Hannah Song, PhD
Contributing Editor, ISCT Telegraft
NIH Clinical Center
United States
Interview with ISCT Chief Medical Officer, Jaap Jan Boelens, MD, PhD
Treating the Cause, Not the Symptoms
What if, instead of treating the symptoms of a disease, we could treat the root cause? Cell and gene therapies (CGTs) are targeted therapies that make use of a patient’s own cells – often immune cells or stem cells – and redirects them to address their own unique disease, with their own cells as the starting material. Examples include engineered T cells for leukemia and lymphoma, and stem cells engineered to overcome congenital diseases like sickle cell, thalassemia, and inborn errors of immunity. Real-world evidence shows that intervening with targeted genetic changes to a patient’s own cells can reverse the trajectory of disease. And when it works, the results can be transformative – a functional cure for previously incurable diseases.
Barriers to Accessing CGTs
Unfortunately, accessing this kind of therapy currently depends on which part of the world you live in. The costs of CGTs are high, and the logistics of delivering them are complex. These two things – high cost and complex logistics – overlayed on top of disparate global healthcare systems means that regions are currently facing their own unique problems establishing access to CGTs. For example, in the United States, access might depend on how close a patient lives to an approved treatment center or what insurance plan they have. In Europe, where population densities are higher and treatment centers are more readily accessible, it may depend more on the patient’s ability to cover the expense of an extended stay at a treatment center, as most CGTs require extensive follow-up. And in low-income countries, it might simply be cost prohibitive. In that case, regional development of cheaper in-house methods – so-called “homegrown solutions” – may be the only path forward to establish access. Still, there is huge common ground amongst regions. That’s why the International Society for Cell and Gene Therapies (ISCT) is bringing together the world’s experts to solve these gaps in access.
Finding Gaps, Identifying Solutions
Dr. Jaap Jan Boelens, M.D., Ph.D., chief of the Bone Marrow Transplant and Cellular Therapies Center at the Memorial Sloan Kettering Cancer Center in New York and the inaugural Chief Medical Officer of ISCT, recognizes the need for clinicians to bring their unique CGT experience to the table. With its new clinical pillar, ISCT is positioning itself to learn from each region’s successes and challenges to ultimately deliver on the curative potential of CGTs. Alongside its established network of translational scientists, global regulators, and industry experts, ISCT is building its member network to include clinical care providers – not only doctors, but also nurses, pharmacists, and other advanced practice providers who contribute immensely to the clinical care integral to CGTs.
While some argue that market adoption of approved CGTs could make or break the field, Dr. Boelens is quick to point out that academic centers are showing they can offer less expensive point-of-care solutions. He points to the ARI program in Spain, as well as ImmunoACT in India, as examples of regional hubs offering CGTs priced well under the status quo (some estimates put it at one-tenth of the cost). And so far, “these therapies seem to deliver similar benefits” as their commercial CGT counterparts, says Dr. Boelens.
Reimbursement — A Hidden Hurdle
Both academic centers and industry therapy developers alike must face the issue reimbursement – who pays for what, and when. For example, the ARI program worked out a reimbursement agreement so that their T cell therapy is fully covered in Spain. However, separate negotiations are underway with the European Medicines Agency (EMA) that would widen access to ARI’s product within Europe, although there is concern about what will happen with the therapy’s cost. In the United States, where patients have insurance coverage through a mix of Medicaid, Medicare, or commercial insurance providers, the reimbursement negotiations are more tedious. In his clinic, Dr. Boelens notes that working out a single case agreement – a one-time agreement between an insurance company and a CGT provider that unlocks the ability for the insurance to cover a therapy – can take months. And then, “as soon as another patient walks through the door, if they have a different insurance, it starts all over,” says Dr. Boelens. Learning from one negotiation helps streamline the next (for example, Dr. Boelens realized he needed to re-negotiate the number of cell collections needed at the start of the process), but even so, it usually takes at least 2 weeks, and that’s on top of obtaining prior authorization.
Ultimately, this insurance delay is no doubt a source of patient attrition – some patients simply don’t have the time before they progress out of eligibility, too sick to receive their specialized therapy.
The Future of CGTs
Despite the complex care environment, the field has made substantial progress establishing pathways to access, amassing a suite of approved CGT products across the hematology and oncology space. And progress continues: the same engineered T cell therapies that were initially developed for leukemia have recently shown remarkable early success in reversing devastating autoimmune disorders like lupus. Still, we have much to learn as approved products hit the real world. That’s where ISCT’s newly established clinical pillar comes into play.
“How can we learn from the real-world data? What can we learn from access models in other regions? Obviously, the needs in Europe might be different than in South America or Asia. The gaps might be different, but there is significant common ground,” says Dr. Boelens. ISCT now embarks on that task of finding and addressing those common gaps.
And the list of approved of CGTs keeps growing. “We’re still going to face questions of access, side effects, and efficacy,” says Dr. Boelens. “It’s only the beginning.”
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