Considerations for Gene Therapy Logistics and Implementation

Omer A. Abdul Hamid, MD

Credit: Linkedin

As gene therapies enter the clinical space and become more common, centers and hospitals seeking to administer these therapies must be educated and equipped for safely treating and following up patients that would receive these therapies. Centers experienced with gene therapy logistics can also help by sharing their learnings and strategies to others looking to expand into the space.

Omer A. Abdul Hamid, MD, pediatric neurologist, Nemours Children’s Health, gave a talk on gene therapy logistics at the 2024 Muscular Dystrophy Association (MDA) Clinical and Scientific Conference, held March 3-6, in Orlando, Florida. CGTLive® spoke with Hamid to learn more about the logistics to consider with gene therapy and working to implement their use.

How can working to implement gene therapy use be a collaborative project?

Omer A. Abdul Hamid, MD: I think we're all trying to learn from each other's experiences and share each other's experiences so that we're not making similar mistakes, and we don't have to reinvent the wheel. I think we've all put in a lot of work to establish an infrastructure and build that infrastructure within our respective centers to try to establish a gene therapy center and offer this treatment to our patients. So, I think it's great that we're learning from each other and using that model.

I feel like we're at this precipice. And we're lucky as neuromuscular neurologists that we get to pave the way for other gene therapies, not just within our space, but within other areas in neurology and others in medicine. So, I think we're going to have a lot to teach others and a lot to learn from each other as well.

What are some common challenges with gene therapy logistics?

Hamid: Firstly, cost. These therapies run upwards of millions of dollars, so it's not something that's easily approved by payers. So having the right team that can authorize and put together the package for submitting it to the insurance companies is really important. Another challenge that's faced by our centers is getting buy-in from everybody in the hospital who would be tangentially involved in the care of these patients. So, making sure that the cardiologist, the pulmonologist, the hepatologist, the immunologist, are all on board and understand what gene entails and the adverse events (AEs) that can come from it, and how to adequately respond to those should they arise.

The actual day of dosing for gene therapy can be kind of an uneventful day, but it's a long day, and it's a very meaningful day for the patients. It’s a day that they'll remember forever and it's important to make sure that it goes smoothly. Making sure that the [pediatric patients] have toys and is cared for, and things like that are really important.

Concerning the day of, there are the complexities of scheduling patients, certain days, where to schedule them, where it's best to infuse these patients - outpatient or inpatient. So, after receiving systemic gene therapy, some providers would want them to stay close by in case there is an AE and they need to get to a center that can treat them quickly. Other providers understand that there's a wide range of socio-economic barriers that prevent people from staying nearby to a large center. So, we need to come up with techniques and ways to adequately treat all patients in an equitable way.

We also want to limit exposure to any external viruses while the patient is in that acute phase after getting gene therapy, we want to make sure that they don't get sick, because that's when they run into problems and start having these AEs. So oftentimes, that includes separating them from other siblings, separating them from their school. So that's challenging for a lot of our families.

Do you have any advice for centers trying to implement gene therapy treatments?

Hamid: The advice that I would give to center that are considering offering this treatment would be to put in a lot of homework and take your time. It can take up to 2, 3 years before you're fully ready, before you have all the infrastructure there. It's better to be overly prepared than underprepared. Because if an AE happens, and you're not prepared, it can have very serious consequences, not just for the patient, but everybody involved in the whole space in general.

This transcript has been edited for clarity.

REFERENCE

Gene Transfer Therapy: Dosing Day Logistics. Presented at: 2024 MDA Clinical and Scientific Conference; March 3-6; Orlando, FL.