''Cell Therapy R & D Guide'', What Obstacles Will Be Faced In The Use Of Cell Therapy In The Real World?

By BioLink & Bio-Valley

Based on its excellent efficacy and broad imagination, CAR-T cell therapy has become a new savior expected by cancer patients around the world. However, worldwide, the number of CAR-T prescriptions that should have grown with the number of CAR-T approvals has increased slowly.

What is the reason that hinders the use of CAR-T in the real world?

International CAR-T usage situation

So far, the U.S. FDA has approved 6 CAR-T therapies, covering five hematological tumors, namely diffuse large B-cell lymphoma (DLBCL), acute lymphoblastic leukemia (ALL), and follicular lymphoma ( FL), mantle cell lymphoma, and multiple myeloma.

Monthly CAR-T prescriptions have not increased in 2021, and prescriptions in the second half of 2021 are similar to those in the second half of 2020, according to a study by the American Cancer Institute.

It is worth mentioning that in 2021, the FDA has approved two new CAR-T cell therapy products. In addition to Breyanzi's indications that already have similar products, another BCMA CAR-T product, Abecma, has been approved for a brand new indication—— multiple myeloma.

With the introduction of new products and indications, the US sales of CAR-T in the second half of 2021 did not rise as we expected but remained the same as, or almost the same as, the second half of 2020.

It is worth noting that in the second half of 2020, the global Covid-19 epidemic is still in a relatively serious state.

Barriers to the use of CAR-T

The real data that does not match the expectations shows that there are relatively big problems in the use of CAR-T in the real world.

Patient health status and disease progression

The current CAR-T cells need to be prepared from the patient's own cells, but most tumor patients who meet the CAR-T medication standards have basically received multiple lines of treatment, and the immune system in the body has been greatly damaged, whether it can provide qualified and sufficient T cells to transform into CAR-T cells, is a more critical issue.

Secondly, such patients are generally patients with recurrent tumors, and the disease generally progresses to an advanced stage. Such patients have very rapid disease progression and may not be able to persist until the cell preparation is completed, usually in 2-4 weeks.

Production plants and manufacturing issues

The preparation of autologous CAR-T cells requires a single person and a single batch, that is, a complete set of production equipment needs to be prepared for a single patient. It is a relatively big challenge and requirement for factory production equipment and space because manufacturers need to build factories in advance, and the cost of factories is very large.

Previously, Bristol-Myers Squibb had said that the production capacity of the CAR-T drug Abecma could not meet the needs of patients in the market. In recent years, several major CAR-T developers have also begun to deploy new cell therapy factories.

After talking about the production capacity problem, when it comes to the manufacturing problem, CAR-T cells actually have a production failure rate. Although qualified T cells are obtained from patients, there will be manufacturing problems from original cells to finished CAR-T cells, or preparation failures, substandard products, and so on.

Medical Sites and Cold Chain Logistics

In the United States, there are currently several hospitals that can support CAR-T treatment, and not just any hospital can carry out CAR-T cell reinfusion. The region, transportation, and cost limit the choice of CAR-T cell therapy for most patients.

Secondly, the transportation of CAR-T cells requires cold chain transportation, and the temperature change on the transportation route will lead to quality problems for the entire product. However, this problem can be solved to a large extent under the deployment of the mRNA new crown vaccine in the United States.

For the Chinese market, the issue of medical facilities and cold chain logistics is still quite challenging.

Prices and Fees

The price range of CAR-T in the United States is in the range of 300,000 to 500,000 US dollars. Although the United States supports innovative drugs and commercial medical insurance, patients do not need to pay in full, but part of the self-payment is also a sum for many patients. Not a small expense, especially after multiple treatments.

In addition to the cost of CAR-T treatment itself, the pretreatment of patients receiving CAR-T treatment before infusion, the management of side effects after reinfusion, hospital care costs, etc., also needs to be calculated separately.

In China, the current medical insurance does not cover CAR-T treatment, and the payment environment for CAR-T cell treatment is still relatively difficult for patients and their families. However, a number of commercial insurances currently cover CAR-T cell therapy, which may provide some support for the accessibility of CAR-T.

Compared with the industry's optimism about CAR-T cell therapy, the commercialization of CAR-T has not met expectations. How to improve the accessibility of CAR-T cell therapy to drive the sales of CAR-T cells is also a very critical issue facing CAR-T cells at present.

BioLink cooperated with Bio-Valley to conduct a multi-party investigation and summary, and launched the "Cell Therapy R&D Guide". Combined with actual cases, it sorted out the cell therapy from project establishment - to drug evaluation - process development/transfer - quality analysis - drug listing registration - process scale-up production, etc. The key nodes and specific content of the link will help you quickly master the key technologies and the whole process of cell therapy drug development.

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