Survey Assesses Community Oncologists/Hematologists’ Perceptions of Gene Therapy for Hematological Disorders

Ajeet Gajra, MD, FACS, Cardinal Health Specialty Solutions, Dublin, OH, discusses a survey which analyzed the perceptions of community oncologists/hematologists regarding gene therapies and barriers to adoption or integration into clinical practice.

The results of this survey were presented at the 2021 ASH Annual Meeting.

Transcript: 

My name is Ajeet Gajra, and I serve as the Chief Medical Officer at Cardinal Health Specialty Solutions. Here, I would like to discuss our study presented at ASH 2021 titled "US-Based Community Oncologists/Hematologists' Perceptions and Future Use of Gene Therapies for Patients with Hematological Disorders."

Gene therapy refers to the strategy for curing or treating disease by modifying, inactivating, or supplying genes or gene products that may characterize a given disease by their presence or absence. Due to the complexity of the gene therapy process, the cost of therapy, and barriers to access, as well as gaps in knowledge, the integration of gene therapy may be limited.

I want to clarify that, while we consider CAR T-cell agents as also certainly a form of gene therapy, our interest in assessing gene therapy knowledge and perception was focused on gene replacement therapies.

In that vein, when we reached out to community-based hematologists/oncologists, we were specific in our ask that we want to understand from them what their perceptions of gene replacement therapy are.

We used a web-based survey between February and April 2021, so right at the heels of ASH 2020, and reached out to almost 400 hematologists/oncologists who were largely community based and asked them a set of questions regarding gene therapy.

The objective was to identify and evaluate their perceptions of the utility of gene therapies and the barriers to adoption or integration into clinical practice. Notably, at that time, only two gene replacement therapies had been FDA approved, and those were in non-hematology areas. We had voretigene neparvovec, which is Luxturna, for a rare form of retinal dystrophy, and then we had onasemnogene abeparvovec, or Zolgensma, for a type of spinal muscular atrophy.

We surveyed 369 hematologists/oncologists. They were community based. They were well experienced and had broad geographic representation. We started with simple questions regarding how aware they were regarding recent gene therapy studies and FDA-approved gene therapy products.

Notably, there seemed to be significant knowledge deficit, or a knowledge gap, where only 3% said they were very aware, and 13% said they were moderately aware. The vast majority were not very aware or not at all aware at over 70%.

We also asked them, to the best of their knowledge, how many gene therapy products were currently commercially available in the US, and those answers were also not accurate because the expected answer was two, but 80% of them did not get that right, so again highlighting that there was need for perhaps greater education.

We also asked them their perceptions regarding what indications may be the first to have a gene therapy-approved product in the United States, and 70% of them chose sickle cell disease; 61% chose hemophilia A; 39% chose hemophilia B.

While time will tell what product indication first crosses the finish line, at least as of ASH 2020, hemophilia B seemed to be the front runner. We certainly have had updated data at ASH 2021 in this regard.

Again, we should highlight that all of these participants were adult hematologists/oncologists. We did not include pediatric hem-oncs [hematologist/oncologists] in this survey. In terms of their perceived barriers, the top three were all cost-related.

Almost half of them thought that cost will limit authorization by payers, and 47% thought cost would be prohibitive to patients, whereas another 37% thought that cost will be prohibitive to practices and hospitals.

Almost a third felt like clinical eligibility will be very narrow, excluding most patients, and about 18% thought that limited efficacy data available in the real-world setting would be a barrier.

In terms of their expectation—this is now in early 2021—the majority felt that gene therapy will be administered and managed by academic centers—once there is an approved product, then they will refer their patients to the academic center—whereas a quarter, almost 27%, felt that new approvals and indications will be very limited and unlikely to impact their practice in any significant way.

However, when asked if there was a gene therapy product approved in an adult hematology indication and was reimbursable, what their level of comfort would be to prescribe it to eligible patients, the vast majority, 83%, had high level of interest.

In conclusion, our descriptive survey-based study demonstrated that the major barriers which are perceived by clinicians when considering gene therapy include prohibitive cost for patients, practices and payers, insufficient education of gene therapy products, and a concern for narrow clinical patient eligibility.

We also found that there were serious knowledge gaps surrounding gene therapy. These findings can inform future work to focus on enhancing education of gene therapy products among community providers as well as identifying support programs to decrease the burden of cost and developing accessible gene therapy options.

Within community practices, it is notable that over half the patients in the United States with hematology/oncology indications are treated within community practices. Thank you for listening and for this opportunity.