Life-saving treatments available, but at exorbitant cost


By AGENCY

CAR-T cell therapy is one type of cell therapy where the patient’s immune cells are collected and genetically modified to better fight lymphoma, leukaemia and multiple myeloma. — TNS

Harnessing the body’s own cells to fight disease – long a medical dream – is finally a reality.

Now comes the bill.

Last month (April 2024), Stanford became the first hospital in the United States to use a new USD515,000 (RM2,428,341) cell therapy to treat a patient with advanced melanoma.

A related approach, costing USD420,000 (RM1,980,423) to USD475,000 (RM2,239,764), is offering hope to patients with lethal blood cancers.

Meanwhile, cells fixed by gene therapy can slow, even stop, the progression of intractable diseases like sickle cell or beta thalassaemia – for the extraordinary price of USD2.1 million (RM9.9 million) to USD4.25mil (RM20.04mil) each.

This is the future of medicine, experts agree.

But the cost of this new class of medical treatment is raising alarm about availability and affordability, even as its potential grows.

It’s time to reimagine our payment models, they say.

“Cell and gene therapies have the possibility to transform thousands of lives, but only if we ensure sustainable access to them for all patients,” say Sarah Emond, president of the Institute for Clinical and Economic Review (ICER), a Boston-based non-profit in the US that assesses the value of medicines.

The prices aren’t yet unmanageable, because so few people are currently treated.

Patients must travel to designated treatment centres around the US, and too few are referred by community physicians.

But demand should increase as more treatments are introduced that serve a wider population.

Most health insurers in the US aren’t set up to support one-time personal therapies that deliver long-term benefits at unprecedented prices.

“These are precision medicines,” says Dr David Miklos, chief of the Stanford Bone Marrow and Cell Therapy Program, where hundreds of cancer patients have been treated with chimeric antigen receptor (CAR) T-cell therapies.

“It’s different than buying a pill at the CVS [chain] pharmacy that can work for anybody.”

Changing the genes

It is a triumph decades in the making.

The promise of cell and gene therapies has intrigued scientists for decades, but progress was slow, with many setbacks.

Now, the long research journey has finally paid off, with US Food and Drug Administration (FDA)-approved products entering the clinic.

“The technology to bring it to life has finally caught up with the ideas behind it,” says Stanford assistant professor of medicine Dr Allison Betof Warner, who is conducting the hospital’s melanoma trial.

Cell-based strategies are delivering the most celebrated cancer treatments to emerge in decades.

In the CAR T-cell therapy approach, patients’ immune cells are collected and genetically modified to better fight lymphoma, leukaemia, and most recently, multiple myeloma.

Another different approach enlists tumour-infiltrating lymphocytes (TILs).

These immune cells are harvested from the tumour, fortified in the lab and then returned to the patient.

In clinical trials, about 30% of patients had their incurable melanoma tumours shrink or disappear.

“I’m very happy that it’s here now. ... I’ve been walking the tightrope and I didn’t fall off,” says a Stanford patient, who asks not to be identified.

Gene therapy also uses engineered cells, with genes replaced, deleted or inserted.

On May 1 (2024), Kendric Cromer, a 12-year-old boy from a suburb of Washington DC, became the first person in the world with sickle cell disease to begin a US FDA-approved gene therapy.

Stanford and the University of California, San Francisco (UCSF) will both offer this treatment.

Scientists are now working to expand the therapies’ repertoire to attack solid tumours, autoimmune diseases, ageing, HIV (human immunodeficiency virus) and more.

“It’s just the beginning of a new era,” says biochemist and UCSF Cell Design Institute director Professor Dr Wendell Lim.

“It shows that we can take a living cell and change what it does, so it makes new sorts of decisions and carries out complex actions.

“It processes information, like a little computer. This is very different from a static thing, like a chemical.”

Deterrents to treatment

It’s still early, and few patients are taking advantage of these new groundbreaking therapies, says physicians.

Why?

Word hasn’t gotten out yet, so sick people aren’t getting referred from their community physicians, says Dr Miklos.

Treatment is risky.

Or patients may live far away from the US’ estimated 30 “centres of excellence”, like Stanford and UCSF, and are daunted by the cost of travel and housing.

Payment by insurance isn’t guaranteed; it’s decided on a case-by-case basis.

US federal insurer Medicare and California state insurer Medi-Cal cover the cost of care when it is determined to be medically necessary.

So does insurance offered by the US managed care consortium Kaiser Permanente.

The great majority of private insurers cover treatments, although sometimes back-and-forth negotiations are needed, says Gary Goldstein, who oversees the business operations of Stanford’s Blood & Marrow Transplant Program.

The sticker price just covers parts – no labour, no warranty.

Drug prices aren’t regulated, like utilities, and there is no cap on what a company can charge.

The total cost for gene therapies over the next decade has been estimated to reach an eye-popping USD35 billion (RM165 billion) to USD40bil (RM189bil).

The bill for future cell therapies, which could help a bigger pool of patients, will likely be higher.

Drugmakers argue that the prices reflect the powerful clinical benefit, and the risks and uncertainties of development.

A one-time therapy for a chronic condition may actually save money, they add, by sparing a lifetime of care.

For some patients, it may be their last best chance.

“I don’t think there’s a price on a life,” says Assist Prof Betof Warner.

“These are patients who don’t have another option.”

If millions of people are helped, as hoped, it will create budget pressures on the US federal government and larger payers, while smaller employers, state Medicaid plans and regional health plans may find providing access financially impossible, warn economists.

Healthcare costs are already outpacing inflation, climbing 7% this year.

“It’s going to put a lot of stress on the system,” says Prof Edwin Park from the Georgetown University McCourt School of Public Policy.

“But the issue is critical because you don’t want the high price of these therapies to result in low-income people not being able to access them.”

Already, governments and commercial insurers are scrutinising the treatments’ effectiveness.

Some are imposing strict restrictions on who is eligible. And they’re asking manufacturers for discounts and rebates.

Different options

Scientific advances could cut costs, says Prof Lim.

One idea is to design cells that are immunologically universal, so that a single source could treat many patients.

Another is to build a large “cell bank” of precursor stem cells.

A third is to ask the body to do its own manufacturing, by introducing an engineered virus that can fix cells.

If manufacturing is localised, that would bring costs down, says Dr Miklos.

As competition grows, prices will fall, he predicts.

Meanwhile, the healthcare system must focus on finding innovative payment solutions, says Emond.

One proposal is to amortise how much insurers pay over time, like a home mortgage.

Another is for drug companies to provide a prorated refund if a patient doesn’t improve – a “pay for performance” model.

Yet another option would be a subscription-based approach, like Netflix, where insurers shell out a monthly fee to access however much therapy is needed.

Each condition, therapy and payer is unique, so a single solution won’t satisfy all situations, say experts.

“As we look to the future,” says Emond, “this is a moment where we can discuss how to do things differently.” – By Lisa M. Krieger/Bay Area News Group/Tribune News Service

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