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ICER’s Methodology Needs to Take Into Account Key Stakeholder Voices

June 29, 2016
Last week, the Institute for Clinical and Economic Review (ICER) released the scoping document for their pending review of therapies that treat non-small cell lung cancer (NSCLC).[1]

The scoping document itself lays out the importance of these therapies given the realities of this deadly disease:[2]

  • Lung cancer is the leading cause of cancer death in the U.S.[3]

  • It is predicted to kill 158,000 Americans this year.[4]

  • NSCLC makes up about 85 percent of lung cancers,[5] and patients with NSCLC commonly present with advanced disease.[6]

  • Five-year survival rates are grim: only 31.4 percent of patients whose cancer has spread regionally are expected to survive, and that number drops to 4.9 percent for patients whose cancer has spread more widely throughout the body at the time of diagnosis.[7]

But despite these statistics, there is reason to hope.  As of 2015, there were more than 120 medicines in development for lung cancer.[8]  The next generation of medicines is taking a smarter approach to attacking these tumors by targeting therapies based on a patient’s genetics and developing medicines that activate a patient’s own immune system to better fight the cancer.  Based on the potential of these therapies, we should be doing everything we can to spur innovation—and attract investment—in this space to be able to deliver ever-more effective treatments to patients and to work continuously toward cures.  Once these innovative therapies come to market, assessing their value to individual patients, the healthcare system, and society as a whole will be important to ensure they are used appropriately, efficiently, and effectively.

With these goals in mind, BIO continues to raise concerns with ICER’s “value” assessment methodology—used to review therapies like those that treat NSCLC—which can vastly undervalue these advancements, and in doing so, contribute to limiting patient access to needed therapies in the short-term and hindering innovation in the longer-term.  We have raised these concerns thoughtfully and in detail to ICER in the past.[9]  These concerns include that ICER’s approach:

  • Can obscure the value of an innovative therapy to an individual patient by relying on flawed metrics, like quality-adjusted life years (QALYs);

  • Shortchanges the value of an innovative therapy to the healthcare system and society by relying on a short-term economic impact analysis that cannot capture the long-term benefits and cost offsets of therapies that treat chronic conditions, which affect patients for years or even decades;

  • Features a value-based pricing benchmark, which can easily be taken out of context, and which is limited by a one-size-fits-all budget impact threshold that penalizes a more productive drug development and regulatory review process (i.e., since the threshold’s denominator reflects the number of Food and Drug Administration (FDA) approvals, as more medicines reach the marketplace, the overall threshold will shrink); and

  • Relies on extremely short deadlines to receive public feedback and has not broadly incorporated stakeholder input—in particular, patients’ perspectives—in the development and refinement of the methodology itself.

The comment period on the NSCLC scoping document closed yesterday, after just a week, and we don’t know what the ICER review will ultimately say.  What we do know is that the broader discussion ICER is contributing to is a critical one, and our member companies are eager to participate—a commitment they made earlier this year through BIO’s Principles of the Value of Biopharmaceuticals.  But a discussion based on unrealistic deadlines and one that is missing so many critical voices will ultimately mislead patients and the public with regard to the value of an individual therapy, and the value of innovation more broadly.  What’s worse, it will risk disrupting the innovation ecosystem that has brought treatment for NSCLC—and so many other diseases—this far, and is poised to bring it much farther.

We continue to urge ICER to work broadly with stakeholders to reform their methodology to put patients at the center of healthcare decision making.  We also continue to urge all stakeholders not to rely on a single voice for guidance on the complex issues of value and innovation—as tempting as that may be.  Rather, we should all work to contribute to an inclusive, evolving discussion on value and innovation that is focused on ensuring patient access and sustaining biopharmaceutical innovation.

[1] For more information on the ICER Drug Review project, see ICER. Programs: CTAF, Midwest CEPAC, New England CEPAC, available at:

[2] ICER. 2016 (June 21). Treatment Options for Advanced Non-Small-Cell Lung Cancer: Effectiveness, Value, and Value-Based Price Benchmarks—Drat Background and Scope, available at:

[3] Centers for Disease Control and Prevention (CDC). Lung Cancer, available at: (last accessed June 28, 2016).

[4] National Institutes of Health (NIH) and National Cancer Institute (NCI). SEER Stat Fact Sheets: Lung and Bronchus Cancer, available at: (last accessed June 28, 2016).

[5] American Cancer Society. Lung Cancer (Non-Small Cell), available at: (last accessed June 28, 2016).

[6] Id.

[7] NCI. SEER Cancer Statistics Review 1975-2013. Section: Lung and Bronchus, Table/Figure: 15.14 “5-Year Relative and Period Survival (Non-Small Cell Cancer),” available at: (last accessed June 28, 2016).

[8] PhRMA. Medicines in Development for Cancer: A Report on Cancer, available at:

[9] BIO. 2016 (May 4). Follow-Up on BIO’s Comments in Response to the ICER Value Framework, available at: