Expert: Navigating Costs, Regulations, and Patient Care in Gene Therapy

Varshini Kanukollu, PharmD, BCOP, pediatric oncology clinical pharmacy specialist at Memorial Sloan Kettering, continued her conversation with Pharmacy Times®, discussing the significant financial and regulatory considerations pharmacists must address when managing gene therapy products, including high treatment costs, insurance approval, and institutional processes. She highlighted the need for staff training, pharmacy and therapeutics committee review, and awareness of evolving regulatory requirements such as the recent removal of REMS for certain CAR T-cell therapies. Kanukollu emphasized that while challenges remain, gene therapies represent a rapidly advancing field where pharmacists play a key role in ensuring safe, effective, and accessible patient care.

Pharmacy Times:
Beyond the clinical aspects, what are some financial or regulatory considerations for a pharmacist managing gene therapy products?

Varshini Kanukollu, PharmD, BCOP: As many people might know, a lot of gene therapy products come with a very high cost, which serves as a major barrier to accessing these therapies, especially for patients who do not have insurance coverage. These financial considerations are discussed upfront when identifying whether or not this is the right treatment choice for a patient and when deciding on treatment options.

Typically, the request is submitted to the patient's primary insurance, and they may require other supporting documents or objective data before providing approval. Once we obtain approval, that is our green flag to move forward with the process for that patient.

Beyond financial considerations, there are also operational and regulatory factors. For example, we have to consider staff education and training to ensure proper storage, preparation, and administration of the product. We also need to decide on the process for pharmacy and therapeutics committee approval, which may vary from institution to institution.

Additionally, some gene therapy products—historically, CAR T-cell products used to treat multiple myeloma and certain leukemias and lymphomas—had REMS requirements associated with them. This came with many regulatory and institutional requirements. Fortunately, the FDA removed these REMS requirements earlier this year, but it remains an important consideration as more of these gene therapy products are approved.

Overall, financial and regulatory processes and considerations vary between institutions and between different gene therapy products. I believe these aspects of gene therapy are a great place for pharmacists to get involved.

Pharmacy Times: Is there anything you would like to add?

Kanukollu: The realm of gene therapies is a rapidly evolving and complex field. Beyond understanding how these therapies work as a whole, there is so much to learn about each individual gene therapy product and the various disease states for which they are approved.

As I mentioned earlier, these include oncologic disease states, hematologic disease states, and other rare genetic conditions. Understanding where each one fits in, along with the clinical pearls for each product and disease state, is critical.

In some areas, such as sickle cell disease and transfusion-dependent beta thalassemia, gene therapies may finally offer a curative treatment option for patients who have been receiving chronic lifelong medications or transfusions and who do not have a matched sibling donor for hematopoietic stem cell transplant, which has historically been the curative option.

There are still many considerations with gene therapy, including appropriate patient selection, financial approval, and adequate patient and staff education. However, I am excited to see how the gene therapy space continues to evolve.

I also mentioned in my presentation that there are still many unknowns about the long-term data with these products. I hope that over the next several years, we will learn more about how these patients do in the long run.