FDA Brass Seems to Suggest US-Only Clinical Trials: Is it Practical or Fair?
(Thursday, January 12, 2023)
At the JPM conference this week, the FDA commissioner stated that he is “100% opposed to offshoring” of clinical trials, basically against sponsors going to other countries for cost savings. This is perhaps one of most myopic views expressed by someone so senior at FDA for many reasons. To be fair, I was not at the conference and so this quote from the Commissioner came as second-hand information that may have taken his statements out of context, but it is very important to dispel some common misperceptions for why sponsors go overseas to conduct clinical trials and why this statement, if true, represents a major misunderstanding of clinical trial off-shoring. The most important reason a sponsor based in the US considers going abroad for their clinical trial is lack of patient populations and investigators for their clinical trials. According to American Medical Association, less than 3% of physicians in the US participate in clinical trials as investigators of which majority belong to a select few large academic centers. Hence, clinical trials are not accessible to most eligible patients. Also, most of these centers do not serve medically underserved populations and nonwhite populations leading to lack of diversity in US-based clinical trials. According to the National Cancer Institute, less than 9% of eligible patients in the US participate in clinical trial and a whopping 41% patients either do not know or not know much about clinical trials. With this double whammy of lack of both patients and investigators, about 23% of cancer trial terminate early for lack of recruitment. To say that sponsor go abroad primarily for cost savings is a misleading. The cost of high-quality clinical trials, particularly for indications such as cancer, is not much different from that in the US even in developing and poor countries when you add the additional cost for regulatory approvals, training, quality assurance, foreign project management, shipping, clinical trial insurance, testing, travel, logistics, and several other tasks needed to manage a non-US trial for a US-based sponsor. For foreign sponsors, doing trials in their home countries may have lower costs but they could have additional costs for US regulatory processes. Going to another country to complete a clinical trial is not a simple financial question. Global clinical trials also help by expediting clinical trial completion, address the deficit in diverse demographic distribution from the US trial sites, create global footprint for a new drug that may be marketed globally, facilitate global health equity, and other advantages. The FDA should be the last party to make a politicized, pseudo-nationalist, short-sited broad statement about clinical trial off-shoring. FDA’s regulations require specific scientific and quality criteria for the clinical data submitted to it in all applications. There are systems in place for FDA to evaluate the merits of clinical data submitted to it for quality and relevance to US populations. The issue of non-US trials came to prominence recently when a few Chinese companies tried to get drug approvals based on China-only clinical data. Those criticisms were fair, but the FDA commissioner’s recent statement is not, particularly as it was presented in the media. Global clinical trials are an important part of clinical development and FDA cannot reject data just because it comes from non-US sites. Any delay in getting treatments to patients is directly responsible for their misery. The FDA needs to do all it can to expedite treatments, not make policies not grounded in science and practical clinical development. The commissioner misspoke.
Dr. Mukesh Kumar
Founder & CEO, FDAMap
Linkedin: Mukesh Kumar, PhD, RAC