Unbiased Analysis of Today's Healthcare Issues
The year 2022 was a good, but perhaps not great year for innovation. According to the FDA’s Center for Drug Evaluation and Research’s (CDER) New Drug Therapy Approvals 2022 report, there were 37 novel drugs approved in 2022. This number is down from the historical rate of approvals between 2013-2021 (43.4 approvals per year) and…
Part of the Affordable Care Act mandated a medical loss ratio (MLR) for health plans in the individual and small group markets. What this means is that if the MLR is 80% and insurers charge $100 in premiums, plan enrollees must receive $80 in services on average. If they do not (say they only receive…
That is the title of an FTI White Paper published this year where I was a co-author alongside Antoine Mialhe, Sabiha Quddus, Anne-Sophie Deman, Suhail Thahir and Agnes Sipiczki. The role of Intellectual property (IP) in the development of COVID-19 vaccines and therapeutics became a central tenant in discussions on how to ensure access to…
That is the quote from Iga Lipska, MD, PhD, MPH from the Medical University of Gdańsk, Clinical Trials Center in Warsaw, Poland. She was one the panelists at the ISPOR Europe Plenary speech. There were a lot of good sessions, many of which I will be posting on throughout the week. The first plenary was…
As part of The 21st Century Cures Act, FDA is required to consider additional mechanisms to expedite access to new drugs, including those currently under investigation. In fact, the 21st Century Cure Act mandates that pharmaceutical manufacturers have to post their policies on expanded access (a.k.a. “compassionate use”) for investigational drugs by: (1) the first…
It is well known that excessive use of alcohol causes significant health impacts. CDC notes that Excessive alcohol use led to more than 140,000 deaths and 3.6 million years of potential life lost (YPLL) each year in the United States from 2015 – 2019, shortening the lives of those who died by an average of…
Today, the Food and Drug Administration (FDA) approved the drug Relyvrio (sodium phenylbutyrate/taurursodiol; also known as AMX0035), which is indicated for the treatment of patients with amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig’s disease. Most people with ALS die within 2-5 years of diagnosis. In justifying the approval, FDA’s press release stated: “This…
FDA has two competing priorities. On the one hand, they want to collect as much data as possible to insure drugs are safe and effective; on the other hand, patients want access to innovative medicines as quickly as possible. To try to balance these priorities, FDA often will approve treatments based on evidence from surrogate…
FDA wants drugs to be more targeted to factors that impact patient’s lives in ways that they care about. To achieve this goal, last month FDA released a third guidance document on patient focused drug development generally applicable to a variety of clinical outcome assessments (COAs), including patient-reported outcome (PRO), observer-reported outcome (ObsRO), clinician-reported outcome…
When new drugs are approved, FDA limits their on-label use to the treatment of specific diseases. A key question is whether the definition of a disease should be narrower or broader. Make the disease definition too broad, then there may not be strong evidence from the clinical trial to support its use among these types…