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OCTOBER 21

Guest editor: Lotte Steuten  |  Vice President

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Perspective is everything. 

 

With pandemic restrictions easing in many countries and horizons finally expanding (hello, sunny beaches!), let me bring you a fresh look at the decade old debate surrounding perspectives on value in Health Technology Assessment (HTA). And why it matters. A lot.

As promised, I won’t start with stating the obvious once again. How we have all experienced first-hand the broad societal impact of the pandemic. How badly we needed some perspective to keep going while missing out on school, work, sports, social activities, while seeing our family either too much or too little. You all know that. You’ve been there. Or still are.

 

Likewise, there’s no need for me to explain that, thanks to highly effective vaccines, the lucky few of us living in countries with high vaccination rates can now start enjoying the broader value of vaccination. Whether that comes to you in the form of grabbing a bite and a drink with friends, or in finally racing that marathon you trained for during lockdown: one way or another, we’re figuring out how to live with COVID-19 rather than fear dying from it as much.

 

For all that has been written on the broader value of vaccines over the last 18 months or so, this idea is not at all new. Health economists have been calling for a broader perspective to assess the value of vaccines long before. Similar calls have been made for assessing the value of new antibiotics, acknowledging that a too narrow perspective ignores much of their value. At OHE we have contributed our parts, investigating incentives for new drugs and vaccines tackling antimicrobial resistance (AMR), estimating vaccines return on investment from a UK governmental perspective, and showing that if broader value elements are unaccounted for in cost-effectiveness analyses, vaccines are very likely to be undervalued. No need to repeat all that.

 

Therefore, my top read of the month is the paper by Shafrin et al., 2021 that shines a light on how important broader value elements are to other interventions than vaccines or antibiotics. The short answer: broader value elements matter more for rare diseases. So now we have evidence of at least three types of interventions that accrue an important part of their value via broader value elements. Some might say, three is a crowd.

 

But why does this matter, for any intervention? First, if prices are linked to value, payers may be underpaying for certain treatments when value is too narrowly defined. If such treatments are undervalued, incentives for innovation in these areas will be reduced. Second, government research funding may not be directed to treatment areas with the highest disease burden.

 

As the importance of broader value elements becomes increasingly clear for various interventions, the question of how to appropriately deal with these as part of HTA becomes more pressing. Our latest journal publication, focused on vaccines, provides some suggestions to improve the evidence, ability, and willingness of all stakeholders involved to consider broader value elements. To truly move the needle however, it is time to start acting.

 

Please check our upcoming Events and Masterclass that will touch on this topic. As co-chair of Virtual ISPOR Europe 2021, I’m taking this chance to alert you to the closing plenary session titled: Broader Value Assessments for Special Populations and Technologies: Are We Closing a Gap or Widening a Divide? World-leading experts and decision makers will be bringing their perspective. Please join and share yours.  

 

For my part: perspective means everything, and it includes everyone. See you soon!

MY TOP READ OF THE MONTH

“Inclusion of all relevant value elements in value assessments will more appropriately incentivize innovation and improve allocation of research funding. Future research should aim to better quantify these
value elements”

For which diseases do broader value elements matter most? An evaluation across 20 ICER evidence reports  | May 2021

Shafrin et al., 2021 address the question “For which diseases do broader value elements matter most?” To figure this out, the researchers reviewed 20 reports published by the Institute for Clinical and Economic Review (ICER) in the U.S., including 49 treatments across 21 diseases of which 2 were ultrarare per ICER’s definition, 8 were rare based on the FDA definition, and 2 were oncology related. They examined to what extent specific value elements (a total of 32 elements across nine categories of broader value incl. productivity, education, household and leisure, health equity, etc.) are associated with rare disease status, treatment cost, and estimated cost-effectiveness.

They conclude that broader value elements matter more for rare diseases. Inherited retinal disease and sickle cell disease had the greatest number of broader value elements mentioned (19 and 18 elements respectively), followed by amyloidosis and Duchenne muscular dystrophy (17 elements each), and depression (16 elements). Cardiovascular disease and diabetes, by contrast, had the fewest (7 elements each).

 

If value assessment ignores a large number of value elements, treatments that have wide-ranging impacts  may be undervalued and receive inappropriately high incremental cost-effectiveness ratios. This study found an association between the number of value elements and the incremental cost-effectiveness ratios, providing some evidence that this may be the case.

Read

NEW OHE LITERATURE

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Towards a Broader Assessment of Value in Vaccines: The BRAVE Way Forward

OHE’s latest journal article in Applied Health Economics and Health Policy describes the gap between broader value elements identified in value frameworks for vaccines and those recognised in HTA of vaccines in nine developed markets, and develops expert-informed, consensus-based recommendations on how hurdles for broader value recognition could be overcome.

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Challenges in the NICE Evaluation of Multi-Indication Medicines for Rare and Ultra-Rare Diseases

Medicines for rare and ultra-rare conditions face distinct economic and ethical challenges compared to medicines for more common conditions. Based on a case study analysis and stakeholder interviews, this recent OHE Report describes these challenges and how NICE approaches them in their assessment of these medicines providing a better understanding of the intricacies of the NICE appraisal process for (ultra-)rare conditions and what this means for NHS England.

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Are user fees in health care always evil? Evidence from family planning, maternal, and child health services

OHE research on family planning goes back nearly 60 years, which provides an interesting historical insight in economists’ thinking on the topic over time. Our latest journal article on this provides evidence on the medium-term effects of introducing user fees on the utilisation of family planning, antenatal and delivery care services, women’s access to health care, and child health status in a middle-income country setting.

NEWS HIGHLIGHTS

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COVID-19 leads to >25% increase in maternal deaths

A study commissioned by UNICEF shows the devastating effect of COVID-19 on maternal and child mortality due to disruption to healthcare provision and access, and increased food insecurity in South Asia. Child mortality was 6% higher and maternal deaths more than 25% higher in 2020 than in 2019. This equates to over 228,000 deaths amongst the under-5s, and 11,000 maternal deaths.

 

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5.6 million people currently waiting for care

 

A new study by the Health Foundation estimates that there are now over 5.6 million people currently waiting for care. A third have been waiting for longer than 18 weeks; 300,000 for more than a year. In addition, there are 7.5 million ‘missing patients’ since the start of the pandemic. The NHS and social care system are going to need nearly £17bn and 10 million more staff to clear the backlog and treat expected rise in patients needing NHS hospital care.

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H.R. 3 disproportionally affects orphan medicines 

The US H.R. 3 legislation targets high-cost drugs and thus disproportionately impacts new treatments against rare diseases and cancer; areas of high unmet need. Along with many flaws in the CBO analysis underpinning H.R. 3 as previously reported by OHE,  Forbes now reports the legislation aims at high-cost drugs but does not distinguish between the value of different drugs. Some high-cost drugs may be of considerable value, that is, cost-effective, particularly in spaces with limited or no availability of treatment alternatives.

UPCOMING EVENTS

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Do patents work? Evidence from pharmaceutical innovation

14 October  |  Professor Margaret Kyle explores the empirical evidence on the effectiveness of patents in the pharmaceutical sector, specifically the rate and direction of innovation linked to patents in pharma.

FREE

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The Role of Unmet Need in Pharmaceutical Innovation

20 October |  In this free webinar will be discussing the different roles of unmet need along the development pathway and how its application might be improved to promote development in areas of genuine health need.

FREE

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Innovation for Small Populations:What Should We Pay for?

9 December  |  There remains significant unmet need with only 5% of the more than 7,000 rare diseases having treatments available today. We will tackle this issue and discuss solutions for moving forward in the area of orphan and ultra-orphan diseases.

£375

Early Bird discount valid until 09.11.2021

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