CAR-T-Zelltherapie an mehr Patienten heranbringen, und das früher

Einige Patienten haben Schwierigkeiten, eine CAR-T-Zell-Therapie zu erhalten, die ihnen potenziell nützen könnte.

Over the past few years, there has been important progress in treating certain blood cancers with chimeric antigen receptor (CAR) T cell therapy, leading to the approval of two of these novel treatments last year. But ensuring patients can access these innovative therapies remains a top concern for doctors, patient advocacy organizations and other stakeholders in the life sciences community.

Those challenges were the focus of a special panel sponsored by Celgene during the 2018 Biotechnology Innovation Organization (BIO) International Conference, which was held from June 4-7 in Boston. Richard Bagger, executive vice president of Celgene Corporate Affairs and Market Access, moderated the panel, which included experts with backgrounds as varied as medical research, economics and health policy.

“We are at the threshold of a very exciting time,” said panelist Nupoor Raje, Massachusetts General Hospital. “The progress that we are seeing is promising.”

This progress also comes with challenges and potential barriers for patients. The panelists agreed that those challenges should not prevent patients who could potentially benefit from CAR T cell therapies from having affordable access to them. Part of the discussion highlighted how a number of stakeholders will need to work together to shape the regulatory and reimbursement environment for optimal patient access.

Richard Bagger


Ensuring Access

Operationally, the production of approved CAR T cell therapies is as complex as each treatment is highly personalized and unique to every patient.  A patient’s T cells are extracted at a treatment center, sent to a manufacturing facility where they are engineered to recognize a specific protein or antigen and attack cells, including cancer cells that have this antigen, and then sent back to be infused into the patient. The process requires an unprecedented level of collaboration between doctors, health centers and manufacturers.

One of the approved CAR T cell therapies is for pediatric patients with relapsed or refractory acute lymphoblastic leukemia. Both approved CAR T cell therapies are for adults with relapsed or refractory diffuse large B-cell lymphoma after two or more lines of therapy.  Celgene CAR T cell therapies are still investigational and are not yet approved by the Food and Drug Administration.

However, a significant blockade to patient access exists—these personalized therapies don’t fit neatly into private payer or government payment models, including Medicare, meaning delays for patients and complications for providers.

The last thing we want to do is deny a patient a therapy that could potentially benefit them.

“Waiting two to four weeks for insurance approval could be too long for many of these patients,” panelist Dr. Gwen Nichols, the Chief Medical Officer of The Leukemia & Lymphoma Society, said.

And despite the financial losses that hospitals face when Medicare and insurance reimbursement does not adequately cover the costs of CAR T cell therapy, many centers continue to try to deliver timely treatments to meet patient demand, according to panelist Beth Roberts, a health policy lawyer at the firm Hogan Lovells.

As a possible solution to expedite patient access and simplify issues for providers, panelist Henry Grabowski, Ph.D., Professor Emeritus of Economics at Duke University, suggested that private and public insurers should explore innovative payment arrangements such as payment plans, flexible pricing and value-based contracts.

Patients Are Waiting

Beyond the issue of reimbursement, the health care system is still increasing capacity to provide CAR T cell therapies to more people. For instance, because of the highly specialized expertise required, only a few community cancer centers can administer CAR T cell therapies, so patients often must travel far for treatment.

Preparing today so patients can access innovative cancer treatments such as the CAR T cell therapies must be a top priority for our health care system. “We need to manage the hype and continue to let the science lead the way,” Raje said. “The last thing we want to do is deny a patient a therapy that could potentially benefit them.”

To learn why CAR T cell therapy represents an important advance for some patients, read “CAR T Cell Research Continues to Advance.”