This perspective, written by David Whitrap, was first shared in our Weekly View e-newsletter which summarizes the week’s most significant drug pricing news. To subscribe, click here. 

Season’s greetings! It’s been a tough year, and we’re all looking forward to vaccines and a return to some semblance of normalcy.

But COVID-19 wasn’t the only thing that happened in 2020. Despite taking a three-month research pause to allow patient groups and other key stakeholders to focus on the pandemic, this year ICER completed reviews of treatments for bladder cancer (just yesterday!), cystic fibrosis, hemophilia A, migraine, NASH, sickle cell disease, and ulcerative colitis, as well as a review of digital health technologies for opioid use disorder. We also launched ongoing assessments of treatments for Alzheimer’s disease, anemia in chronic kidney disease, atopic dermatitis, high cholesterol, lupus nephritis, and multiple myeloma, as well as reviews of supervised injection facilities for opioid use disorder and service dogs for US veterans with post-traumatic stress disorder. We unveiled our new Value Assessment Framework and published white papers on Cornerstones for Fair Patient Access and Alternative Policies for Drug and Vaccine Pricing During a Pandemic. And stay tuned for next month’s report on Unsupported Price Increases.

Reminiscing about these varied accomplishments, I’d like to do something a little different with this final Weekly View of 2020, and recap some of the more important ICER storylines from the past 12 months. A more typical edition of this newsletter will be back in your inbox in early January. Until then, from all of us here at ICER, we wish you a happy holiday season and a healthy new year. And as always, we thank you for your continued support of our efforts to nudge the US health system toward fair drug pricing and fair patient access. OK, let’s take a trip down memory lane…

ICER Analytics™ Learn more


Last month, we launched ICER Analytics, a new cloud-based platform that will revolutionize the ability of payers, life science companies, patient groups, and others to develop formularies, negotiate drug prices, and explore new ways to apply evidence in a drive toward a health system that can achieve fair prices and fair access for all. While all of ICER’s reports will remain in the public domain as downloadable PDFs from our website, ICER Analytics comprises two tools geared toward frequent users of ICER’s work: the ICER Evidence Compendium™, a rapidly searchable and sortable summary of results from ICER’s library of evidence reports; and the ICER Interactive Modeler™, which enables stakeholders to customize our economic models based on their own assumptions and cost structures. We’re working with the National Health Council to enable many patient organizations to access ICER Analytics for free. In addition, we’re providing free access to all HTA organizations from low- or middle-income countries. For all other stakeholders, subscription prices will be scaled by organization type and size. Any potential users interested in learning more are encouraged to complete this survey. And if you missed yesterday’s webinar, where representatives from both the payer and pharmaceutical industries demoed the tool and discussed how they plan to turn ICER insights into policy action, you can watch the recording here.

ICER ‘s COVID-19 Work Learn more

This summer, ICER published a white paper summarizing the advantages and disadvantages of six alternative approaches to price treatments during a global pandemic: unrestricted pricing (i.e., status quo), cost-recovery pricing, value-based pricing, monetary prizes, compulsory licensing, and advanced market commitments and subscription models. To complement the white paper, ICER hosted a three-part online colloquium series featuring leading health policy and industry experts who debated which of these approaches would strike the optimal balance of innovation, affordability, and access for forthcoming COVID-19 treatments.

In addition, ICER constructed a model calculating what a cost-effective price and a cost-recovery price would be for remdesivir (Veklury®, Gilead Sciences) as a treatment for COVID-19, and we published rolling updates to our model to reflect emerging data throughout the summer and fall. In our most recent update, we suggested that a cost-effective price benchmark for a course of remdesivir would be $2,470 for hospitalized patients with moderate-to-severe disease, and $70 for patients hospitalized with milder disease. For Gilead to simply recoup its costs, we recommended a price range of $5-$600 if only considering the marginal cost of production, and a range of $1,005-$1,600 if also considering the company’s forecasted 2020 clinical development expenses related to remdesivir. Our remdesivir analyses have informed the national debate among policymakers around what a “fair” price would be, and the report continues to be broadly cited across media outlets like ABC News, The Associated Press, Axios, Barron’s, BBC, BioPharma Dive, Bloomberg, The Boston Globe, Business Insider, CBS News, CNN, The Economist, Endpoints News, FiercePharma, Forbes, Fortune, Inside Health Policy, Investor’s Business Daily, JAMA, NBC News, The New York Times, NPR, Pink Sheet, Politico, Reuters, Rolling Stone, Slate, STAT, US News & World Report, Vox, The Wall Street Journal, The Washington Post, Wired, and USA Today

ICER’s Contribution to HTA Methods Development Learn more

While ICER continues to emerge as a nonprofit health technology assessment (HTA) organization within the US, we have taken several strides to help evolve HTA methods around the world. As part of updating our 2020 Value Assessment Framework, we concluded a year-long collaboration with the National Institute for Health and Care Excellence (NICE) in the UK and the Canadian Agency for Drugs and Technologies in Health (CADTH) to develop a modified approach for assessing the value of these potential cures that include: 1) reporting on both optimistic and conservative scenarios around long-term durability of benefit; 2) voting on additional non-clinical elements of value, such as a new treatment choice that offers a different balance or timing of risks and benefits; and 3) proposing alternative price benchmarks that would reflect a drugmaker’s choice to “share” some of the savings of extreme cost offsets back with the health system. We demonstrated these modified methods for the first time in our recent assessment of hemophilia A therapies.

Also this year, we announced a first-of-its-kind program that will enable all HTA agencies to gain access to ICER’s cost-effectiveness models via ICER Analytics. The goal of this initiative is to enhance the transparency of HTA assessments around the world, enable access to ICER models for all HTA groups, including those from lower and middle income countries, and to enable the international HTA community to inform governmental decisions about drug pricing and coverage with economic model results that are rapidly available, based on the best available science, and locally tailored to reflect diverse health systems. International HTA organizations already participating in this program include the United Kingdom’s National Institute for Health and Care Excellence (NICE), Australia’s Technical Assessment and Access Division (a division of the Australian Government Department of Health), the Canadian Agency for Drugs and Technologies in Health (CADTH), Quebec’s Institut national d’excellence en santé et en services sociaux (INESSS), and MOSAIC, a South African health economic modeling collective. Already, INESSS has cited its use of ICER’s COVID-19 model to inform the Institute’s conditional reimbursement recommendation for remdesivir to Quebec’s Minister of Health and Social Services. Within the ICER Interactive Modeler, INESSS was able to evaluate and adjust key model inputs — e.g., clinical improvement and mortality assumptions — to match the province’s population, cost structure, and approved indication.

ICER’s Collaboration with the US Department of Veteran’s Affairs Learn more

Three years ago,the Department of Veterans’ Affairsbegan integrating ICER’s research into its pricing negotiations to provide greater value to Veterans and taxpayers. At the time, there was some consternation from industry, with the National Pharmaceutical Council asking whether the partnership could simply be “…a means to justify an even more limited and cost-constrained formulary?” But in a 2020 article in Health Affairs, leaders from the VA and ICER looked back at the actual impact of the collaboration, revealing that the VA has never used a negative ICER report to limit access to a medication, but instead has used our work to achieve better pricing from manufacturers, while expanding access to the treatments that are most valuable to Veterans and their families. After providing several specific examples, the authors concluded:

“The collaboration shows that a health care system in the US can utilize independent cost-effectiveness analyses as an additional information resource to help make more focused clinical and financial decisions. Through this effort the VA has gained an objective, transparent standard to guide its drug price negotiations, and the results have not undermined in any way the clinical focus of the VA drug coverage process. We look forward to a continued collaboration on behalf of our Veterans and US taxpayers in the quest to provide crucial medications at the most reasonable prices possible.” In August, we signaled continued growth of this relationship when we announced that the VA has commissioned a special ICER assessment of the cost-effectiveness of trained service dogs for American veterans with PTSD.

New York Medicaid Saves $300 Million Learn more

Following a presentation from ICER’s President Steve Pearson, New York’s Drug Utilization Review Board voted unanimously (14-0) to seek a supplemental Medicaid rebate for Spinraza, Biogen’s treatment for spinal muscular atrophy treatment. If achieved, the resulting price would represent a 79% discount of Spinraza’s list price — consistent with ICER’s recommended health-benefit price benchmark. During the public meeting, the state’s Department of Health reinforced that these upcoming negotiations with Biogen will not jeopardize access for any patient; the state is simply seeking a price that better aligns with the treatment’s benefits.

New York has saved nearly $300 million since 2018 by using this process — anchored by a potential public meeting where an ICER analysis would be presented — to negotiate supplemental Medicaid rebates on approximately 50 drugs.  It’s no wonder other states like Massachusetts are pursuing similar approaches.

NASHP’s Model Law on Unsupported Price Increases Learn more

In July, NASHP unveiled a model law that would allow states to use ICER’s Unsupported Price Increases reports as an independent method of determining which price hikes can’t be justified by new clinical evidence. With that information, state legislatures would be able to fine manufacturers for those unsupported price increases, and then use those fines to fund financial assistance to patients. Health policy experts Michelle Mello and Stacie Dusetzina provided a detailed implementation roadmap for states considering NASHP’s recommended approach, concluding:

“The Massachusetts and ICER models are promising models for curbing excessive prescription drug prices due to large price increases. Both models bring large price increases under scrutiny while acknowledging that when evidence shows a drug is delivering good value, high prices may be worth paying.”

Stay tuned for our next Unsupported Price Increases report, to be published next month

ICER’s White Paper on “Fair” Access Learn more
  • ICER Publishes White Paper on Cornerstones for Fair Patient Access to Prescription Drugs, Launches Annual Assessment of US Payer Policies

    In September, ICER published a new white paper proposing cornerstones of “fair” patient access. The paper analyzes the ethical/practical dimensions of insurance coverage, and it presents criteria for assessing the fairness of specific cost sharing and utilization management policies.

    In conjunction, we launched a new annual assessment into how coverage policies of major US insurers and PBMs align with fair access criteria, with our first report to be published by mid-2021. Our external working group — comprising more patient advocates than all other stakeholders combined — will help us develop a review protocol for this annual initiative. We all want every American to have sustainable access to high-value care, and the entire US health system will need to blend fair pricing and fair access to get the job done.
ICER’s Patient Engagement Program Learn more

In our 2020 Value Assessment Framework, we announced the creation of a more comprehensive Patient Engagement program where we’ve committed to 1) offering to help patient organizations prepare for ICER assessments; 2) using what matters most to patients as the North Star of ICER assessments; 3) translating patient input into our economic models; 4) getting patient feedback on the assessment as it progresses; 5) having patients at the head table throughout public deliberation; and 6) partnering with patient organizations after the completion of an ICER assessment to encourage the broader adoption of key policy recommendations by the FDA.

We are deeply committed to working with the patient community — those who know the condition, know the diversity of patient experience, and who can bring a truly comprehensive view to the table on what matters most to them. Only when patient community input is made central to any assessment of value can we usefully judge the evidence on how well drugs improve the quality and length of patients’ lives.

ICER’s Patient Engagement team welcomes the opportunity to engage with patient and consumer advocacy organizations to help everyone understand ICER’s role in the US healthcare system, as well as provide practical information on how to participate in an ICER assessment for a specific healthcare innovation. We’re committed to helping patient communities have a positive experience working with ICER on individual assessments as well as on longer-term partnerships to advance fair pricing, fair access, and future innovation.

ICER and Aetion on Real-World Evidence Learn more
ICER’s Open Letter to the Cystic Fibrosis Community Learn more

An Open Letter to the CF Community

In August, we hosted our first virtual public meeting, to review treatments for cystic fibrosis. Trikafta, Vertex’s newest CFTR modulator, earned an “A”, ICER’s highest and rarest rating for comparative clinical effectiveness, indicating that we have high certainty that the treatment delivers substantial health benefits. However, the prices set by the manufacturer — costing many millions of dollars over the lifetime of an average patient — are out of proportion to the CF drugs’ substantial benefits. When a manufacturer has a monopoly on treatments and is aware that insurers will be unable to refuse coverage, the lack of usual counterbalancing forces can lead to excessive prices. Patients who receive the treatments will benefit, but unaligned prices will cause significant negative health consequences for many unseen individuals — those throughout society who will experience financial toxicity and may have to delay care, forego care, or even abandon insurance as their out of pocket costs and premiums are driven upward. Nevertheless, as we emphasized throughout our public meeting and in our open letter to the CF community, we do NOT believe that the appropriate reaction to the overpricing of these treatments is for insurers to decline coverage.

ICER’s Podcast: “A Prescription for Fair Drug Pricing” Learn more
  • An ICER Podcast: A Prescription for Fair Drug Pricing

    In ICER’s new podcast series, neurologist Jason Crowell sat down with ICER’s President Steve Pearson to pry open the black box of US drug pricing and to wrestle with each of the financial and ethical tensions that undergird our current medical infrastructure. They dove into why prescription drugs are so expensive in our country, uncovered the tradeoffs that American patients face when they can’t afford drugs, explored how other countries use clinical data to ensure a drug’s price reflects how well it works, and recommended individual steps that all of us can take — from physicians to patients to policymakers — to achieve both fair pricing and fair access across the US health system.

    This is a must-listen for patients, physicians, and caregivers who are concerned that prescription drugs not only can have health side effects, but financial side effects, too. Find our episodes on AppleGoogleSpotify, or wherever you typically get your podcasts. We also list all the episodes on our website. (Quick confession: this week’s episode — which explores the non-quantifiable considerations policymakers should consider alongside cost-effectiveness models — is my favorite so far.)
Supervised Injection Facilities and Digital Health Technologies for Opioid Use Disorder Learn more
Reuters Profile: Big Pharma Wages Stealth War on Drug Price Watchdog Learn more
  • Reuters: Big Pharma wages stealth war on drug price watchdog

    In a feature article, Reuters’ reporter Caroline Humer went to great length to detail how ICER has grown more influential in helping the US health system better align a drug’s price to how well the treatment works, and how some pharmaceutical lobbyists have responded by launching a “phony grassroots campaign” to disingenuously question our methods and motives. While the whole article is worth reading, here’s one important excerpt:“Last year, ICER invited input as it revamped its assessment methods. Two of more than 50 comment letters came from six California veterans’ groups, who blasted an ICER contract with the U.S. Department of Veterans Affairs (VA), saying its formula denies veterans care and ‘inherently discriminates’ against people with disabilities. But no one from the veterans’ groups wrote the complaints. Officials from the organizations told Reuters they lent their names to letters that were composed instead by Peter Conaty, the pharmaceutical industry’s go-to lobbyist in California. A half dozen health policy specialists told Reuters that the veterans’ complaints look like part of an “astroturf” campaign — a phony grassroots movement backed by corporate interests. Conaty did not respond to requests for comment. Such under-the-radar PR efforts underscore the industry’s determination to protect its pricing power in an era when expensive new drug therapies are increasingly under fire for their role in soaring U.S. healthcare costs.”
Drug Pricing Legislation Under the Biden Administration Learn more
  • BioCentury: What about the German negotiation model? Biden steers drug pricing debate to a showdown
  • Endpoints News: Biden, Germany And Bringing A National Drug Pricing Negotiation Process To US
  • Pink Sheet: Biden looks to Germany for answers on how to tackle high U.S. drug prices: analyst
  • Politico: Biden team looking to Europe on drug pricing

    When President-Elect Biden suggested that Medicare drug-price negotiations should follow a model similar to what’s used in Germany, where the negotiations between government and drugmaker are informed by an independent analysis of how much better a new drug is than what’s already on the market, several media outlets mentioned ICER as one organization that could provide this sort of unbiased assessment. Reflecting on the possibility in early November, BioCentury concluded, “ICER’s focus on value-based pricing has positioned it to be a major voice on drug pricing in the US, whoever wins the election.” 

    A few excerpts from other experts…

    Dan Mendelson, Founder of Avalere Health: “ICER has developed and emerged as one of the only credible voices on comparative effectiveness, cost-effectiveness and the value of medicines… I think there are benefits [for the US government] having outside voices to opine on things politically contentious.”  

    Edmund Pezalla, CEO of Enlightenment BioConsult and former VP for Pharmaceutical Policy at Aetna: “We’re seeing a trend of payers paying a lot more attention to ICER. We’re looking to see what we missed, what they missed, what they viewed as important and how they viewed the quality of the studies… No one has put ICER forward for a government function, but they’re certainly set up to do that. They have the expertise, the track record, and the kinds of people needed, and they could probably scale up.” 

    Roger Longman, CEO of Real Endpoints: “It would not be unprecedented for the government to think about using a similar concept with cost-effectiveness that it does to make Medicare coverage decisions in oncology and other categories. It names five sources on which it bases a significant reimbursement program. I could see the same structure for cost-effectiveness analysis, and I think ICER will be one of perhaps several cost-effectiveness organizations.” 

    Jane Barlow, EVP and COO of Real Endpoints: “[With other assessment frameworks largely funded by specific stakeholders] there’s often a lack of credibility relative to [ICER’s] assessments, and [those others] are not really meant for use broadly in the ecosystem.” Michael Sherman, Chief Medical Officer of Harvard Pilgrim Health Care:“In many conversations with pharmas, we’re hearing that they’re seeing it as a strategic advantage to align with ICER now [to negotiate for broader payer coverage by pricing drugs at their measurable value]. It’s what ICER calls the ‘Grand Bargain.’”