Jigar Thakkar, Pharm D, MBA, MHCDS, FACHE is the Founder and CEO of Longitude Rx.
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Imagine a system with a malfunction that can be controlled as long as someone is continuously making adjustments. Those adjustments may work extremely well, but if they stop, the underlying problem can reemerge.
Modern medicine has become increasingly precise, with targeted therapies that act on specific drivers of disease. These therapies have transformed the treatment of many diseases, but many require patients to remain on treatment to maintain that control.
Cell and gene therapies introduce a different possibility: changing the biology itself. Some gene therapies can correct or replace faulty genetic instructions, while others, such as CAR T cell therapy, reprogram a patient’s own cells to find and eliminate the disease. For some patients, a single treatment can produce a durable or potentially curative response, reducing the need for years of ongoing therapy and the associated burden, side effects and cost.
There are more cell and gene therapies today than ever before. Some reports estimate the U.S. will spend more than $25 billion on gene therapies alone in 2026.
These therapies are creating new possibilities for patients with conditions like spinal muscular atrophy or hemophilia, including diseases for which treatment options were historically limited. But turning this scientific progress into broader patient access presents challenges, forcing organizations to reconsider how care is identified, financed and delivered.
The Value Of Cell And Gene Therapy
Cell and gene therapy has the potential to treat previously untreatable genetic disorders and cancers, such as advanced leukemia and sickle cell disease.
These therapies also offer cost savings to both patients and health systems. Instead of a patient returning over and over again for care as symptoms pop up, cell and gene therapies may eradicate the problem once and for all.
For example, the cost of a gene therapy to treat hemophilia B can climb to $3.5 million for a single treatment. However, the 10-year cost of ongoing treatment for the same condition can add up to much more. And that doesn’t account for the patient’s extreme daily burdens, including chronic pain, physical mobility limitations and psychological distress.
While immeasurably valuable, the steep upfront costs associated with cell and gene can severely limit access. It challenges healthcare organizations to rethink the economics and infrastructure of chronic care. Thus, payers, health systems and pharmaceutical companies will need to work together to manage costs and improve access.
Improving Access And Lowering Costs
The barriers to expanding access can be divided into three areas:
1. Cost
Payers are skeptical of including cell and gene therapy in healthcare contracts because they want to see more predictability in terms of patient outcomes. They also want to ensure patients are receiving the right treatment. Predictability is difficult to prove when only an estimated
eligible for cell and gene therapies actually receive them.
As a result, many cell and gene therapies are handled on a single case agreement between the payer and provider, which can lead to reimbursement delays of well over
. That’s compared to the typical 45-day reimbursement turnaround for more common treatments.
To fix this, providers must ensure they are staying up to date on available breakthrough treatments so they’re identifying all eligible patients. A culture needs to be created where patients feel comfortable asking their provider for alternative treatments if they no longer are responding well to the standard of care.
2. Finding The Right Patients
The majority of patients eligible for cell and gene therapy never end up receiving this care. This is because most healthcare facilities offering these treatments are top-tier health systems and academic centers, which are typically found in more urban areas.
For patients in rural areas, access to these breakthrough treatments requires more coordination, support and financial means to travel to larger medical centers. It also requires the improvement of communication between rural and urban providers.
To fix this, the infrastructure must be modernized to allow health systems to build scalable cell and gene therapies. This includes enhancing transparency of patient records with appropriate providers and creating a care coordination system that directly supports patients who are eligible for cell and gene therapies.
3. Improving And Expanding Clinical Expertise
Hundreds of new therapies come out each year, and the American Society of Gene + Cell Therapy estimates there are currently more than 4,000 cell and gene therapies in development. Eventually, the goal is to expand cell and gene therapy access so that even rural hospitals can offer them.
Understanding and awareness is limited by treatment complexity, specialized infrastructure gaps and small patient populations. These cutting-edge modalities require rigorous handling protocols and long-term tracking and medical training, details that community healthcare settings are not yet equipped to provide.
The relatively small patient population receiving cell and gene therapies is one of the challenges in ensuring rural providers are properly trained. However, this step is crucial in expanding the number of eligible patients taking part in these therapies. Only when more eligible patients are participating can efforts to expand cell and gene therapy offerings in rural locations be possible. However, even then, rural facilities will need to ensure they have the necessary resources to offer these often complex therapies.
These barriers are deeply interconnected. Cost and predictability cannot improve without broader patient access. Expanding access requires stronger communication between community providers and health systems, along with greater clinical expertise to identify and refer eligible patients.
The Future Of Cell And Gene Therapies
The amount of time, money and resources being allocated to developing new cell and gene therapies indicates that the market will continue its rapid growth. In order to support this growth, the infrastructure of the system must improve. Costs must come down, the number of patients treated must go up and knowledge of and access to cell and gene therapies must be improved.
Addressing these barriers will take a collaborative approach with better coordination among providers, payers and health systems, combined with stronger data-sharing, patient identification and clinical education. With the right systems in place, I believe we can help make cell and gene therapies move from highly specialized interventions to a more scalable part of modern healthcare.
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