Gene Therapy: Proper Care for This Life-Saving Innovation
Of all the innovations we’re seeing today in healthcare, none is getting more attention than gene therapies – for good reason.
Gene therapies introduce genetic material into a person’s DNA to “edit” and replace faulty or missing genetic material that leads to disease. Unlike nearly all other medications that are repeatedly taken over time, these therapies typically are administered once with the intent to provide long-term benefit, and in some cases, a cure.
Express Scripts is focused on providing appropriate clinical and financial management for gene therapies and other curative and transformative therapies coming to market. In fact, when LuxturnaTM, the first in vivo gene therapy approved in the U.S., came to market earlier this year, Accredo specialty pharmacy and CuraScript SD specialty distribution were selected to exclusively handle the dispensing and distribution services due to the specialized handling required.
How Gene Therapy Works
The term “gene therapy” is used throughout the industry as an umbrella term for varying types of cellular and genetic modification approaches – all of which are aimed at altering or improving the function of genes that cause inherited medical conditions. Gene-replacement therapy adds new or corrected DNA to replace missing or broken genes, while CAR-T therapy, now on the market, is a type of treatment in which a patient’s T cells (a type of immune cell) are changed in the laboratory so they will bind to cancer cells and kill them.
Gene therapy differs from other targeted therapies in that treatment is intended to provide long-term benefit at a defined level. To justify clinical cost and risk, conditions appropriate for gene therapy usually have high rates of mortality or progressive co-morbidities, such as permanent organ damage and severe disability. They may also require complex and expensive medical treatment and other interventions. Though these unique, life-changing treatments offer substantial improvements in patients’ health, gene therapy may not be completely “curative.” For example, existing organ damage or disability is not always reversible.
Pioneers in Patient Access to Gene Therapies
Gene therapies can be life-changing, highly effective treatments for conditions like cancer, bleeding disorders and other rare diseases which have few or no treatment options available today.
Express Scripts understands that these therapies require payment and patient care systems which are as novel as the medications themselves. Our proven clinical and supply chain models give us a leading position in pioneering solutions, the ability to exclusively manage unique therapies, and the experience in caring for patients who take them.
Accredo has the protocols and expertise in place through our Therapeutic Resource Centers (TRCs) to effectively support patients, physicians, and payers as healthcare continues to evolve. Specialist pharmacists, nurses, and other clinicians in our condition-specific TRCs are best able to help physicians and patients navigate medication regimen, side effects, and barriers to access in a time-sensitive manner.
In addition, as the first and only distributor of the first gene replacement therapy, CuraScript SD offers a unique model to satisfy the high-cost, complex needs that go along with them.
The Need for Customized Care
Because of the complexities and therapeutic nuances specific to genetic conditions, we believe that each gene therapy will require a customized management approach. Administration and related procedures, patient acuity, timing and window of clinical efficacy may vary greatly from therapy to therapy, as each one is unique.
These new therapies will also pose new challenges in clinical, procedural, safety and storage support. For example, many of these therapies will require complex shipping and handling, time-sensitive delivery, and maintenance controls for extremely cold temperatures. We’ve built drug-specific processes, with a proprietary cold-chain technology system and state-of–the-art refrigeration mechanisms to support the unique needs of these complex medications.
The diverse requirements of gene therapies and the individual disease states they address require clinical and fulfillment models that are customized case-by-case.
Due to variability in long-term data from clinical trials associated with gene replacement therapies, there may be post-market surveillance requirements, or data collection, on patients receiving these new treatments. We can partner with stakeholders to institute a patient monitoring model that meets FDA and manufacturer guidelines on a drug-by-drug basis.
Approximately 4,000 diseases are linked to gene disorders, and many lack any effective treatment. Gene therapy has the potential to treat – and cure – some of the most debilitating diseases patients face.
With hundreds of gene therapies in the pipeline for varying conditions, we can expect gene therapy treatments will continue to be introduced, and Express Scripts, Accredo, and CuraScript SD are ready and able to support these life-changing – and life-saving – innovations.