Four Unique Considerations of Gene and Cell Therapies
There are thousands of diseases with very few or no treatment options. Gene and cell therapies continue to be developed as a long-term treatment and even potential cure, by introducing new cells or genes to treat the disease.
With some therapies already approved and many products in the pipeline, the gene and cell space is a growing area of specialty pharmacy. Although these products have high potential for improving patient lives, especially those with limited treatment options today, there are unique considerations that must be discussed.
1. Gene and cell therapies carry some risks, known and unknown. The most common method to deliver a gene is through what is known as a viral vector. Due to their ability to infect cells, viruses are the most common vectors of choice in both approved treatments and those currently in the pipeline. Although viral vectors are altered to prevent illnesses, it is still possible to trigger an immune reaction. The intention of gene therapies is to achieve permanent or long-lasting disease improvement. Therefore, delayed adverse events are possible and these events have been documented during clinical trials. Long-term animal studies have shown that these adverse events can arise years later.1 To closely monitor for potential delayed effects, it is important for patients to be monitored for an extended timeframe after the therapy is administered.
2. Administration of gene and cell therapies can vary. Two delivery methods are available for gene and cell therapy – in vivo and ex vivo. In vivo therapy entails the direct administration of DNA into affected cells already inside a patient. In contrast, ex vivo involves the removal of cells from the host, genetically modifying them in a lab, and reintroducing them back into the patient. Donor cells can come from the patient (autologous) or from a non-patient donor (allogeneic). Allogeneic ex vivo therapy provides the benefit of treating more patients in a timelier manner with reduced cost; however, it may cause undesirable immune reactions due to the cells being foreign to the patient. Autologous ex vivo therapy carries a lower risk of immune reactions but must be done on a patient-by-patient basis.
3. Gene and cell therapies come with a large price tag. Like all medications, gene and cell products go through the research and development process. This process requires a large amount of money to develop and test the therapy. With such a significant expenditure in research for each disease state, combined with a limited number of patients per disease, a large price tag is often necessary for manufacturers to receive return on investment.2 Already approved gene and cell therapies average between $400,000 and $850,000, with one product priced just over $2 million dollars.3 Despite the high costs, manufacturers argue the upfront cost is worth the long-term savings in many cases.
4. The specialty pharmacy will play a large role in gene and cell therapy. Specialty pharmacies like AllianceRx Walgreens Pharmacy are experienced in the unique requirements of dispensing specialty medications. Many gene and cell therapies will need ongoing patient monitoring and reporting well after the therapy is administered. Specialty pharmacies monitor patients through clinical management programs. These programs can be utilized for ongoing clinical monitoring and reporting for gene and cell therapy, too. In addition to clinical monitoring, specialty pharmacies have experience with the purchasing, dispensing, and storage requirements of specialty medications. Since gene and cell therapies will likely have similar requirements, if not more complex, specialty pharmacies like AllianceRx Walgreens Pharmacy are in a position to relieve some burden from manufacturers.
Gene and cell therapies offer the potential for long-term treatment or potential cure of so many disease states with little or no current options. However, with these therapies, there are many aspects that must be considered. With many years of experience in these areas, AllianceRx Walgreens Pharmacy has the ability to care for these patients now and well into the future.
Researched by Adrienne Brennan, PharmD, CSP, and Marcie Morris, PharmD, CSP, clinical program managers at AllianceRx Walgreens Pharmacy.
Sources
1 Gene therapy needs a long-term approach. Nat Med 27, 563 (2021). Accessed July, 7, 2021 from doi.org/10.1038/s41591-021-01333-6
2 Datamonitor. Gene Therapy Commercialization – Opportunities and Barriers. Accessed April 24, 2020 from service.datamonitorhealthcare.com (subscription required)
3 ASH Clinical News. The High Price of Hemophilia. Accessed April 24, 2020 from ashclinicalnews.org/spotlight/feature-articles/high-price-hemophilia
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