Fifty years ago, scientists Theodore Friedmann and Richard Roblin supported the development of gene therapy techniques but noted significant challenges in developing gene therapies. In the subsequent five decades, important advances have occurred in gene and cell therapies — along with some setbacks.
Today, cell and gene therapies are entering the market to treat a number of conditions, including rare diseases. Research in cell and gene therapies is leading to specialty pharmacy trends, indicating that options available to patients will continue to expand in the coming years.
Understanding the current state of cell and gene therapies through real world evidence analytics can help specialty pharmacies position themselves for rare and orphan drug success as these therapies increase their prevalence in patient treatment.
Current Practices in Cell and Gene Therapies
The first gene therapy — Glybera, a treatment for lipoprotein lipase deficiency — became available to European patients in 2012. Since then, the number of cell and gene therapies launched has increased every year, “with over 50% of launches having occurred in the last two years alone,” write Tobias Handschuh, Marie-Lyn Hecht, and Allegra Hohn at Oliver Wyman, a management consulting firm. Market growth for new cell and gene therapies has expanded 153 percent between 2017 and 2021.
Today, several new cell and gene therapies are appearing. Many are driven by the study and use of viruses to introduce new genes into existing cells.
For example, “there’s been a huge explosion in gene therapy and the eye,” says Christine Kay, a surgeon at Vitreoretinal Associates in Gainesville, Florida. The eye’s limited immune responses make it easier to introduce gene therapies into treatment of eye and vision disorders.
Another area attracting significant study is the use of chimeric antigen receptor (CAR) T-cell therapies. CAR T-cell therapy is a form of immunotherapy that alters immune system cells to incite these cells to attack specific targets, such as the metabolic changes found only in cancer cells, according to the National Cancer Institute.
Since 2017, six CAR T-cell therapies for various forms of cancer have become available on the market. They include treatments for cancers like leukemias and lymphomas. “[CAR T cells] are now widely available in the United States and other countries and have become a standard treatment for patients with aggressive lymphomas,” says Steven Rosenberg, chief of the Surgery Branch in the NCI Center for Cancer Research (CCR).
The significant price tags on rare and orphan drug and cell and gene therapies make their use daunting for some patients. The focus of cell and gene therapies on rare diseases has also limited the number of patients who need — and thus create a market for — these treatments. As treatment options expand, however, markets are likely to expand as well. The number of patients who rely on their specialty pharmacies to fill prescriptions, provide information and help them navigate payment will increase.
Real World Evidence Analytics in Cell and Gene Therapies: The Next Five Years
Real world evidence analytics and research on cell and gene therapies continues to produce useful therapies. This research also reveals links between various conditions, unlocking new potential avenues of treatment for various diseases.
For example, Audrey Kapelanski-Lamoureux and fellow researchers discovered a link between hemophilia and the development of liver cancer, they write in a 2022 article in Molecular Therapy. The common factor lies in misfolded proteins in the liver cells. Since protein replacement therapy is currently a standard treatment for hmophilia A, understanding how proteins misfold may help researchers develop better treatments for both hemophilia A and certain forms of liver cancer.
Protein replacement therapy is not the only cell or gene therapy producing valuable results in research. Another option, CAR T-cell therapy, is already showing promise for treating some cancers, and is also under study for its potential value in treating other conditions.
In a December 2022 study published in Science, Kyle G. Daniels and fellow researchers created a “library” of CAR signals that could be used to generate various types of T cells. Using machine learning, the library determines which signals to send to cells based on various inputs. While the technology is still in its infancy, it shows promise in allowing for treatment of individual gene-related diseases with specific, targeted treatments.
Combining AI with cell and gene therapy research, as in the CAR machine learning study, may further drive cell and gene therapy development, write Mayank Bhandari and fellow authors in the Life Science practice at McKinsey.
Another study published in Science Immunology by Jaeu Yi and fellow researchers focuses on the use of CAR T-cells to treat an autoimmune condition in mice similar to multiple sclerosis (MS) in humans. The study found that CAR T-cells could be modified to focus only on the immune system components that were misfiring and causing autoimmune symptoms.
Currently, many treatments for autoimmune conditions focus on suppressing immune system activity as a whole. While this approach can alleviate symptoms of the autoimmune disorder, it also exposes patients to a higher risk of contracting other illnesses — and of being unable to fight off those illnesses when they appear. CAR T-cell treatments that allow the immune system to function normally by weeding out only malfunctioning cells could represent a significant breakthrough for patients.
These and similar studies indicate a promising future for cell and gene therapies. Within the next five to ten years, specialty pharmacies will inevitably find themselves managing many more cell and gene therapy prescriptions.
Impacts of Expanding Cell and Gene Therapies on Specialty Pharma Trends
As cell and gene therapies become more widely available, the number of patients prescribed these treatments is likely to increase.
For specialty pharmacies, as for others in the cell and gene therapy chain, cell and gene therapies call for an organized strategy that creates “a clear pathway and focus to ensure that tasks and projects are aligned,” writes Dea Belazi, president and CEO at AscellaHealth.
Cell and gene therapies typically take place at a hospital or treatment center specified in a patient’s treatment plan. Thus, they differ from many medications that specialty pharmacies dispense directly to the patient. A specialty pharmacy’s approach to these treatments must differ as well.
Specialty pharmacies handling cell and gene therapies need to coordinate with patient services teams to create the “clear pathway and focus” Belazi calls for. Coordination on items like treatment delivery, distribution schedules, and transportation and logistics can ensure that patient services, pharmacies, and distributors stay on the same page with patient and provider needs.
When specialty pharmacies provide clear pathways to distribution and communication, they also provide patients with pathways to access treatments that are rarely ordered and often prohibitively expensive, write Sarabvijay Singh and Tobias Handschuh at Oliver Wyman.
Specialty pharmacies can support patient journeys in several ways. One foundational approach is the use of Hub technology to connect everyone involved in cell and gene therapy treatment — patients, providers, payers, pharmacies and pharmaceutical manufacturers and distributors.
From a single digital source, specialty pharmacies can align tasks and projects, such as:
- Managing incoming prescriptions.
- Educating patients and providers on treatment options, methods and risks.
- Coordinating with payers to ensure authorizations are handled and cost coverage is available.
- Communicating with manufacturers and distributors for efficient, timely delivery, particularly when a cell or gene therapy treatment requires refrigeration or other specialized handling during transport.
Cell and gene therapies are likely to continue emerging to treat rare diseases and other conditions. In the coming years, specialty pharmacies can expect to also see orphan drug treatments become available for a wider range of conditions and to see different treatment options arise for individual conditions.
By understanding real-world evidence analytics and the trends in cell and gene therapy, specialty pharmacies can position themselves to help patients navigate their options, collaborate with providers, secure support from payers, and better manage distribution. Integrating an all-in-one Hub approach can bolster these efforts.
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