CER Daily Newsfeed

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

Beneficiaries with Cancer Spend Less Out-of-Pocket in Medicare Advantage

(9/12, Victoria Bailey, Health Payer Intelligence) reports “Medicare Advantage beneficiaries with cancer experienced lower out-of-pocket costs and had access to more benefits than those in Medicare fee-for-service (FFS), research conducted by ATI Advisory and commissioned by the Better Medicare Alliance found...Specifically, those with Medicare Advantage spent $3,996, while those with FFS spent $6,091, for a difference of $2,095.” Full

The Evolution of Precision Medicine in Pharmacy

(9/11, Rayn Oswalt, Pharmacy Times) comments “...[P]recision medicine holds immense promise in revolutionizing the field of pharmacy and transforming patient care. By leveraging patient-specific genetic, environmental, and lifestyle information, pharmacists can tailor drug selection, dosing, and treatment monitoring to optimize outcomes and minimize [adverse effects]. As technology and research progress, the future prospects for precision medicine are bright, with expanded genetic markers, increased availability of pharmacogenomic testing, and personalized treatments for rare diseases on the horizon. However, challenges related to data privacy, cost, and patient awareness must be addressed to realize the full potential of precision medicine.” Full

Japan Takes New Steps To Build On Real-World Data Foundations

(9/11, Lisa Takagi, Pink Sheet) reports “...As Japan widens its window for the use of real-world data to support drug development though revision of the Next Generation Medical Infrastructure Act, the pharma industry is hoping for more regular inclusion in regulatory filings. A dedicated group within the PMDA shares its views on how the area is developing and practical measures being taken to drive RWD adoption.” Subscription Required

CMS Clarifies Factors It Will Consider In Negotiating Drug Prices

(9/8, Gabrielle Wanneh, InsideHealthPolicy) reports “...Relating to evidence available on therapeutic alternatives to the selected drugs, CMS will also consider: The extent to which the selected drug represents a therapeutic advance compared to existing therapeutic alternatives; The costs of the therapeutic alternatives; The FDA-approved prescribing information available for selected drugs and their therapeutic alternatives; The comparative effectiveness of selected drugs, including the impact for specific populations, such as individuals with disabilities, the elderly, the terminally ill, children, and other populations.” Subscription Required

Pharmaceutical Innovativeness Is Highly Variable, As CMS Will Soon Find Out When It Assesses Drugs It Selected For Negotiation

(9/9, Joshua Cohen, Forbes.com) comments “...The key question when conducting comparative effectiveness evaluations is whether the pharmaceuticals being analyzed are clinically better than existing standards of care. And if so, then HTA must probe whether the incremental benefit is worth the price premium? As was said above, among the top-selling prescription drugs sold to Medicare most were not considered high value products.” Full

Big Pharma's Battle With the Biden Administration Could Have Legs

(9/9, David Wainer, The Wall Street Journal) reports “...[Carmel Shachar, assistant clinical professor at Harvard Law,] ultimately thinks the law has a good chance of surviving, but she notes that in an era where courts have shown growing skepticism toward administrative agency action anything is possible. She says the industry argument that the government is unfairly taking their property, which is protected by patents, will be looked at seriously by some courts.” Subscription Required

Real-World Data Can Include EUA Observations, US FDA Final Guidance Says

(9/10, Bridget Silverman, Pink Sheet) reports “...Agency clarifies expectations for non-interventional studies providing RWD to support regulatory decisions while continuing to emphasis familiar themes around early interaction and stringent data practices...The considerations guidance focuses on non-interventional clinical study designs, which are not subject to IND regulations; other RWE-using trial designs, like external controls, have been highlighted in their own guidance documents. "There weren't huge changes" between the draft and final versions, Aetion head of scientific strategy Ulka Campbell observed in an interview with the Pink Sheet.” Subscription Required

Uncertainties Still Loom Over IRA Negotiations

(9/7, Angela Maas, AIS Health) reports “...‘The decisions around the key question of which therapeutic alternatives will be selected as comparators, how clinical superiority and clinical net benefits are demonstrated and how CMS plans to assign a price value to that difference in this first year will come too late for the manufacturers selected today and might come too late for the manufacturers selected for the next year of negotiation as well,’ [NPC president and CEO John O’Brien, Pharm.D.] maintains. ‘Manufacturers currently face uncertainty about what evidence should be developed and submitted, whether decisions explained in the concise justifications during the first year of negotiation under the program will be the same decisions made in future years and whether the people making the decisions next year will be the same. How this list was put together is a great question for the White House and HHS,’ he tells AIS Health. ‘The many questions people have about today’s announcement are a reminder of the lack of transparency and tremendous uncertainty surrounding the process.’” Full

Biden's Price Control Scheme Harms Patients, Compromises Free Market

(9/7, Tom Price, RealClearHealth) comments “...To preserve the pipeline of new innovative treatments, therapies, and vaccines, drug companies must have the opportunity to recoup investment rather than being forced to sell products at below market rates. Unlike Uncle Sam, businesses don't have the option to continuously spend beyond their means and take on trillions of dollars in debt. The Biden administration doesn't seem to grasp this concept. The White House's price control scheme-approved by his allies in Congress-is moving forward at full steam ahead. As a result, the free-market incentive to create new medicine will fade-compromising the health of future Americans.” Full

The Reality of Drug Pricing Negotiations Isn't a Slam Dunk for Patients

(9/7, Kenneth E. Thorpe, RealClearHealth) comments “ ...Congressional Budget Office prepared an estimate projecting changes in utilization rates for a similar drug price negotiation program that preceded the IRA. To achieve expected savings under the program, the score estimated reduced utilization for some medications of more than 25 percent. This disruption to access has no medical justification. Price negotiations will also have a chilling effect on drug research - and thereby diminish the number of new medicines available to patients in the years and decades to come. One recent study from Vital Transformation estimates that over the next decade, the IRA's drug price negotiations will result in 139 fewer medicines being developed.” Full

Video: Jeffrey Sparks, MD, MMSc: Treatment Strategies for Rheumatoid Arthritis

(9/7, HCP Live) “...[T]he number of choices has also made it more challenging to select the most suitable therapy. Additionally, most of the trials leading to drug approvals have compared the new drugs with placebos, with a limited number of head-to-head trials. In his presentation, he discusses these head-to-head trials to provide evidence of efficacy. Further, he explores real-world evidence, examining comparative effectiveness studies that assess different drug classes against each other.” View Video

NAACOS: Medicare Payment Incentives Favor Clinicians in Fee-For-Service

(9/8, Victoria Bailey, RevCycle Intelligence) reports “...According to the NAACOS blog post, MACRA’s incentives for Payment Year 2026 and Performance Year 2024 will favor clinicians who do not participate in advanced APMs and remain in the Merit-Based Incentive Payment System (MIPS). In 2026, MIPS clinicians will receive a 0.25 percent conversion factor update and can receive additional positive payment adjustments in MIPS. The average MIPS adjustment is typically 3 percent, meaning the total potential payment adjustment would be 3.25 percent. Meanwhile, clinicians in advanced APMs will only receive a 0.75 percent conversion factor update. Incentives will not favor clinicians in advanced APMs again until 2032.” Full

Lilly's Diabetes Drug Mounjaro Approved by UK Watchdog

(9/8, Reuters) reports “Eli Lilly's diabetes drug Mounjaro has gained the backing of Britain's healthcare cost-effectiveness watchdog, which said it would be a good option for patients with poorly controlled type 2 diabetes. The National Institute for Health and Care Excellence said in draft final guidance that it estimates some 180,000 people could benefit from the new treatment.” Full