February 13, 2019
Steven D. Pearson, MD, MSc, FRCP
Institute for Clinical and Economic Review
One State Street, Suite 1050
Boston, MA 02109 USA
RE: Draft Unsupported Price Increase Assessment Protocol
Submitted electronically via: firstname.lastname@example.org
Dear Dr. Pearson:
The National Pharmaceutical Council (NPC) shares your interest in optimizing the value of health care spending, and we have launched the Going Below the Surface initiative to broaden the conversation around the use of health care resources. Hence, another ICER initiative that is focused solely on drugs, rather than the entire health care system, is truly a missed opportunity. Drug spending accounts for only 16% of the U.S. health care dollar. A fully informed and productive dialogue must consider the other 84%, too.
NPC is a health policy research organization dedicated to the advancement of good evidence and science, and to fostering an environment in the United States that supports medical innovation. NPC is supported by the major U.S. research-based biopharmaceutical companies. We focus on research development, information dissemination, education and communication of the critical issues of evidence, innovation and the value of medicines for patients. Our research helps inform critical health care policy debates and supports the achievement of the best patient outcomes in the most efficient way possible.
NPC’s Guiding Practices for Patient-Centered Value Assessment are a useful lens for viewing the concerns we have with ICER’s proposed unsupported price increase assessment protocol. Of particular note are the breadth, robustness, and subjectivity of the assessment process. Seven guiding practices particularly relevant to ICER’s proposed protocol are presented below.
Value assessments should focus broadly on all aspects of the health care system, not just on medications. (Guiding Practice VII)
Optimizing our health care resources by shifting our health care system from a volume-based focus to a value-based focus requires an examination of the entire system. Medications account for only 16% of health care spending, yet ICER puts almost 100% of its resources towards examining medications.
NPC recommends that ICER shift resources to meaningfully examine the rest of the health care system.
Sufficient time, staff and resources should be dedicated to support a thorough and robust assessment process. (Guiding Practice VI)
What ICER hopes to accomplish with this report — in a relatively short timeframe — is an incredibly time-intensive and unprecedented undertaking. ICER notes in its draft protocol that “ICER does not have the capacity to perform full economic analyses on the large number of therapies that will be subject to analysis as part of this new report process. …Therefore, these UPI reports are not intended to determine whether a price increase for a drug is fully justified by new clinical evidence.” Considering these resource constraints, ICER should avoid making determinations, or at least add extensive caveats and acknowledge limitations.
NPC recommends that ICER add caveats to any determinations and acknowledge their limitations.
ICER notes it does not have the resources to answer the question of whether price increases are supported by new evidence and, hence, does not seek to answer this question. This approach is only designed to identify cases where ICER believes the price increases are unsupported; it does not seek to identify supported price increases.
This one-sided methodology will only present the biopharmaceutical industry in a negative way without highlighting any positives. Taking a one-sided approach runs contrary to ICER’s previously stated mission: “Our aim is not to support one side in a negotiation; it is to provide what our health care system has lacked for so long: an independent, trustworthy source of information that can bring all voices into the discussion on value.” This one-sided approach does not contribute to a constructive discussion about drug prices and health care spending.
NPC recommends that ICER undertake more comprehensive reviews that can identify cases where price increases are aligned with value.
The measurement of value should include a broad array of benefits that are important to patients and society. (Guiding Practice XIII)
While manufacturers are invited to share information based on non-clinical information, this will not be incorporated into ICER’s considerations: “… non-clinical rationales will not be evaluated by ICER as a determinant in whether the drug is categorized as having its price increase unsupported by clinical evidence.”
Patients and society value more than clinical outcomes, including economic and humanistic types of outcomes. Ignoring non-clinical information such as health care resource utilization, medical cost offsets, work productivity, patient preference and/or caregiver burden dismisses these factors. We should encourage investment in all aspects of the patient experience and not place emphasis solely on clinical development.
NPC recommends that ICER expand its analyses to include non-clinical information.
Subjective evidence should be used minimally, if at all, and its inclusion should be clearly labeled. (Guiding Practice XXV)
Sensitivity analyses should be performed, taking into account input from external stakeholders. (Guiding Practice XI)
Whether a product’s price increases are labeled as unsupported hinges on ICER’s subjective assessment of the size of the clinical effect demonstrated by new evidence — if the effect is deemed “small,” the increase is labeled unsupported; if the effect is considered “substantial,” the unsupported label is not applied.
There are no transparent criteria to differentiate between “small” and “substantial” effects — the categorization process lacks specificity and is not replicable. Further, there are no sensitivity analyses to explore the range of effects that lie between the binary choices of “small” and “substantial.”
NPC recommends that ICER use transparent and replicable ratings criteria and incorporate sensitivity analyses.
Methods, models and assumptions should be transparent and assessment results should be reproducible. (Guiding Practice IX)
ICER’s proposed methodology lacks specificity. As noted above, the categorization of evidence scoring is subjective, and no complete definition or academic references have been provided by which to assess best-use cases. It is unclear how ICER will weight outcomes (overall survival vs. progression-free survival, for example), or whether evidence related to a new indication will be weighted more or less than additional outcomes or safety evidence for an older indication.
ICER should avoid using terminology and phrases that are imprecise or lack objectivity, e.g., “extremely high price increases”; “fell just below”; and “raise concerns about fairness.” Such terms may potentially alienate stakeholders, particularly in the absence of transparent and clearly stated assumptions. The normative basis of “fair,” “unsubstantiated,” “substantial,” etc., should be made explicit and transparent.
NPC recommends that ICER clarify methods and terminology to facilitate transparency and reproducibility.
Stakeholders should be given the opportunity to submit relevant evidence, such as clinical trial and real-world evidence beyond the public literature. (Guiding Practice XXI)
While ICER does give manufacturers the opportunity to submit relevant evidence, only some of this evidence will be protected. Proprietary clinical information will be protected under the ICER’s “academic in confidence” policy; proprietary confidential financial information, however, will not be protected. Manufacturers’ confidential commercial and trade secret information have significant trade protections under law and regulations in many contexts. These protections should be recognized by ICER and extended to information manufacturers may choose to submit in response to an ICER inquiry. Failure to provide complete protection will limit the types of information that manufacturers can submit and, therefore, provide an incomplete picture of value.
NPC recommends that ICER fully protect the confidentiality of manufacturer information.
In addition, the evidence review limits the amount of evidence considered by an arbitrary cutoff of approximately 10% or more of the drug’s use. There is a huge need for better treatment of rare conditions and many pediatric indications, and this arbitrary cutoff appears to disregard important areas such as these. This disadvantages products with multiple indications and is in opposition to Food and Drug Administration incentives to research and invest in smaller, yet high burden, disease areas.
NPC recommends that ICER reconsider the 10% utilization threshold when examining new data.
ICER states in its draft protocol: “there has been no systematic approach at a state or national level to determine whether certain price increases are justified by new clinical evidence or other factors.” ICER’s proposed approach does not achieve this objective. NPC encourages ICER to:
- focus on the entire health care system,
- undertake more comprehensive reviews that can identify cases where price increases are aligned with value,
- add caveats to determinations and note their limitations,
- expand its analyses to include non-clinical evidence,
- use transparent and replicable ratings criteria and sensitivity analyses,
- clarify methods and terminology to facilitate transparency and reproducibility, and
- protect the confidentiality of manufacturer information.
Robert W. Dubois, MD, PhD
 ICER Unsupported Price Increase Assessment Draft Protocol. https://icer-review.org/wp-content/uploads/2019/01/ICER_UPI_Draft_Protocol_011719.pdf. Accessed January 22, 2019.
 Kleinrock M, et al. Reconciling the Seemingly Irreconcilable: How Much Are We Spending on Drugs? Value in Health, 2019, online ahead of print. https://www.valueinhealthjournal.com/article/S1098-3015(18)36310-1/fulltext. Accessed January 29, 2019.
 Guiding Practices for Patient-Centered Value Assessment. National Pharmaceutical Council. http://www.npcnow.org/sites/default/files/npc-guiding-practices-for-patient-centered-value-assessment.pdf. Accessed January 22, 2019.
 ICER FAQ. https://icer-review.org/wp-content/uploads/2016/01/ICER-FAQ-052316.pdf. Accessed February 11, 2019.