Ideally, researchers determining the clinical and economic effects of a new treatment would have both short-term and long-term data, explained Steve Pearson, MD, MSc, president of the Institute for Clinical and Economic Review. If not all of this data is available, however, they may have to use surrogate outcomes or perform indirect comparisons.
Ideally, researchers determining the clinical and economic effects of a new treatment would have both short-term and long-term data, explained Steve Pearson, MD, MSc, president of the Institute for Clinical and Economic Review. If not all of this data is available, however, they may have to use surrogate outcomes or perform indirect comparisons.
Transcript (slightly modified)
What forms of evidence are ideal to balance long-term and short-term pros and cons of a new therapy?
In a sense, the ideal evidence that we would have would allow us to understand well what the short-term clinical benefits are and be able to fairly accurately project those into the future. That’s always going to involve some degree of uncertainty, though, and everybody’s aware of that.
We have to try to understand that we are looking at the shorter-term effects from the clinical trials and we want to make sure that we don’t overly focus on what we know about the short-term, because in many cases drugs will really show their greater value in the long-term by improving patient outcomes or preventing adverse events years and years downstream, and that has both clinical effects as well as economic effects.
Good data to us allows us to understand very clearly which patients are being treated in the short-term and how that generalizes to broader patient populations. It allows us to understand very clearly, even if we have to do an indirect comparison, how different treatment options line up. Then, hopefully, we have some longer-term data to show that the clinical effects and the economic effects continue through time, so that we can build a model that has fewer assumptions about long-term effects, and more data based on real studies.
Standard Criteria for Loss of Ambulation Needed in DMD
April 19th 2024A recent study suggests the differences between ambulation definitions for patients with Duchenne muscular dystrophy (DMD) can impact the identification of ambulant vs nonambulant individuals, and standard criteria across settings are needed.
Read More
Early Involvement Critical in Treating Immunotherapy-Induced Overlap Syndrome
April 19th 2024A series of case studies reveals the importance of early diagnosis and involvement of special teams of clinicians when dealing with potential cases of overlap syndrome, which encompasses myocarditis, myasthenia gravis, and immune checkpoint inhibitor–related myositis.
Read More
Government agencies have created an online portal for the public to report potential anticompetitive practices in health care; there are changes coming to the “boxed warning” section for chimeric antigen receptor T-cell therapies (CAR T) to highlight T-cell blood cancer risk; questions about the safety of obesity medications during pregnancy have arisen in women on them who previously struggled with fertility issues.
Read More