Ajeet Gajra, MD, FACS, Chief Medical Officer at Cardinal Health Specialty Solutions speaks about the ASH 2021 Abstract – 4010 Barriers to Referral for Chimeric Antigen Receptor T Cell (CAR-T) Therapies Among U.S. Community Hematologists/Oncologists (cH/O).
Link to Abstract:
https://ash.confex.com/ash/2021/webprogram/Paper148165.html
Introduction:
Gene therapies, which include inserting, removing, or changing genetic information into a patient’s cells to treat a specific disease, are a big step forward in medicine, with the potential to cure or greatly improve the outcomes of a variety of benign and malignant hematologic disorders (American Society of Gene Therapy). For subgroups of people with spinal muscular atrophy or retinal dystrophy, gene therapies have been approved, and almost a thousand studies are actively enrolling patients for various gene therapy trials. Aside from CAR-T treatments, various gene therapies for hemophilia, hemoglobinopathies, and congenital immunodeficiency syndromes are under advanced clinical trials in the United States. Many of these trials’ findings were reported at the 2020 ASH annual meeting. While several gene therapy products are projected to receive FDA clearance in the near future, the complexity of treatment, provider knowledge gaps, or impediments to accessibility or cost may prevent these treatments from being integrated into ordinary clinical practice. As a result, the current study polled community oncologists/hematologists (cO/H) in the United States to assess their impressions of gene therapy utility and impediments to acceptance or integration into clinical practice.
Methods:
cO/H from around the United States were invited to participate in a web-based poll about gene treatments between February and April 2021. The poll also included questions about physician demographics and practice characteristics. The responses were tallied and descriptive statistics were used to examine them.
Results:
The poll was completed by 369 cO/H, with 36 percent identifying as a medical oncologist and 63 percent identifying as a hematologist/oncologist. On clinic days, cO/H had an average of 19 years of clinical experience, spent 86 percent of their time in direct patient care, and saw an average of 17 patients each day. Half of the cO/H said they were unaware of new gene therapy results for adult hematology/oncology indications (35 percent said they were “not very informed” and 15% said they were “not at all familiar”). When asked how many licensed gene therapy treatments (excluding CAR-T products) were available in the United States in early 2021, 27 percent said there were none, 24 percent said there were one, and 20 percent said there were two. In the next two years, 53 percent of cO/H predict gene therapy utilization for adult hematologic/oncologic indications to be primarily administered and managed by academic institutes to which they will send their patients; 27 percent expect indications to be limited and unlikely to effect their practice (Table 1). The main impediment to implementing gene treatments into clinical practice, according to cO/H, is cost; particularly, cost constraints by payers (49 percent), prohibitive cost to patients (46 percent), and prohibitive cost to practices/hospitals (46 percent) (37 percent ). Limited real-world efficacy data (18%) and long-term problems were other hurdles to adoption (13 percent ). Most cO/H said they would be comfortable prescribing a gene therapy for adult hematology/oncology indications if it were reimbursed (39%) or high (43%).
Conclusions:
Many cO/H were unaware of recent gene therapy product data, and more than half expected to direct their patients to large academic institutes for gene therapy. While most cO/H would be willing to prescribe gene treatments to their patients, the expense was seen as prohibitive. This information can help build the groundwork for gene therapy products in hematology for a variety of stakeholders, including patients, advocacy groups, pharmaceutical producers, payers, and professional associations. Future research should concentrate on improving community provider education on gene therapy products as well as establishing cost-cutting support programs.