BIO Submits Comments Re: ICER Value Framework
RE: National Call for Proposed Improvements to its Value Assessment Framework
Dear Dr. Pearson:
The Biotechnology Innovation Organization (BIO) appreciates the opportunity to provide feedback in response to the Institute for Clinical and Economic Review’s (ICER’s) National Call for Proposed Improvements to its Value Assessment Framework (the “Framework”).1 BIO is the world's largest trade association representing biotechnology companies, academic institutions, state biotechnology centers, and related organizations across the United States and in more than 30 other nations. BIO’s members develop medical products and technologies to treat patients afflicted with serious diseases, to delay the onset of these diseases, or to prevent them in the first place. In that way, our members’ novel therapeutics, vaccines, and diagnostics not only have improved health outcomes, but also have reduced healthcare expenditures due to fewer physician office visits, hospitalizations, and surgical interventions.
BIO appreciates the efforts ICER has made over the last several months toward improving the public comment process associated with each step of a Drug Review, including the current national call for feedback on the underlying Value Framework methodology. The responsibility to take into account broad stakeholder perspectives and address stakeholder concerns is increasingly important given the emerging evidence that ICER Reviews have the potential to impact patient access to needed medicines.2 However, while ICER has taken steps to improve the ability to collect stakeholder feedback, the Institute’s stated goal of “work[ing] collaboratively with patient groups, clinical experts, and life science companies” requires a much clearer understanding with regard to how stakeholder input is meaningfully incorporated.3 In this letter, BIO addresses this and other outstanding issues and responds to the four major categories of issues ICER identifies in the call for comments.
Though the majority of the methodological concerns BIO has raised in the past persist,4 and are reiterated throughout this letter, we continue to raise several primary issues that must be resolved in the new iteration of the Framework.
- First, ICER should not continue to conflate the impact of a therapy on patient health outcomes, including quality of life, with the potential budget impact to any individual payer or group of payers. To clarify, BIO supports the concept of assessing the value of different therapies to an individual patient to facilitate the right medicine getting to the right patient at the right time. However, we also adamantly assert that the final decision of which therapy, or combination of therapies, is most appropriate for a patient must: be left to the patient working with his or her provider; consider the individual clinical circumstances of the patient; and assess the impact of a therapy on a patient over the long term. The updated Framework should facilitate—not hinder—this aim.
- Second, we continue to strongly urge ICER to ensure that the Framework relies on robust and validated methodological standards and applies them consistently and transparently.
- Third, we appreciate and agree with ICER’s stated goal of undertaking a value assessment that, among other aims, seeks to “fairly reward innovators for the value they bring to patients, and provide them ample incentive to pursue the investments and research that will lead to the innovative treatments of tomorrow.”5 However, the Framework does not work to achieve this aim. For example, it does not address significant concerns with regard to the potentially negative impact on the innovation ecosystem, nor does it attempt to quantitatively account for the need to sustain innovation. Moreover, the current structure of the budget impact threshold penalizes increased regulatory efficiency that results in bringing more therapies to market each year.
- Fourth, while we acknowledge ICER’s progress with respect to broader stakeholder engagement (e.g., evidenced by the extended duration of certain public comment opportunities), we strongly urge the Institute to establish a robust engagement strategy with patients in particular. The Institute should work with patients to identify how their input can inform each step of the process, how to standardized engagement with this community, and identify the primary point of contact at the Institute for patients who may have questions outside of an official comment period.
These issues, as well as related methodological and process concerns, are discussed in detail throughout the remainder of this letter. We urge ICER to address each of these issues—and BIO’s accompanying recommendations—in an updated version of the Framework, or, describe why ICER believes these changes were not warranted.