Value assessment of oncology drugs using a weighted criterion‐based approach

Abstract

Background Globally, the rising cost of anticancer therapy has motivated efforts to quantify the overall value of new cancer treatments. Multicriteria decision analysis offers a novel approach to incorporate multiple criteria and perspectives into value assessment. Methods The authors recruited a diverse, multistakeholder group who identified and weighted key criteria to establish the drug assessment framework (DAF). Construct validity assessed the degree to which DAF scores were associated with past pan‐Canadian Oncology Drug Review (pCODR) funding recommendations and European Society for Medical Oncology Magnitude of Clinical Benefit Scale (ESMO‐MCBS; version 1.1) scores. Results The final DAF included 10 criteria: overall survival, progression‐free survival, response rate, quality of life, toxicity, unmet need, equity, feasibility, disease severity, and caregiver well‐being. The first 5 clinical benefit criteria represent approximately 64% of the total weight. DAF scores ranged from 0 to 300, reflecting both the expected impact of the drug and the quality of supporting evidence. When the DAF was applied to the last 60 drugs (with reviewers blinded) reviewed by pCODR (2015‐2018), those drugs with positive pCODR funding recommendations were found to have higher DAF scores compared with drugs not recommended (103 vs 63; Student t test P = .0007). DAF clinical benefit criteria mildly correlated with ESMO‐MCBS scores (correlation coefficient, 0.33; 95% CI, 0.009‐0.59). Sensitivity analyses that varied the criteria scores did not change the results. Conclusions Using a structured and explicit approach, a criterion‐based valuation framework was designed to provide a transparent and consistent method with which to value and prioritize cancer drugs to facilitate the delivery of affordable cancer care.

Potential approaches for the pricing of cancer medicines across Europe to enhance the sustainability of healthcare systems and the implications

“…While there were some differences in value preferences between respondents (assessors and experts) in the given countries, drug rankings in terms of the relative value of the different medicines for prostate cancer 2020) used a multi-stakeholder approach incorporating 2 patients, 2 public members, 2 patient advocacy group leaders, 2 pharmacists, 1 industry representative, 6 oncologists, 1 ethicist, 3 health economists, 3 members of an appraisal committee (pCODR), 2 cancer agency members, and a Ministry of Health government representative. They identified the criteria through published literature, and let the stakeholders assign weights equaling 100 [104]. The highest weights assigned included quality of life (weight of 19), overall survival (weight of 15), and unmet clinical need (weight of 15), with the lowest weights being for disease severity (weight of 5) and caregiver well-being (weight of 4) [104].…”

“…They identified the criteria through published literature, and let the stakeholders assign weights equaling 100 [104]. The highest weights assigned included quality of life (weight of 19), overall survival (weight of 15), and unmet clinical need (weight of 15), with the lowest weights being for disease severity (weight of 5) and caregiver well-being (weight of 4) [104].…”

An Arm and a Leg: The Rising Cost of Cancer Drugs and Impact on Access

“…[51][52][53][54] A Canadian drug assessment framework, developed to promote transparency and consistency in oncology drugfunding decisions, also includes valuation of unmet need, equity, and disease severity as part of structured decisionmaking. 55 Despite the establishment of value frameworks, multiple studies confirm that cancer drug prices do not correlate with value or clinical benefit. 6,44,56,57 Drugs that provide less value to patients are often higher priced than those that provide greater value.…”

Understanding Statistical Significance and Avoiding Common Pitfalls

“…It offers an opportunity to integrate information about values and goals. For example, a patient may be more prepared to accept the possibility of a false-positive result for the benefit of a particular drug if the side-effects are minimal, or if there are no alternative treatment options [12].…”