Amgen’s Response to ICER’s Call for Comments on its “Updated” Value Assessment Framework

Executive Summary

Amgen welcomes the opportunity to comment on ICER’s “Updated Value Assessment Framework”. We appreciate ICER’s engagement of stakeholders in revising its Framework and we hope that ICER will seriously consider and incorporate our recommendations for the sake of better achieving its goal of a more sustainable healthcare system for all patients.

The US needs more rigorous systematic assessments of new technologies to help decision-makers better understand the value of new interventions relative to other innovations and existing care. Organizations that seek to play a potentially important role and command greater credibility should define their role as one that offers guidance and informs decisions with a systematic and consistent approach to the evaluation of evidence. ICER therefore, has an opportunity to take a longer-term view of its own role in health care valuation by being a credible partner in helping understand the complex science of health care assessment and bringing this potential thoughtfulness to all of the stakeholders in medicine. Budget-holders and decision-makers can benefit if ICER focuses on key pillars of evidence and its strengths, clarity on the key assumptions required to assess value, identification of the real drivers of the value of interventions, and robust analytics using state of the art methods and the identification of important areas of uncertainty. This ultimately allows the budget-holders and decision-makers to leverage ICER’s insights in making decisions. These should be decisions that are truly based on a firm understanding of value, not the healthcare equivalent of a “top ten list”, over-simplification of decision problems, methods and data inputs that are known to be arbitrary or inaccurate, and decision rules that “anchor” panelists to arbitrarily fixed decision states. This leads to the mischaracterization of clinical complexity and the value of technology. 

Defining its role as such also aligns ICER with its stated mission to help the United States evolve towards a health care system that provides sustainable access to high-value care for all patients”.1 This mission is not well-served by the current paternalistic and clearly agenda-driven approach of pushing for nationwide willingness-to-pay and budget impact thresholds that are arbitrary and entrenched in invalid methodology. An objective assessment of the value of a technology, leaving the door open for interpretation, discussion and debate, is also more durable and intrinsically valuable than imposing one’s philosophy regarding what society should pay and can afford to pay for various services. The extremely dynamic nature of healthcare delivery and the multidimensionality of health technology assessment are best served with broad ranges and robust science rather than one-dimensional hard thresholds and arbitrary math.

ICER’s updated Value Framework unfortunately take steps that are counter to what the US healthcare system needs right now, and we believe run counter to ICER’s “stated” mission. We hope that ICER will take the opportunity to correct these major shortcomings before publishing its Final Updated Framework. While the original ICER Framework had many limitations, there were at least some critical elements of flexibility and judgment (“other contextual considerations”) built into the panel’s discussion and voting that enabled the panels to better contemplate value beyond the confines of a rigid threshold. We are concerned that many of ICER’s proposed updates now take steps in the wrong direction using an arbitrary points scale to force the panel into an arbitrarily lower QALY threshold range and confining judgments on value with limited flexibility and specificity to the US market. This flexibility and ICER’s potential ability to tap into the expertise of the panel to consider the complex but qualitative side of value is an example of a key aspect of the process that enabled some credibility into discussions of value that would be largely lost in the new process. 

What is Needed What ICER is Doing Now
  • Eliminate the judgmental QALY range and simply compute QALYs objectively
  • Inappropriate QALY range specification
  • Do not institute another arbitrary points scale for "other considerations" but let the panel continue to exercise discretion
  • New and arbitrary scale for additional considerations
  • Introduce more patients, providers, and other health care stakeholders to the panel
  • Panel still tilted towards insurance and payers
  • Total transparency of models
  • Still unable to replicate ICER analyses
  • Identification of the most important areas of uncertainty with extensive sensitivity testing
  • Still too focused on one answer and "picking winners"
  • Abandon the groundless ~$900M budget impact threshold
  • Budget impact threshold still arbitrary
  • Abandon picking a single price and discuss ranges instead
  • Price calculations are designed for shock value, not real discussion and debate

We urge ICER to consider and incorporate the below recommendations to help align its role with its stated mission and enable more objective and robust dialogues that inform decisions on value: 

  1. ICER’s potential future value lies in the objective systematic assessment of various technologies to provide guidance and inform decisions on value. ICER’s legitimacy and accountability in setting national arbitrary thresholds will be subject to substantial contention. 

    Recommendation    : Focus on a role centered on guidance, informing decisions on value based on key pillars of evidence and its strengths, robust analytics and the identification of areas of uncertainty; and do so with flexibility, inclusiveness, scientific integrity, transparency and patient centricity, in the absence of ‘one size fits all’ absolutes and thresholds. This ultimately allows the budget-holders and decision-makers to leverage ICER’s insights in making their decisions on value. 

  2. ICER’s proposed QALY threshold lacks scientific merit and specificity to the complex US healthcare system. Also, ICER’s attempt to anchor their QALY threshold at $50,000 is irrelevant to the current dynamic environment.

    Recommendation    : Do not attempt to define national QALY thresholds on behalf of the US public, and instead leave thresholds up to each respective decision-maker. Disaggregate QALY assessment from the QALY thresholds.

  3. ICER’s revised process disempowers its public appraisal committees (voting panels) in determining the impact of contextual criteria

    Recommendation: Eliminate the arbitrary points weighting and QALY threshold constraints imposed upon the committee by the new process. More directly include valuation methods in the framework (network meta-analysis and cost-effectiveness model) that allow value and contextual considerations to have a greater impact.

  4. In defining value, the patient perspective and that of other key stakeholders remain muted on ICER’s public appraisal committees

     Recommendation: Though there has been some progress in this area, ensure more equal stakeholder representation in defining value within ICER’s Framework. Democratize the public appraisal committees (not just the policy roundtable) so that they are composed of more patients, caregivers, patient advocates, clinical experts and manufacturers to help determine the value of new treatments.

  5.  ICER’s limitation of costs to the health system will lead to decisions that shift costs to patients, their caregivers, employers and society

     Recommendation: Payer-borne and monetary costs are only one aspect of healthcare burden. Include costs and cost savings resulting from treatment that are relevant to all stakeholders, including direct non-medical costs, such as patient and caregiver out-of-pocket costs and lost productivity costs within ICER’s Framework (e.g., cost-effectiveness model) as is recommended by the Panel on Cost-effectiveness in Health and Medicine.

  6. ICER’s decision-making based on its proposed budget impact model (which is arbitrary and lacks scientific merit) continues to be detrimental to the health needs of patients

     Recommendation: ICER should not attempt to set a national budget impact threshold. In defining its role as one that is advisory, ICER should help articulate to the decision-makers what types of elements should be included in their specific budget impact calculations. As such, ICER should base its budget impact on credible assumptions, rather than arbitrary math (e.g., consider the heterogeneity across diseases and indications, and displacements costs), and allow objective third parties to validate their approach. 

  7. ICER models used for analysis continue to lack transparency, availability and replicability

     Recommendation: Make research methods, assumptions, data inputs, and equations available in a completely transparent manner, such that results are fully reproducible by third parties and are reviewed by known experts.  Put in place a process that allows for the external validation of model calculation, structure and variables.

  8. ICER Assessments lack consistency in timing, approach and assumptions from assessment to assessment

     Recommendation: Develop reliable and valid methods, based on appropriate timing and fair-balanced disclosures and assumptions. For example, do not make specific assumptions on pricing if price is not set. Testing ranges would be more appropriate. 

The above represents the Executive Summary of Amgen's Response. Amgen’s Full Response is available upon request.

REFERENCES

  1. Institute of Clinical and Economic Review (ICER). opt cit p.2 Link