CER Daily Newsfeed

NPC Evaluates ICER’s Revised 2023 Value Assessment Framework: A Missed Opportunity for Patients and the Field

(9/25, National Pharmaceutical Council: E.V.I.dently Today) comments “...Consistent with NPC’s comments, ICER did not lower its threshold range when stating its opinion on the value of treatments. However, ICER continues to threaten to put its thumb on the pricing scale of innovative treatments. Doing so signals a narrowly focused interpretation of a treatment’s value and can do more harm than good to patient access. ICER documented enhanced approaches to patient engagement. However, it remains to be seen whether these efforts will lead to more patient-focused inputs being incorporated into ICER’s analyses and results vs. being another box-checking exercise.” Full

ICER Finalizes Its Approach to Value Assessment. Industry Remains Dissatisfied

(9/26, Brian Reid, Cost Curve) comments “...The topline conclusion is that ICER’s approach, at its core, isn’t going to change that much...Industry had hoped for better. ‘Look, value assessment is not a rule, it is a tool comprised of both science and opinion,’ NPC’s John O’Brien said via email. ‘We're very concerned about the areas that weren't addressed because they have the potential to harm patients and do a disservice to those who've dedicated their lives to the scientific application of value methods.’” Full

Practice Points Developed for Treatment of Outpatient COVID-19

(9/26, HealthDay News) reports “...‘More research evaluating the efficacy, effectiveness, and comparative effectiveness, as well as harms, of pharmacologic and biologic treatments of COVID-19 in the outpatient setting is needed, particularly in the context of changing dominant severe acute respiratory syndrome coronavirus 2 variants and subvariants,’ the authors write.” Full

Advancing Value-Based Care: AMA, AHIP and NAACOS Team up to Launch New Playbook

(9/26, Dan Cook, Benefits Pro) reports “...[A] collaboration of three major organizations has launched a campaign to address the barriers that have so far clipped VBC’s wings. AHIP, the American Medical Association (AMA), and the National Association of ACOs (NAACOS) unveiled their barrier-busting strategy recently with the release of a playbook designed to work VBC adopters over and under the obstacles. The playbook–The Future of Value-Based Care and Payment–went public with the support of a half-hour webinar devoted to the first phase of implementation: data sharing.” Full

FDA’s Califf: Expect to See More RWE-Based Regulatory Decisions

(9/22, Ferdous Al-Faruque, Regulatory Focus) reports “...[FDA commissioner Robert Califf] said that using RWE to expand product indications has been a sound theory, but it has taken a long time to make it a reality. ‘I'm optimistic we're going to make a real run at it in this upcoming year, across the whole federal government, not just within the FDA,’ said Califf. ‘I believe that we're moving very rapidly now to a place where more and more of the data we use for evidence is going to come from the so-called real-world clinical environment. After all, the reason we do a randomized trial is to take a sample of people and reach a causal inference that allows us to extrapolate to the population,’ he added.” Full

Finding The Sweet Spot For Real-World Evidence: Aetion Stresses Early Feasibility Assessment

(9/24, Bridget Silverman, Pink Sheet) reports “...Robust natural history data sets with well-chosen outcomes are fundamental in Aetion’s regulatory feasibility tool for real-world evidence, built through analysis of US FDA case studies and guidance.” Subscription Required

Editorial: Here's How Medicare Should Negotiate Drug Prices

(9/22, Bloomberg) comments “...[Other countries including Australia and the UK] regularly use cost-effectiveness assessments and, unsurprisingly, their prescription-drug spending is a fraction the US’s. Unfortunately, the process for Medicare won’t be so straightforward. With QALYs banned, it will need to use other (less tested) methodologies. Even so, effective alternatives exist, including some that have been developed to minimize the disparities that disability advocates cite. Medicare should embrace these metrics and be transparent with the public about its pricing methods.” Subscription Required

Congressman Yakym Introduces Legislation to Bring Greater Awareness to Lifesaving Medical Devices

(9/22, Kathy Bottorff, WTCA 106.1) reports “This week, Congressman Yakym (IN-02) introduced H.R. 5458, the ‘Baby Observation Act’ or ‘BO’s Act.’...‘BO’s Act’ requires the Patient-Centered Outcomes Research Institute to study the use of home cardiorespiratory monitors (medical devices that track the respiratory effort and heart rate of a sleeping infant) to reduce and prevent sleep-related sudden infant death syndrome or sudden unexpected infant death.” Full

How Can Predictive Analytics Help ACOs Boost Value-Based Care Delivery?

(9/25, Shania Kennedy, Health IT Analytics) reports “...In the past, ACOs have relied on Medicare data and risk adjustment factor scores to gain insights into their patient populations, but these are limited in that they cannot capture all the complexities associated with a patient and her health. Digital twins—digital or virtual representations of people, objects, processes, or systems designed to help simulate a potential outcome—represent a ‘third wave’ of analysis, [David Clain, chief product officer at the Health Data Analytics Institute,] posited, tying de-identified data from these and other sources to an ACO population.” Full

Gene Therapy HTA: How Do The European, Australian And Canadian Systems Shape Up?

(9/25, Eliza Slawther, Pink Sheet) reports “...A report comparing the health technology assessment methods used in nine European countries, Australia and Canada found that England has the most favorable reimbursement landscape for gene therapies – but outlined several areas for improvement across the board.” Subscription Required

Industry-Funded Study: CBO Underestimated IRA’s Impact On Drug R&D

(9/22, Gabrielle Wanneh, InsideHealthPolicy) reports “A policy brief recently released by the University of Chicago and partially funded by drug maker Gilead suggests that Medicare’s new drug price negotiation authority could lead to a reduction in research & development that translates to 79 fewer small molecule drugs developed and 116 million life years lost over the next two decades.” Subscription Required

Rx Price Controls Mean Hard Choices for Investors

(9/21, John Stanford, RealClearHealth) comments “...Small molecule drugs currently make up 75% of all FDA-approved medications, including some modern blockbusters. Several of the first medicines subject to government price controls are small-molecule drugs that have had significant impacts on disease -- including one that helps lower blood-sugar levels for patients with type 2 diabetes and another that inhibits B-cell proliferation to slow the spread of blood cancers. It's fair to ask whether their developers would have proceeded with these medicines if they had known that their products would be among the first subject to price controls.” Full

After FDA Rejection, Lilly's Ulcerative Colitis Drug Omvoh Picks up Support from England's NICE

(9/22, Eric Sagonowsky, Fierce Pharma) reports “...Friday, England’s National Institute for Health and Care Excellence recommended the drug for the treatment of patients with moderate to severe active ulcerative colitis who can’t tolerate—or haven’t responded to—certain other treatments. The NICE blessing sets up the drug, known in the U.K. as Omvoh, to be the first IL-23p19 inhibitor to be used in NHS England, Lilly said in a release.” Full

Panel: Alternative Funding for Pharmaceuticals Brings Numerous Risks

(9/21, Karen Blum, Pharmacy Practice News) reports “...Alternative funding models may pose significant risks to individual patient access and the health system, said Mr. Einodshofer and co-panelists Corey Belken, PharmD, a national account manager for Genentech, and John Michael O’Brien, PharmD, MPH, the president and CEO of the National Pharmaceutical Council, a health policy research organization. Disadvantages include the following: They may interfere with patients receiving timely treatment since delays in care could lead to disease progression or a decline in health status; Patients with complex health needs may not have access to the right level of specialty support through the alternative funding vendor.” Full

How Patient Apps Can Help Unlock The Benefits Of Real-World Data

(9/21, Kal Patel M.D., MBA, Forbes.com) comments “...Many pharma companies look to companion apps or patient trackers to initiate patients onto new treatments and ensure that they’re adherent and compliant with those regimens...That initial RWD can offer some valuable insights immediately to help optimize digital tools and reveal patient journey pain points. And RWE holds even greater potential, both for improving patients’ lives and pharma’s bottom line. But to gain this untapped potential, brand and RWE teams must not be kept in separate silos—that prevents developing the necessary holistic approach that can drive value and insights for both teams.” Full

A Powerful Methodology to Guide Health Decision-Making

(9/21, Harvard T.H. Chan School of Public Health) reports “...[O]bservational studies from the past decade or so suggested that patients who took statins, which are drugs used to treat heart disease, had around half the risk of cancer. However, randomized trials showed that there was no such effect, suggesting that statins should not be used for cancer prevention. To resolve the discrepancy, Dickerman and her colleagues analyzed a U.K. national database of electronic health records and found that using causal inference methods would have resulted in the correct conclusion found in the randomized trials. ‘This was an important case study to show how you can run into major bias simply by applying the wrong analytic methods to observational data,’ she said. ‘It also proposed a better way forward that may serve as a model for investigators who use electronic health records for cancer comparative effectiveness research.’” Full

Two Pediatric Sinusitis Antibiotics Found Equally Effective

(9/29, Chris Dall, MA, CIDRAP) reports “The results of a large observational study in children show no difference in treatment failure rates between the two most commonly prescribed antibiotics for pediatric acute sinusitis, researchers reported today in JAMA. Overall, treatment failure for sinus infections was rare in children treated with amoxicillin or amoxicillin-clavulanate. But the latter was associated with more adverse events...” Full

Value-Based Payments Are Making it Harder to See Your Primary Care Doctor on Short Notice

(9/20, Jeffrey Millstein, STAT) comments “...Physician leaders must push for value-based incentives that better align with patient needs and goals, and allow primary care clinicians the autonomy to balance routine and acute/subacute visits to meet practice demands. For instance, limiting yearly comprehensive visits to patients in the highest risk subgroups should meet the metric threshold.” Full

Shutdown Threat Clouds Start of Biden Drug-Pricing Negotiations

(9/20, Alex Ruoff, Bloomberg Law) reports “...A potential government shutdown at the end of the month risks complicating the Biden administration's top health priorities, just as drugmakers are supposed to report data for pricing negotiations and states are pushing people off Medicaid rolls...A shutdown would bump up against an Oct. 2 deadline for some drugmakers to report a slew of data to Medicare as part of how the agency will eventually determine new prices for medicines selected for negotiation.” Full

Real-World Data Or Studies Of Competitor Drugs Can Serve As Confirmatory Evidence For US FDA

(9/18, Brenda Sandburg, Pink Sheet) reports “...Draft guidance describes seven types of confirmatory evidence that can be used with one adequate and well-controlled clinical study to demonstrate substantial evidence of effectiveness, including evidence from expanded access, real-world data, and studies of other drugs in the same class.” Subscription Required

FDA's Single-Trial Guidance Calls for the Good, Bad and Ugly Data to Support Effectiveness

(9/19, Annalee Armstrong, Fierce Biotech) reports “...To show confirmatory evidence, drug sponsors have plenty of options and they may not have to reinvent the wheel to find it. The FDA provided some examples...Options include efficacy evidence from a previously submitted or approved indication for a drug; preclinical data from an established animal mode; evidence from therapies in the same pharmacological class; natural history data; real-world data or evidence; and data from an expanded access program.” Full

Paxlovid and Lagevrio Benefit COVID Outpatients in Omicron Era

(9/19, Diana Swift, Medscape) reports “...‘The practice points only address [whether] treatments work compared to placebo, no treatment, or usual care,’ cautioned Linda L. Humphrey, MD, MPH, MACP, chair of the ACP's Population Health and Medical Science Committee and a professor of medicine at Oregon Health and Science University VA Portland Health Care System. The ACP continues to monitor the evidence. ‘Once enough evidence has emerged, it will be possible to compare treatments to each other. Until that time we are unable to determine if there is an advantage to using one treatment over another.’” Subscription Required

Dr Ryan Haumschild Discusses Payer, Provider Perspectives on Prior Authorization in Rare Diseases

(9/19, Pearl Steinzor, The American Journal of Managed Care) reports “...[Ryan Haumschild, PharmD, MS, MBA:] As we're evaluating rare diseases, we need to look at the value they bring. We need to look at value not just on acquisition cost, we need to look at it from total cost of care perspective or quality of life perspective, or even looking at the ICER and the quality to say, ‘Does this meet the minimum threshold?’ And if it doesn't, we need to have those open conversations with providers. Maybe there might be a sub-population that may benefit the most, but not all populations are going to benefit. And be very clear about that.” Full

Is ICER's New Digital Health Value Assessment Framework Relevant as Technology Pivots to OTC?

(9/15, Kimberly Westrich, LinkedIn) comments “...Last week, No Patient Left Behind published a white paper titled, ‘Getting the Math Right When Measuring the Value of New Medicines,’ which found that traditional cost-effectiveness analysis methods often omit sources of social value that, when incorporated, move the needle on cost-effectiveness estimates. The authors reviewed ICER assessments for 20 medications, only 8 of which were found to be cost-effective using ICER’s traditional CEA methods. However, after applying generalized cost-effectiveness analysis (GCEA) to account for 2 additional dimensions of value, including diminishing returns to health improvement and dynamic pricing, the authors found that at least 17 of the 20 assessed medications offered good value for money from a societal standpoint.” Full

Blackstone's $250M Bet Beats Xarelto in Bleeding Trial, Making Case for Anthos' Ex-Novartis Asset

(9/18, Nick Paul Taylor, Fierce Biotech) reports “...The data show Anthos Therapeutics’ abelacimab significantly cut bleeding compared to Bayer and Johnson & Johnson’s Xarelto, adding to evidence that the biotech's candidate can treat thrombosis without affecting hemostasis.” Full

EMA Waves Goodbye To HTA Network As Collaboration Framework Enters New Phase

(9/18, Eliza Slawther, Pink Sheet) reports “...A report published by the European Medicines Agency highlights milestones reached under its collaboration with EUnetHTA over recent years and outlines areas for further development once the initiative changes hands under the new HTA Regulation.” Subscription Required

Real-World Data in a Post-Pandemic World

(9/15, Surani Fernando, Medical Marketing & Media) reports “...As for the FDA, when asked via email about source-related challenges, the spokesperson responded: ‘In regards to real-world data sources, we find issues related to data reliability and clinical relevance, a need for linkage to other data sources, missing or “mistimed” data and insufficient capture of endpoints. For non-randomized study designs, issues include the threat of residual confounding, problems with index date (“zero time”) and use of an inappropriate comparator.’” Full

Three Outstanding Questions About CMS’s Ambitious New AHEAD Model

(9/14, Troyen A. Brennan, Health Affairs Forefront) comments “...As a hospital executive, your key strategy, perhaps your only strategy, has been to increase in size, gain leverage with insurers, bargain for better fee-for-service rates, and do more procedures. Working under a prospective budget blocks that strategy. So one might ask about Pennsylvania, would centers like UPMC in the west, and Jefferson or University of Pennsylvania in the East, be ready to abandon the fee-for-service revenue generation program for one based solely on value-based care that improves population management. I wish that were true, but it seems doubtful.” Full

Americans Overwhelmingly Support Medicare Drug Negotiations, But Biden Sees Little Political Boost

(9/15, Seung Min Kim and Linley Sanders, Associated Press) reports “...Three-quarters of Americans, or 76%, favor allowing the federal health care program for the elderly to negotiate prices for certain prescription drugs. That includes strong majorities of Democrats (86%) and Republicans (66%), according to a new poll from The Associated Press-NORC Center for Public Affairs Research. About one in five Americans are neutral on the issue, while 6% outright oppose it. But the same poll shows Biden's approval rating, at 40%, is about where it's been for the last year.” Full

US FDA Commissioner Pushes Real-World Evidence To Guide Use Of Alzheimer’s, Obesity Drugs

(9/13, Kate Rawson, Pink Sheet) reports “...Califf has been particularly interested in using real-world evidence in the post-approval setting, which he described as a ‘system in shambles.’ He reiterated that ‘we're not talking about the fundamental structure of drug and device approval. I think that works pretty well. We are talking about what happens after that, where FDA is only part of the system.’ Califf reiterated his view that other countries have a longer life expectancy and a better quality of life (he mentioned Singapore, specifically), ‘because they have organized approaches to information.’ Israel uses real-time electronic health data, he noted, and the UK National Health Service enables a streamlined approach in which clinical trials are integrated directly into clinical care, ‘in other words, real-word evidence.’” Subscription Required

Pushing Both Sides of the Drug Pricing Aisle

(9/14, Asher Mullard, Nature Reviews Drug Discovery: An Audience With) reports “Few topics are as controversial in healthcare as drug pricing. As prices edge ever higher, budgets are stretched ever thinner. And while some patients and clinicians clamor for new and better therapeutics, others question the value of the drugs that are reaching the market. The Institute for Clinical and Economic Review...has leaned into this tension, developing systematic and transparent methods to assess the cost-effectiveness of new treatments. With ICER’s founding President Steve Pearson now stepping down, President-elect Sarah Emond is ready to take up the medical mantle.” Subscription Required

England's NICE Backs Pfizer's Vydura as Treatment for Acute Migraines

(9/13, Kevin Dunleavy, Fierce Pharma) reports “...This time, NICE still stopped short of recommending Vydura for primary use. According to the draft guidance, rimegepant is endorsed for adults who have had unsuccessful results with at least two triptan medicines. Vydura also is suggested for use by those who can't take triptans and had unsuccessful results with nonsteroidal anti-inflammatory drugs (NSAIDs) or the beta blocker paracetamol. ‘NICE understands that rimegepant could eventually be used in primary care but should initially be started in secondary care,’ the regulator said...” Full

Osimertinib Plus Chemotherapy Demonstrated Greater PFS Benefit in Patients With EGFRm Advanced NSCLC Than Osimertinib Alone

(9/12, Justina Petrullo, The American Journal of Managed Care) reports “Osimertinib with the addition of chemotherapy showed a statistically significant and clinically meaningful progression-free survival (PFS) benefit compared with only osimertinib in patients with epidermal growth factor receptor–mutated (EGFRm) advanced non–small cell lung cancer (NSCLC) in the FLAURA2 study, according to results presented at the International Association for the Study of Lung Cancer 2023 World Conference on Lung Cancer.” Full

A Framework For Prioritizing Pharmaceutical Supply Chain Interventions

(9/13, Marta E. Wosinska, et al., Health Affairs Forefront) comments “...To assess criticality is to address the following question: Without which pharmaceuticals will individual patients suffer great harm? The answer to this question will necessarily consider the severity of the condition the medicine treats and the comparative effectiveness of the drug relative to alternatives. In the criticality assessment, it is important to consider heterogeneity of treatment effect and the drug’s importance to specific vulnerable subgroups that face challenges in accessing needed medical care.” Full

Pharma Cries Procedural Foul in Next Front of Drug Price War

(9/13, Ian Lopez, Bloomberg Law) reports “...AstraZeneca's complaint in particular has gotten the attention of legal experts. The company argues that the HHS overrides the statutory definition of Qualifying Single Source Drug, for which there's no biosimilar or generic competition, and essentially lumps together separately approved products and justifies making them eligible for negotiations...‘They're claiming an excess of statutory authority,’ and ‘they have a point,’ said Robert Charrow, who served as HHS general counsel in the Trump administration.” Subscription Required

America’s New Drug-Pricing Rules Have Perverse Consequences

(8/30, The Economist) comments "…According to research published in 2021, once their spending on research and development is treated as an investment rather than an expense, pharma firms are not making outsize returns compared with the average firm in the S&P 500. If they doubt that they will make a sufficient profit on their investments, they will spend less on finding new drugs. Sure enough, studies suggest that falling revenues hit research and development spending hard. This is typical of the unintended and undesirable effects from price regulation. Lower prices are popular with patients today and mean less of a drain on the public purse in the near term. But if they discourage investment in new medicines, that will be to the detriment of patients and society tomorrow.” Subscription Required

Pharma Industry Urges HTA Bodies To Look Beyond Survival Data For Cancer Drugs

(9/13, Eliza Slawther, Pink Sheet) reports “...A new report by industry association EFPIA says that health technology assessment bodies often rely too heavily on overall survival data when assessing the value of novel cancer drugs, and should take into account other clinical and patient-reported outcome measures where appropriate.” Subscription Required