HTA

What is wrong with QALYs?

A paper by Rand and Kesselheim (2021) in Health Affairs this month conducts a systematic literature review to answer this question. Based on 113 articles they identified in peer-reviewed journals, they identify the following 10 criticisms categories. The graph above has each criticism category and the number of peer-reviewed articles that mention this critique type. …

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How to balance the patient voice atop the shifting plates of HTA

The tectonic plates of healthcare technology assessment (HTA) have shifted – but where does the patient voice fit within this new evaluation paradigm? Recent advances in medical science, from genomics and gene therapies to artificial intelligence (AI), have transformed the healthcare landscape, and triggered a review of NICE’s methods and processes. Consultation on the proposals …

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Do most Medicare Part D drugs have CEA studies?

That is the question that a recent study by Tisdale et al. (2021) aim to answer. They first look at the top 250 Medicare Part D drugs in terms 2016 total spending based on the 2016 Medicare Part D Prescriber Public Use File. Drug characteristics were identified from the 2016 FDA Orange Book. The authors …

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How ICER will conduct it’s “Barriers to Fair Access” Assessment

Last fall, ICER published a white paper titled “Cornerstones of ‘Fair’ Drug Coverage: Appropriate Cost-Sharing and Utilization Management Policies for Pharmaceuticals.” To paraphrase, ICER’s goal is to limit access restrictions (e.g., cost sharing, coverage limitations, prescriber restrictions, step therapy) when a drug is deemed to be cost effective. How will they do this? Their Barriers …

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Should supply chain quality inform treatment value?

In an article in Health Affairs, Rick van der Vegte and I argue that in four specific cases, the answer is yes. …the International Society for Pharmacoeconomics and Outcomes Research (ISPOR) developed a “value flower,” which aims to more comprehensively capture all elements of value relevant for measuring the value of a new health technology. …

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ISPOR Summit on Value Assessment

On June 10, I will be presenting at ISPOR’s Summit on “Value Assessment: New Insights, Approaches, and Applications,” which will highlight evolving practices, new methods, and practical applications in value assessment. The Summit will provide a multistakeholder perspective, including recent developments in the incorporation of uncertainty and risk aversion into value measures, covering both the …

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Rethinking access barriers to innovation

Market access issues don’t stop once a product has passed HTA – and this is especially true for disruptive therapies like ATMPs. Experts from Executive Insight discuss how a holistic, multi-stakeholder approach can help companies overcome access barriers at all stages. This article appears in our digital magazine Deep Dive: Market Access 2021. Read on …

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Galapagos UK’s Michael Smyth on RNA-based tech: the pharmaphorum podcast

Episode 35 of the pharmaphorum podcast hears from Michael Smyth about Galapagos UK and joining the company as medical director just as COVID-19 hit. With RNA-based technology hitting the headlines in recent months due to its use in Covid vaccines, he also discusses the version of it that Galapagos uses and how it can produce …

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Deep Dive: Market Access

It’s fair to say this is the most interesting time ever for market access. Not only has COVID forced companies, governments and healthcare systems to work towards approving drugs and vaccines in record times, the sector is also facing an influx of digital therapeutics and advanced drugs that don’t fit neatly into existing access frameworks. …

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A value framework for diagnostics in Latin America

That is the topic of a recent paper by Augustovski et al. (2021). The authors conduct a targeted literature review of previous diagnostics value frameworks in other countries. A full list of these articles is below. Then the researchers pool the criteria and created a proposed value framework. A panel of experts was then met …

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Is value in the eye of the beholder? Value frameworks and inter-rater reliability

A new drug comes on the market. Is the health benefit worth the cost? What is it’s value? Value assessment frameworks are one way to answer this question. Value frameworks take the evidence of the treatments benefits, risk and cost and output some notion of the treatment’s value. Ideally, if anyone applied a given value …

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ISPOR Value Flower and Alzheimer’s Disease

Standard cost-effectiveness measures the value of a treatment based on the cost for an additional unit of health where units of health are measured in quality-adjusted life years (QALYs). For Alzheimer’s disease, however, this approach may be limited. A paper by Garrison et al. (2021) identifies the petals of the value flower that are particularly …

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Technology assessment in rare diseases

What is a rare disease? The definition varies across countries, but according to the European Medicines Agency (EMA) rare diseases are those with a prevalence of <5 cases per 10,000 people; ultra-rare diseases are those with <1 case per 50,000 people. In most cases, rare disease must not only be rare but also severe, often …

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Is the value of a QALY constant?

Standard cost-effectiveness analysis assumes that any gain in quality-adjusted life years (QALYs) should be valued equally. This does not sound unreasonable, but is it true in practice? Consider two potential violations of constant value of QALY gains: scope insensitivity and severity independence. I define each of these below: Scope insensitivity. This assumes that individuals value …

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Societal vs. health system perspective: does it matter?

Cost effectiveness analysis aims to capture identify (and monetize) all of a treatments benefits, risk and costs and then determine if the treatment is worth the money. Traditionally, however, benefits, risk and costs only related to benefits that accrue to the health care system. Many studies in the academic literature have advocated for a societal …

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2021 market access prospects for France

In the latest of a suite of features looking at the biggest markets in Europe, Leela Barham takes stock of what 2021 could bring for market access in France. In September 2020, the French government published the draft of their annual review of spending on health and social care, which will form the basis of …

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What kind of HTA does the US need?

Should the US have a formal, centralized health technology assessment (HTA) organization? In the United Kingdom, the National Institute for Health and Care Excellence (NICE) plays this roles and has a significant impact on drug pricing in the UK. However, the UK relies on a single payer system and NICE’s recommendations can be directly implemented …

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Getting gene therapies to market faster

The FDA approval process is slow. Even for COVID-19 vaccines, the process can be time consuming. However, regulators around the world do have some expedited programs. However, a number of countries have programs that expedite review, particularly for the diseases with unmet need, orphan drugs, or when new treatments represent a significant advance over the …

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Okra says AI-based drug price predictor is 90% accurate

UK artificial intelligence startup Okra Technologies has launched a new software platform that it claims can take the guesswork out of the price that can be charged for new drugs, years ahead of launch. The AI system – called ValueScope – can predict the price  as well as the likely outcome of negotiations with health …

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HTA are ignoring the value of reduced caregiver burden

While many health technology assessment (HTA) organizations acknowledge that many new treatments reduce the burden placed on caregivers, the value that these new treatments provide is rarely incorporated into formal cost effectiveness analyses. This is the finding from Pennington (2020) based on a review of ll published technology appraisals (TAs) and highly specialized technologies (HSTs). …

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HTA criteria used to evaluate diagnostics

When evaluating a new diagnostic, HTA agencies must assess two separate issues: analytical and clinical validity. Analytical validity basically indicates whether the test works; is it able to accurately predicts the presence or absence of a particular biomarker of interest. Clinical validity is whether the test matters in clinical practice.  It could be the case …

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HTA criteria used to evaluate diagnostics

When evaluating a new diagnostic, HTA agencies must assess two separate issues: analytical and clinical validity. Analytical validity basically indicates whether the test works; is it able to accurately predicts the presence or absence of a particular biomarker of interest. Clinical validity is whether the test matters in clinical practice.  It could be the case …

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PCORI to incorporate cost but not cost-effectiveness in its new principles

Created by the Affordable Care Act (ACA) in 2010, the Patient-Centered Outcomes Research Institute (PCORI) has funded a wide variety of research comparing different treatments to one another. One thing PCORI was forbidden from funding, however, was cost-effectiveness analyses. Section 1181 of the Social Security Act, however, has expanded the scope of the types of …

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Where — and Why — ICER Really Matters

Check out my interview on the Redeeming Value podcast with Brian Reid. PRECISIONheor Vice President Jason Shafrin pops in to talk about the Institute of Clinical and Economic Review and the broader question of where the group’s influence is centered and the elements of ICER reports that stakeholders are paying attention to. Do listen to …

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How does HTA for orphan drugs differ across europe?

New research looks at the factors that speed up and slow down HTA appraisals for rare disease medicines across Europe. Rare diseases drugs have always faced challenges when it comes to HTA approvals, even as governments bring in more regulatory policies that make their path through assessment easier. Several factors make it difficult for HTA …

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