Unbiased Analysis of Today's Healthcare Issues
Check out an event this Friday, December 10, at 10am EST/7am PST on at The Hill on Rare Diseases: A Conversation on Value Assessments. A summary of the topic and the speaker list are below. One in 10 Americans suffers from a rare disease—that’s 30 million Americans who need access to treatment. According o the NIH,…
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…
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.…
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…
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…
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…
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…
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…
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…
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).…