Synthetic control arms (SCAs) are best suited for situations when a single arm trial is run in a patient population which is molecularly defined, allowing for a clearly defined historical or real-world control group to be created. The desire to speed up and lower the cost of drug development, coupled with increased availability of rich real-world data, have contributed to the increased openness towards using SCAs as supplementary evidence to accompany regulatory submissions using single arm trial data only. For instance, in oncology, where enrichment and stratification strategies had resulted in nearly 24% of oncology trials resembling rare disease studies, SCA has led to the expedited approval of several new therapies. But is their successful application limited to Oncology?
In the final part of this blog series, we talk about the incorrect notion that SCA is utilized only in Oncology.
Myth 4: Synthetic controls only work in oncology
A substantial proportion of synthetic control applications for regulatory purposes are in the oncology space. There are some advantages that are seen in using SCA in oncology trials in relation to some of the other therapeutic areas. In many oncology trials, outcome heterogeneity is (typically) smaller, diagnosis is often to an international agreed standard and data vendors have invested heavily.
However, it does not necessarily mean that the application of these methodologies is only seen in the oncology space. Many applications of SCA can be thrown under the broad umbrella of rare disease. Rare disease is highly represented, owing to operational challenges in conduct for these trials. Getting a control group is often difficult and randomizing to control is next to impossible because the disease is rare, and it is challenging to get patients. And when you break down what therapeutic areas these rare diseases overlap with, you will observe a reasonable representation in non-oncology applications.
In non-oncology indications, single-arm trials are often used in combination with natural history studies. Using established knowledge of natural disease progression, Novartis was able to obtain approval for Zolgensma (gene therapy for spinal muscular atrophy) after enrolling only 22 patients.
In conclusion, there is more to synthetic controls than oncology!
Watch the complete webinar to know more about the other myths surrounding synthetic control arms and learn how to overcome some of the challenges in using these methodologies. Read Part 1. Read Part 2. Read Part 3.
About the Author of Blog:
Mansha Sachdev specializes in content creation and knowledge management. She holds an MBA degree and has 11 years of experience in handling various facets of marketing, across industries. At Cytel, Mansha is a Content Marketing Manager and is responsible for producing informative content that is related to the pharmaceutical and medical devices industries.