Would you care for a pragmatic understanding of cost-effectiveness for new #cardiovascular drugs?💊💵
In our new @Heart_BMJ article, we use 3 case studies to clarify key concepts like cost-effectiveness & affordability: #tafamidis, #PCSK9i & #DOAC.
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https://tinyurl.com/BMJcea 
2) How do we decide if a new therapy represents societal value for money? Well, it depends on how we define “value”.
3) We often use #ICER. The Incremental Cost Effectiveness Ratio evaluates the Cost (numerator) and "Benefit" (denominator) of new drug 🚨 compared to old drug 🚨. If #ICER is lower than what we are willing to pay as a society (in the US ~ $100K/QALY),the new 💊 is cost-effective.
4)Did you say #QALY? Quality-Adjusted Life-Years can compare "apples to oranges", i.e. standardize the benefit of different clinical outcomes (ex. avoiding a stroke vs a bleed). It includes prolonged survival & quality of life on a utility scale from 0 (death) to 1 (full health).
5) Next, let’s define cost. Key caveat: Cost = TOTAL Cost = cost of new💊 PLUS all downstream expenses: monitoring, side effects, additional healthcare due to ⬆️ survival, but also savings when new💊 ⬇️cardiovascular events. Most new💊 likely ⬆️net costs. Is it worth the benefit?
6) Let’s see these principles in action with our 3 case studies: #tafamidis, #PCSK9i & #DOAC.
7)In 2019, #Tafamidis entered market at $225,000/yr 😱, but offered impressive 1.3 QALY gain over lifetime for #amyloid pts (compared w/ no other available rx).This generates #ICER $880,000/QALY vs usual care. To meet $100,000/QALY, cost would need to ⬇️ to $16,650/yr (93% drop).
8) In 2016, #PCSK9i entered market at $14,500/yr 😬 -> #ICER $415,000/QALY (vs statin+Zetia) 👎. Thanks to market pressure, in 2018 both manufacturers ⬇️ price by 60% to $4,500/yr -> #ICER $56,600/QALY for pts w/ high #CV risk = Cost-effective 👍. BUT are these meds affordable?
9)Affordable to who? For patients, affordability = out of pocket💵. For payors=budget impact, i.e. net change in expenditure if all eligible pts receive new💊. So even for cost-effective💊, rare vs common disease matters! Often, ⬆️cost for payors =⬆️co-pay = unaffordable for pts.
10)Total spending matters too, as in #DOAC vs #warfarin. #ICER for DOAC vs warfarin: $54,000/QALY👍. But as DOAC adoption⬆️, Medicare spending on anticoagulation also⬆️from $750Million in 2012 to $4.7Billion in 2016. Unsurprisingly, Medicare formularies⬆️cost-sharing/prior auths.
11) Thus, #DOAC represent a perfect example of a 💊 that is both clinically effective AND cost-effective, but may nonetheless be unaffordable for many patients. Certainly, upcoming patent expiration in 2022 will likely ⬇️ budget impact, ⬇️ out of pocket 💵, and ⬆️ affordability.
12)Take-home #1: clinicians should complement rigorous understanding of clinical trial results w/ cost-effectiveness principles, to understand both health needs & economic priorities of pts & health systems. This ensures high-cost new💊 reach pts most likely to benefit from them.
13) Take-home #2: the 60% historical price reduction of #PCSK9i exemplifies how rigorous cost-effectiveness analyses can help decrease cost, promote access, and mitigate healthcare disparities – especially if timely conducted to match trial release and guide pricing negotiations.
14)Thank you @kardiologykazi and Blake Liu for sharing your wisdom & expertise on how to interpret cost-effectiveness analyses and their broader societal implications on affordability. Thank you also to @SmithBIDMC for the support on this project! #MedTwitter #CardioTwitter
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