Senator Scott Questions FDA Nominee on the Future of mRNA and CRISPR Technologies

WASHINGTON – Today, U.S. Senator Tim Scott (R-S.C.) questioned Dr. Robert Califf, President Biden’s nominee for Food and Drug Administration (FDA) Commissioner, on the future of mRNA and CRISPR technologies and their potential to treat and cure rare diseases like Sickle Cell Anemia.


Click to watch the senator’s questions

A full transcript is below.

Senator Scott: Thank you, Dr. Califf, for spending some time with us today and talking about some really important issues that we could all spend a day talking about. I only have five minutes, [which is] good news for you.

My first question is: You’re coming into a very important position in the midst of, really, headwinds in the public health space. And I appreciate the enormity of your responsibilities if you are confirmed to lead the FDA. How do you view the FDA’s role in granting Emergency Use Authorization (EUA) for the new, as well as repurposed, products to address the COVID-19 pandemic? Should the FDA use a wider latitude to ensure multiple avenues—for instance drugs like budesonide, as well as COVID-19 testing kids—making sure that they’re available to combat the pandemic?

Dr. Califf: Thanks senator, it’s good to be here with a fellow South Carolinian. … You’re well aware, and it was during my tenure that the EUA guidance was written that the potential benefits need to be weighed against the potential risks. That’s the authority the FDA is given.

So there’s a lot of latitude on the types of things that you mentioned, yet I would still point out we need to have enough evidence that we can make a fair assessment of what the potential is, and we need a system in this country, as I’ve emphasized over and over, that produces the evidence more quickly because under that framework, it’s possible you could get it wrong. It’s different if you’ve actually measured the benefits and risks and then you’re confident about it. And so it’s finding that right balance that’s really in play for the FDA.

Senator Scott: No doubt. I would suggest that, based on the severity of the challenge in the public space, that taking the appropriate calculated risk seems to be necessary nowadays more than it has been in the past, and I think we can do that in a way that involves coming to good decisions consistently.

On the long view of public health, I am excited about the role that the mRNA played in bringing the vaccine to the forefront and saving millions of lives. What’s the future of that technology for future needs in the vaccine arena, number one?

Number two, I think about the 7,000 rare blood diseases that have very few therapies, [or] treatments of any sort. And I focus a little bit more on CRISPR technologies for things like Sickle Cell Anemia and that path going forward. Do you see: A) more application for CRISPR in the rare blood disease space? And B) on the future forefront of the mRNAs and vaccines, is that future as bright as it appears it could be with the use of these technologies?

Dr. Califf: This is one of the things, senator, where the excitement of being part of this is what keeps me going and coming back. I mean, I’m old enough to remember when the Human Genome Project was just being developed, and now we have the code for the genome, and what you’re describing are the downstream consequences of that with reading the code and producing proteins and other substances that determine whether we’re sick or healthy. And there are a whole array of technologies, you know, specifically for mRNA. I think, as a platform for vaccines, this is something that we’ve only dreamed about, but it’s a reality now. And you know, this took a collaboration of FDA scientists and, of course, academia and the private sector all working towards a common goal over decades.

You also mentioned CRISPR, and the ability to change the fundamental genome is an amazing possibility but also has significant risks. And so, like everything else, we’ve got to come up with the ideas, try them out, and measure things and see what really works, but almost nothing could be more exciting than curing a rare genetic disease with a gene substitution, perhaps for life. And I think, for people with Sickle Cell Disease, I’ve done a lot of work … I think this is something that’s really needed, and it ties in. You know, chronic treatment for Sickle Cell Disease is not going so well in this country because many of the patients live off the beaten path of the big high-tech centers, and we’ve got to get treatments that are more effective.

Senator Scott: I think we should at least take a second and thank Dr. Francis Collins for his work on the project that really has produced amazing results for our nation and, frankly, for the world. I know he’s retiring or just retired. His work has been amazing for all of mankind, number one. Number two, I would suggest that, as we look into that future, the ethical issues around the new technologies like CRISPR will continue to pop up around the world. I know that China has had some challenges already with the use of CRISPR, and so what becomes a solution also becomes a problem. That’s called reality, and hopefully we will wrestle with those ethical issues in the public forum sometime in the very near future.