We're in the middle of a cancer drug innovation gold rush -- but it has one major side effect

The world of cancer treatments is an explosive areas, especially as treatments get more targeted.

But with that are going to come some big costs. The IMS Institute for Healthcare Informatics expects cancer treatment costs to hit $150 billion by 2020, up from $107 billion in 2015.

Enter a new potential side effect: debilitating cost. How do doctors and researchers factor in the costs that a patient is paying? That’s the kind of thing Dr. Derek Raghavan, president of Levine Cancer Institute at Carolinas HealthCare, thinks about a lot.

“We are spending more and more time thinking about cost for patients,” he told Business Insider. He pointed to a study by University of Chicago researcher Dr. Jonas de Souza who uses the term “financial toxicity” to look at how much of a role finances play in a cancer patient’s quality of life.

Cancer isn’t just one disease: It’s a whole group of cancers. It’s gotten so big that whole companies are devoted to developing cancer treatments. IMS cites more than 500 companies that are developing cancer drugs. In fact, pharmaceutical company Novartis just split out its oncology business into a separate branch from its other disease areas.

And because it’s so complex, there’s a whole web of decision-making that needs to happen as cancer progress, while at the same time, newer drugs become more targeted and more specific. Here’s how leading cancer researchers and pharmaceutical executives are addressing it all.

The questions to ask

Raghavan said there are two main questions he thinks about when determining how to treat a cancer patient.

“The first question is how much survival benefit will you get from the patient?” he said. “Three weeks? Not worth it. Three months, maybe. But probably not. If you’re talking a year plus, that’s a relatively easy one to solve.”

But that three months gets a little complicated if it means there aren’t any extreme side effects (either physical or breaking the bank). If that three months means seeing a major life event, like a holiday or graduation, it’s likely worth it.

“Everything is contextual. If you’re dealing with an 83-year-old who’s had a stroke and a coronary, and you can do something that’s going to make them really sick, but will give them an extra three months, you’d have to be an idiot to say that’s worth it to them,” he said. “But you also have to ask them: what’s important to them?”

Newer treatments, including the fast-growing field of immunotherapy (drugs that harness the immune system to better attack cancer cells), are seeing much better and longer survival rates, which is also where costs will factor in.

Adding one expensive drug to another

Within immunotherapy, researchers are trying to figure out how to get more people to respond to treatment. Take Keytruda, the drug that’s credited with helping get President Jimmy Carter cancer-free after he was diagnosed with melanoma that had spread to his liver and brain. Only about 30% of metastatic melanoma patients using Keytruda alone respond completely. That’s still better than the average response rate of chemotherapy treatments, on their own, in cases of metastatic melanoma, but it’s not 100%. However, companies think the solution may lie somewhere in combination with other drugs, potentially even other immunotherapies.

But the problem is, these drugs can often be upwards of $100,000 for a course of treatment, depending on how long you stay on the drug. Add that to another $100,000 drug, and the cost for such good results is a bit staggering.

Trials are already being run for combinations of two and even three immunotherapies at the same time. Beyond that, companies are looking at ways to pair drugs together to help them work better. For example, ones that target the programmed cell death 1 (or PD-1) receptor to essentially take the “foot off the brake pedal” in the immune system might be put together with other immunotherapies that activate the immune system in different ways. Or, those PD-1 inhibitors might be matched up with targeted chemotherapies, or other tools in the cancer treatment toolbox.

Pfizer, which has a number of immunotherapy compounds in development and a partnership with Merck KGaA to develop the PD-L1 drug avelumab, said it plans to work closely with other areas of health care to set prices on combinations based on how well they work.

“I think that the concern that’s being raised time and time again is more about the future and around combinations,” Pfizer’s global president of oncology Liz Barrett told Business Insider. “And I think when you start to think about that, that’s where I feel like Pfizer’s in a unique position, because we have the multiple I-O drugs and the targeted therapy drugs.”

The questions left to answer

Before companies even get to that pricing question, there are a lot of unknowns still left in the immunotherapy world. Merck & Co., the company that makes Keytruda, thinks a solution lies in answering the question about where combinations are necessary, and where drugs like Keytruda can work the best on its own.

“We know for some patients, just single application or single modality treatment with a PD-1 antibody is enough,” the company’s senior vice president of clinical development Roy Baynes told Business Insider. But for those who don’t, there are a few questions he pinpointed that need to be answered next: “Can we identify the group that is most likely to benefit from single-agent therapy? Next, who needs a combination and can we identify which combination?”

Ideally, the clinical trials that are ongoing or in the works will give researchers some hints as to what those answers may be.

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