Gene Editing Revolution: Economic Implications, Breakthrough Potential, and Regulatory Imperatives

Gene editing utilizing the CRISPR-Cas9 system modifies individual nucleotides to stop certain diseases or viruses. This offers cures to partially treatable conditions, significantly improving human quality of life. The lack of an appropriate comparative cost framework has resulted in tepid government and industry support, which hampers the ability to develop cures for deadly diseases. The American Cancer Society estimates 1.9 million new cancer cases in 2022 within the US (“Risk of Dying from Cancer Continues to Drop at an Accelerated Pace”, 2022), National Library of Medicine finds that about 1.2 million people in the US have HIV (Bosh et al., 2021), and the CDC reported that only 32% of kids from the age of 0-3 survive sickle cell disease (“Data & Statistics on Sickle Cell Disease”, 2022). To properly form conclusions about the effects of gene editing on society, investment in research and further evaluation of the socioeconomic perspectives must be considered.

Distorted Perception of Prices

Current cost assessments of gene therapy are not being compared on an equal scale relative to other treatments. The primary reason for this distortion is the one-time high cost of gene therapy, as opposed to diseases requiring longer-term medical care. For example, cancer is a deadly disease without a cure, accounting for nearly one in six deaths worldwide, hence “cancer therapy … and harmful toxic side effects continue to be barriers to survival time and quality of life” (Zhang et al., 2021, p. 1). Indirect socioeconomic costs associated with reduced productivity, lost opportunity costs, and decreased quality of life have not been appropriately considered in the overall cost of treating diseases like cancer. Similarly, Louis P Garrison Jr, who received a Ph.D. from Stanford University, noted that “challenges remain in creating a ‘level playing field’ across different disease-modifying or potentially curative innovations”. Applying the theory of second best, Garrison concludes that market price does not appropriately account for social opportunity cost and adjustments should be made. His proposal considers an adjustment “based on an appropriate variable cost-effectiveness threshold” to enable proper evaluation of the economic benefit of gene therapy versus lifelong cancer treatment. (Garrison Jr et al., 2021). This distortion of cost has led to the prevailing public perception as well as the hesitancy of regulatory approval for gene therapy because companies, insurers, and patients are worried about the cost-effectiveness of the treatment.

Additionally, gene therapy for extremely rare diseases has sensationalized the perception of costs. For example, Glybera, a gene therapy treatment available to fewer than ten patients, lingers at a price of $1 million, and Yescarta, which is available to about 7,500 patients, costs around $373,000. “If the targeted disease is more common, ‘there will be more pricing pressure,’ Sanchez says”(Mullin, 2017). This correlation between the size of the patient population and the price of therapy suggests that as gene therapy targets more common diseases, prices will be forced down, resulting in greater public accessibility.

Success in Patients Reduced the Economic Burden

Gene therapy has been proven to be successful in patients with uncurable lifelong diseases, reducing their economic burden. For example, sickle cell is an inherited disorder resulting from a change in a gene that causes hemoglobin production (“Sickle cell anemia”, 2022). A study done by Blood Advances, a scientific journal part of the American Society of Hematology found that insured people with sickle cell disease accumulate about $1.7 million in disease-related medical expenses over their lifetime (Sampson, 2022). This economic cost brings up the question of whether or not gene therapy could be a more cost-efficient solution for the disease. Gene therapy would be a one-time cost that completely eliminates the need for multiple expensive treatments throughout your lifetime.

Not only is it cost-efficient, but gene therapy has also been proven to be an effective treatment. A study done by the New England Journal of Medicine has found gene therapy to be successful in multiple patients. Patient 2 was a 33-year-old female with sickle cell disease who had on average seven severe vaso-occlusive episodes per year, “3.5 SCD-related hospitalizations and five RBC transfusions per year.” After treatment, the results showed that the CRISPR-Cas9 system was successful in editing the intended genes resulting in “high levels of fetal hemoglobin expression, and the elimination of vaso-occlusive episodes or need for transfusion” (Frangoul et al., 2021). Gene therapy helped improve the quality of life of these patients as they no longer experience the deadly effects of sickle cell disease.

However, the above study does have limitations and will require the completion of a “fuller analysis” to assess any long-term effects of this treatment, along with adjustments to lessen the short-term effects. Some short-term symptoms that Patient 2 experienced were “sepsis in the presence of neutropenia, cholelithiasis, and abdominal pain. (Frangoul et al., 2021). All 3 adverse events resolved with treatment.”, indicating that despite some side effects, the researchers found the side effects to be treatable. Further, the report suggests that “ the preliminary results reported here support further experimental testing of CRISPR-Cas9 gene-editing approaches to treat genetic diseases.” The New England Journal of Medicine argues that scientists have made a great leap in the advancements of gene therapy although side effects have been present (Frangoul et al., 2021). As the study of gene therapy increases, regulations loosen, and more companies undertake trials, gene therapy will increase in efficacy and become a more reliable treatment. The wider use and availability to the public will increase competition in the market and result in downward pricing pressure.

Need for Change

Current treatments for high-risk conditions are not truly effective because they do not completely “cure” the condition. HIV is a virus that has a treatment, but no cure. Retroviral therapy prevents the replication of HIV and makes sure that it does not permeate the body. Although this seems like an effective treatment, it requires patients to take medication for the rest of their lives.

“The estimated total direct expenditure for HIV/AIDS care and treatment between 2002 and 2011 was $10.7 billion annually.” The study estimates that the discounted lifetime medical costs for an individual who acquires HIV at 35 years of age is $326,500 ($597,300 undiscounted), with 60% of the costs attributable to ART.9 " This is because HIV medication has more than doubled since 2010 and is in the top five therapeutic non-discounted spending classes (“Guidelines for the Use of Antiretroviral Agents in Adults and Adolescents with HIV”, 2022).

Early gene editing research for HIV treatment using the CRISPR Cas9 system had been found to be successful, with scientists at Temple University and the University of Nebraska Medical Center, removing “the virus completely from mice during preclinical testing using a combination of CRISPR and antiretroviral therapy" (Armstrong, 2021). Although this was a test on mice, it provides an optimistic view of future gene therapy. By completely removing the virus, patients no longer have a lifetime dependency on the medication, which will not only decrease their expenses but also improve their quality of life.

In conclusion, gene therapy may be an economically viable option for curing life-threatening conditions like cancer, HIV, or sickle cell disease in the near future. Price distortion has led to incomplete analysis, therefore hesitancy of medical companies, insurance companies, and the government to support its development. With a more comprehensive cost assessment methodology, the comparative cost of gene editing which incorporates socioeconomic implications may provide an investment framework that better directs research to cure diseases. It has been proven that conditions like cancer, HIV, and SCD have had long-lasting impacts on patients both socially and economically as it reduces their quality of life and requires lifetime medical-related expenses. By ensuring that appropriate investment and regulatory support are directed into gene therapy research, additional testing to assess long-term effects can be conducted, therefore making gene editing a medical option available to patients.

References:

Armstrong, A. (2021, September 17). Excision's CRISPR gene editing therapy for HIV is heading into human testing after FDA clearance. Fierce Biotech. Retrieved December 11, 2022, from https://www.fiercebiotech.com/biotech/excision-hiv-crispr-gene-editing-therapy-cleared-for-human-studies-by-fda

Bosh, K. A., Hall, H. I., Eastham, L. C., Daskalakis, D., & Mermin, J. (2021, June 4). Estimated Annual Number of HIV Infections ─ United States, 1981–2019. Morbidity and Mortality Weekly Report. https://www.cdc.gov/mmwr/volumes/70/wr/pdfs/mm7022a1-H.pdf

Cost considerations and antiretroviral therapy: NIH. Cost Considerations and Antiretroviral Therapy | NIH. (2022, September 21). Retrieved December 11, 2022, from https://clinicalinfo.hiv.gov/en/guidelines/hiv-clinical-guidelines-adult-and-adolescent-arv/cost-considerations-and-antiretroviral

Data & Statistics on Sickle Cell Disease | CDC. (2022, May 2). Centers for Disease Control and Prevention. https://www.cdc.gov/ncbddd/sicklecell/data.html

Frangoul, H., Al., E., Author AffiliationsFrom the Sarah Cannon Center for Blood Cancer at the Children’s Hospital at TriStar Centennial, Malech, H. L., Walters, M. C., Others, E. B. E. and, Others, M. J. D. and, Others, A. C. and, & Others, M. W. R. and. (2021, January 21).

CRISPR-Cas9 gene editing for sickle cell disease and β-thalassemia: Nejm. New England Journal of Medicine. Retrieved December 11, 2022, from https://www.nejm.org/doi/full/10.1056/NEJMoa2031054

Louis, G. P. (n.d.). Gene therapy may not be as expensive as people think: Challenges in assessing the value of single and short-term therapies. Journal of Managed Care & Specialty Pharmacy. Retrieved December 11, 2022, from https://www.jmcp.org/doi/full/10.18553/jmcp.2021.27.5.674?role=tab

Mullin, E. (2020, April 2). Tracking the Cost of Gene Therapy. MIT Technology Review. https://www.technologyreview.com/2017/10/24/148183/tracking-the-cost-of-gene-therapy/

Risk of Dying from Cancer Continues to Drop at an Accelerated Pace. (2022, January 12). American Cancer Society. https://www.cancer.org/latest-news/facts-and-figures-2022.html

Sickle cell anemia - Symptoms and causes - Mayo Clinic. (2022, March 9). Mayo Clinic. https://www.mayoclinic.org/diseases-conditions/sickle-cell-anemia/symptoms-causes/syc-20355876

The Cost of Living with Sickle Cell Disease - Hematology.org. (n.d.). https://www.hematology.org/newsroom/press-releases/2022/the-cost-of-living-with-sickle-cell-disease

Zhang, H., Qin, C., An, C., Zheng, X., Wen, S., Chen, W., Liu, X., Lv, Z., Yang, P., Xu, W., Gao, W., & Wu, Y. (2021, October 1). Application of the CRISPR/cas9-based gene editing technique in basic research, diagnosis, and therapy of cancer - molecular cancer. SpringerLink. Retrieved December 11, 2022, from https://link.springer.com/article/10.1186/s12943-021-01431-6My post content