Celgene Patient-Centered Principles on Value Assessments

Celgene’s Patient-Centered Principles on Value Assessments are rooted in the company’s long-held belief that value should be assessed holistically and over the long-term, accounting for the following four value pillars: value to patients, value to the health system, value to society and the economy, and value to future innovation. The ultimate goal of Celgene’s Patient-Centered Principles on Value Assessments is to optimize access to therapies for all patients who stand to benefit.

Patient-Centered Principles on Value Assessments

1. Value assessments should be patient-centered

A. Measure patient-relevant outcomes, accounting for varying treatment responses, preferences, needs and values

  • The perspectives of patients and healthcare professionals should be appropriately accounted for in value assessments
  • Value assessments take a population-level approach, but should still recognize that what constitutes a meaningful outcome varies with patients’ characteristics, preferences, needs and values

B. Allow for consistent, timely and active involvement of patients and other relevant stakeholders throughout the process

  • Patients, caregivers, disease experts, and other relevant stakeholders should be involved in the definition and validation of the outcomes evaluated by value assessments. The process of stakeholder involvement should be carried out in a timely and consistent manner to ensure the appropriate representation of stakeholder experiences and needs
  • Value assessment authorities should have a formal and transparent process for engaging with a range of stakeholders, promoting open dialogue and ensuring stakeholder perspectives are accounted for in final decisions

2. Value assessments should be multi-dimensional

A. Consist of a definition of value that considers value to patients, the health system, society and the economy, and future innovation

  • Value is multi-dimensional and should account for benefits that accrue to:
    ? Patients: clinical benefit and improvement in patient outcomes and quality of life
    ? Health system: impact on health system resources and budgets
    ? Society and economy: patient productivity, family/caregiver quality of life and economic burden
    ? Future innovation: the long-term viability of the R&D ecosystem

B. Utilize a broad range of relevant and reliable evidence to capture the full spectrum of value

  • In addition to randomized controlled trials (RCTs), other rigorous types of evidence, data sources and methodologies are critical to a complete understanding of the ways in which a treatment adds value
  • Depending upon the timing of a value assessment in a product’s lifecycle, different types of evidence and data sources may be appropriate, such as clinical studies, patient-reported outcomes, real-world evidence, and health economic data
  • Methodologies such as systematic literature reviews, meta-analyses, indirect treatment comparisons, and comparative effectiveness may provide additional valuable information

C. Appropriately account for disease- and product-specific factors in the appraisal of the evidence

  • Value assessments should account for disease-specific factors such as the burden of the disease on patients and society, including its physical, emotional, social and psychological dimensions
  • Value assessments should use a broad definition of unmet need that accounts for available treatment options, treatment practices, outcomes associated with alternative interventions, resource use and access issues
  • Value assessments should consider product-specific factors in order to better recognize the complexity of innovative specialty medicines and the inherent challenges in evidence development for rare diseases

3. Value assessments should yield timely, high-quality outputs through flexible and transparent processes

A. Deliver timely decisions that facilitate patient access to new treatments

  • Value assessments should have well-defined timelines and facilitate rapid patient access to new treatments
  • Where applicable, value assessments should align closely with market authorization timing
  • Where applicable, value assessments should facilitate funding decisions for patients and be implemented quickly by the authorities
  • The development of additional evidence should not delay patient access

B. Ensure use of flexible processes that yield high-quality outputs

  • Value assessment processes should be flexible to account for the complexity of evidence development and a range of methodologies and data souces, including clinical studies, patient-reported outcomes, real-world evidence, and health economic data
  • Where applicable, value measurement should be developed based on multi-stakeholder input with the ability to evolve over time
  • Value assessment institutions should ensure they have the resources and capabilities needed to evaluate a broad range of evidence and analyses to ensure high quality outputs

C. Guarantee transparency in assessment processes and outputs

  • The value assessment process and methodology should be explicit and clearly set out in guidelines
  • The extent to which value assessment outputs have a role in treatment, access decisions or pricing negotiations should be transparent
  • The process should allow for constant communication and engagement that is patient-centered and solution-oriented, including the opportunity for appeal