BIO Submits Comments Re: ICER Proposed Value Assessment Framework Revisions
April 11, 2017
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) Call for Comments on Revised Value Assessment Framework (the “Value 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 that ICER has provided an additional opportunity for stakeholder comment in advance of finalizing revisions to the Value Framework. While some of the proposed revisions have the potential to make progress toward aligning the Framework with the principles of individualized patient care and holistic value assessment, ICER must provide additional details with regard to how these proposed revisions will be operationalized before stakeholders can assess whether the revisions will meaningfully address critical gaps in the Framework.
However, we remain concerned that the proposed revisions to the Value Framework do not address the central concerns that many stakeholders raised during the initial comment period. Specifically, even with the proposed revisions, the Value Framework would still:
Inappropriately 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;
Fail to uniformly rely on robust and validated methodological standards, and apply them consistently and transparently; and
Fall short of fulfilling ICER’s stated goal of “fairly reward[ing] 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.”[2]
The balance of this letter will provide feedback that is intended to align the Value Framework with established research methodologies and standards for analytic robustness and accuracy. BIO’s recommendations on ICER’s proposed revisions fall into two broad categories: (1) those revisions that require additional detail, and potentially even operationalization, to determine whether they are wholly responsive to BIO’s recommendations; and (2) critical issues that ICER did not address—or did not address adequately—in the proposed revisions. We urge the Institute to work collaboratively with a diverse range of stakeholders, including manufacturers and patients, to revise the Value Framework further to promote patient-centered healthcare and incentivize biopharmaceutical innovation over the longer term.
While our recommendations throughout this letter are made with specific reference to the Value Framework, we urge ICER to apply our recommendations in the context of the Institute’s broader work as feasible. For example, we recognize that ICER is studying certain types of therapies, including gene therapies and medicines that treat patients with rare diseases, outside of the existing Drug Review and Value Framework process. We believe this approach is appropriate given the unique issues associated with these types of therapies. In particular, BIO appreciates that ICER has acknowledged that the revised Value Framework will not apply wholesale to ultra-orphan disease therapies due to considerations around clinical trial design and evidence, size of the potential patient population, differences in demands of research and development, and the need to weigh other benefits and disadvantages and contextual considerations. That said, we nonetheless strongly urge ICER to ensure that its other initiatives employ robust and transparent analyses, are patient-centric, and do not underestimate the value of innovative new therapies.
Under the 340B program, participating manufacturers must offer 340B pricing on their covered outpatient drugs by covered entities, as a condition of having those drugs federally payable under Medicare Part B and Medicaid. Critically, Congress…
BIO submitted these comments in response to the United States Patent and Trademark Office’s May 10, 2024, Notice of Proposed Rulemaking regarding Terminal Disclaimer Practice to Obviate Nonstatutory Double Patenting.
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) Call for Comments on Revised Value Assessment Framework (the “Value 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 that ICER has provided an additional opportunity for stakeholder comment in advance of finalizing revisions to the Value Framework. While some of the proposed revisions have the potential to make progress toward aligning the Framework with the principles of individualized patient care and holistic value assessment, ICER must provide additional details with regard to how these proposed revisions will be operationalized before stakeholders can assess whether the revisions will meaningfully address critical gaps in the Framework.
However, we remain concerned that the proposed revisions to the Value Framework do not address the central concerns that many stakeholders raised during the initial comment period. Specifically, even with the proposed revisions, the Value Framework would still:
Inappropriately 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;
Fail to uniformly rely on robust and validated methodological standards, and apply them consistently and transparently; and
The balance of this letter will provide feedback that is intended to align the Value Framework with established research methodologies and standards for analytic robustness and accuracy. BIO’s recommendations on ICER’s proposed revisions fall into two broad categories: (1) those revisions that require additional detail, and potentially even operationalization, to determine whether they are wholly responsive to BIO’s recommendations; and (2) critical issues that ICER did not address—or did not address adequately—in the proposed revisions. We urge the Institute to work collaboratively with a diverse range of stakeholders, including manufacturers and patients, to revise the Value Framework further to promote patient-centered healthcare and incentivize biopharmaceutical innovation over the longer term.
While our recommendations throughout this letter are made with specific reference to the Value Framework, we urge ICER to apply our recommendations in the context of the Institute’s broader work as feasible. For example, we recognize that ICER is studying certain types of therapies, including gene therapies and medicines that treat patients with rare diseases, outside of the existing Drug Review and Value Framework process. We believe this approach is appropriate given the unique issues associated with these types of therapies. In particular, BIO appreciates that ICER has acknowledged that the revised Value Framework will not apply wholesale to ultra-orphan disease therapies due to considerations around clinical trial design and evidence, size of the potential patient population, differences in demands of research and development, and the need to weigh other benefits and disadvantages and contextual considerations. That said, we nonetheless strongly urge ICER to ensure that its other initiatives employ robust and transparent analyses, are patient-centric, and do not underestimate the value of innovative new therapies.
[1] ICER, 2017 (February 1), Institute for Clinical and Economic Review Posts Revised Value Assessment Framework for Public Comment, available at: https://icer-review.org/announcements/vaf-revision-public-comment/ (last accessed February 28, 2017).
[2] ICER, 2016, Addressing the Myths About ICER and Value Assessment, p. 2, available at: http://icer-review.org/wp-content/uploads/2016/08/icer_myths_facts.pdf (last accessed August 31, 2016).