survival outcomes of the patients. The possibility of avoiding permanent stoma and various complications related to it is appealing to rectal cancer patients, thus increasing popularity of watch and wait strategy.

The decision that a patient has to take about which treatment pathway to take from available alternatives in rectal cancer is complex and there is little help available to aid them in decision making. Specifically, no work to-date has been done to investigate what outcomes are valued most by the patients and the relative value attached to different types of outcomes when undertaking either watch and wait or surgery. With value based healthcare gaining momentum across Europe, it has been increasingly important to use the concept of value based outcomes for the commissioning of treatments especially in case of adoption of new treatment such as watch and wait. Hence, this study will help to understand the relative value attached to outcomes for watch and wait strategy for patients with cCR after nCRT.

Methods: Over the three years (2021-2023), research work will be carried out in three stages. In first stage, a systematic review will be performed to identify core outcomes associated with watch and wait strategy and surgery after nCRT. After identifying core outcomes, a set of value weights will be developed by using Multi-Criteria Decision Analysis (MCDA) technique in the second stage.

MCDA is a tool to aid decision-making in the context of multiple, potentially conflicting objectives by taking into account multiple criteria in a structured and explicit manner. This technique has been widely used to make decision in various sectors however its use in healthcare is still a new concept. We will identify the most suitable MCDA method and use it to generate value weights for outcomes identified from stage one from a panel of patients and clinician experts. In the third stage, a consensus method such as nominal group technique will be used to discuss and understand how value weights generated in the second stage could be implemented in practice to inform service commissioning in rectal cancer in the context of NHS.

Dissemination: The findings of the research work will be disseminated through scientific publications, presenting the research in conferences and scientific communities. Additionally, the findings will be communicated through various social media platforms, newspaper articles, community engagement programmes for wider reach.

Impact: This evidence-informed value scale will inform whether or not to adopt the strategy of watch and wait in rectal cancer into standard care (e.g. through NICE or ESMO guidelines).

Funding:This project is a part of EU funded CAST programme (Call: H2020-MSCA-ITN-2019). The goal of CAST (Active Monitoring of Cancer as An Alternative To Surgery) is to implement novel approaches in deciding when or whether cancer surgery is needed.

Further details about the programme are available on the CAST website 

Considering recommendations from OnCoRe regarding the absence of evidence around patient preferences and value based outcomes associated with watch and wait strategy versus surgery, we are working on two projects, PrefCoRe and ValCoRe, to provide evidence to aid in shared decision making in rectal cancer treatment. The following table highlights the differences between the two studies:

	PrefCoRe Project	This Project
Title	Quantifying and implementing patient preferences for the treatment of high‐risk rectal cancer, including the new strategy of organ preservation.	Value of benefit from a new cancer treatment: clinical complete response and avoidance of major surgery in rectal cancer.
Aim	To understand patient preferences for watch-and-wait vs surgery following a clinical complete response to chemoradiotherapy for locally advanced rectal cancer.	To derive a set of valuation weights for outcomes to inform value based commissioning in the clinical setting of nCRT for high-risk rectal cancer followed by radical surgery, with up a proportion managed by W&W without surgery versus those managed by radical surgery only.
Objectives	– To quantify the trade-offs that individuals are willing to make between watch-and-wait and major resection surgery for high-risk rectal cancer. – To develop an online patient decision aid based on these trade- offs. – To conduct preliminary tests of the patient decision aid to understand its position and acceptability in the current care pathway.	– To identify core outcomes associated with watch and wait strategy and surgery after nCRT. – To derive valuation weights for identified core outcomes. – To understand how value based outcome framework can be implemented to inform service commissioning in rectal cancer.
Methodology	Preferences will be elicited through a Discrete Choice Experiment (DCE) technique.	Value weights for outcomes will be elicited through a MCDA (Multi Criteria Decision Analysis) technique.
Outputs	– Quantifying trade- offs that individual make between watch and wait and major resection. – Develop patient decision aid	– Derive set of valuation weights for relevant outcomes for watch and wait strategy and surgery.
Expected Impact	Implementation of patient decision aid for decision making in rectal cancer treatment.	This evidence-informed value scale will provide evidence of whether or not to adopt the strategy of watch and wait in rectal cancer into standard care. (for eg: through NICE or ESMO guidelines)