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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.
https://tinyurl.com/BMJcea
In our new @Heart_BMJ article, we use 3 case studies to clarify key concepts like cost-effectiveness & affordability: #tafamidis, #PCSK9i & #DOAC.
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.
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?
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?
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).
, 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?
-> #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.
. 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 alsofrom $750Million in 2012 to $4.7Billion in 2016. Unsurprisingly, Medicare formulariescost-sharing/prior auths.
. But as DOAC adoption, Medicare spending on anticoagulation alsofrom $750Million in 2012 to $4.7Billion in 2016. Unsurprisingly, Medicare formulariescost-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.
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.
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
