The Most Important ICER Storylines from 2020

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 Publishes White Paper on Alternative Policies for Drug and Vaccine Pricing During a Pandemic, Announces Cross-Stakeholder Discussion Series
• ICER Provides Second Update to Pricing Models for Remdesivir as a Treatment for COVID-19

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 Finalizes Method Adaptations for Assessing Potential Cures and Other High-Impact Single or Short-term Therapies
• In Landmark International Program, HTA Agencies Around the World Access ICER’s Customizable COVID-19 Cost-Effectiveness Model

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.

• Health Affairs: VA And ICER At Three Years: Critics’ Concerns Answered

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.

• STAT: New York panel seeks extra Medicaid rebates from Biogen for pricey rare disease drug
• The Incidental Economist: In Pursuit of Value-Based Drug Prices in the US: New York and Beyond
• The Boston Globe: Reining in the spending on drugs: What Mass. can learn from other states

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.

• National Academy for State Health Policy (NASHP): NASHP’s New Model Law Penalizes Drug Manufacturers for Unsupported Price Hikes

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 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 Appoints Yvette Venable as Vice President of Patient Engagement

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 Partner to Develop Real-World Evidence for Value Assessment of Treatments
  
  Shortly after the announcement of our 2020 Value Assessment Framework, which promised to expand use of real-world evidence (RWE) throughout our reports, ICER and Aetion announced a partnership and platform to generate decision-grade RWE.
  
  Aetion shares our general view of both the strengths and weaknesses of RWE: there are areas where insights can be gleaned appropriately, and there are other areas where researchers should remain skeptical. We hope that this partnership will set new standards for how RWE can better inform the consequential decisions pharmaceutical companies and health insurers make around drug pricing and patient access. News of our partnership was covered by Reuters, Pink Sheet, BioPharma Dive, STAT, Endpoints News, Politico, MedCity News, and The Washington Post. Stay tuned next year for the results of our initial RWE pilot.

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.

• 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 Apple, Google, Spotify, 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.)

• ICER Publishes Final Evidence Report and Policy Recommendations for Digital Health Therapeutics for Opioid Use Disorder
• ICER Publishes Evidence Report Finding That Supervised Injection Facilities Save Lives and Money
  
  We assessed the comparative clinical effectiveness and economic value of supervised injection facilities (SIFs) and digital health technologies (DHTs) for opioid use disorder. Along with past ICER assessments of related topics such as abuse-deterrent opioid formulations, medication-assisted treatment for opioid use disorder, and non-drug interventions for lower back pain, these two new opioid-related non-drug topics reinforced ICER’s commitment to provide policymakers with an independent evaluation of the evidence around interventions that address the public health crises stemming from the US opioid epidemic.
  
  Our Final Evidence Report on DHTs found that the current evidence is inadequate to demonstrate a net health benefit for reSET-O, Connections, or DynamiCare digital technologies, and that reSET-O at its current price represents low long-term value for money. To prove the value of digital health technologies that have a therapeutic function, experts on ICER’s policy roundtable called for randomized controlled trials, or at a minimum, high-quality observational studies using an appropriate comparator and patient-relevant outcomes. Our SIFs Evidence Report found that, for communities which experience high rates of overdose, the evidence is adequate to demonstrate that SIFs not only prevent overdose deaths, but that they also lower overall community costs by reducing the need for emergency care.

• 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.”

• 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.’”