How to Save Cancer Research
from Regulation and Red Tape

There is plenty of exciting new research on cancer. But the research can’t make it to the doctor's office until it goes through clinical trials — and there are reams of red tape standing in the way.

infographic link

The cure for cancer could be sitting in a dusty medical journal right now, part of a growing list of ideas for treating cancer that haven’t been tried. This is largely because researchers can’t recruit enough patients to test them all.

There’s not a lot of public outcry about clinical trials that don’t meet their patient recruiting goals, but the problem creates a major bottleneck for medical research.

Low turnout for clinical trials is not new, and many of the problems that fuel it are hardly surprising. Patients fear new treatments — sometimes fairly, and sometimes because they misunderstand the risks and safeguards laid out in sprawling legal consent documents. They may not meet narrow eligibility rules or live close enough to research centers to get to regular appointments.

There’s a lot of bureaucracy involved in running a trial, too, from the government and from within research centers themselves.

Bureaucratic processes can insert five months or more between the moment a trial gets the green light and the moment it recruits its first patient, a study at the Vanderbilt-Ingram Cancer Center found. By that time, the research may be less exciting to patients and their doctors.

All told, fewer than 5 percent of adult cancer patients in the United States choose to participate in a trial.

Low participation rates are, however, an increasingly important barrier for cancer researchers to overcome. Advances in the scientific understanding of cancer have made clinical trials more crucial than ever, at a time when medical research budgets are shrinking.

section 2

An Abundance of Riches

Dr. Carl June at the University of Pennsylvania has developed perhaps the most exciting experimental treatment in oncology in recent years. June and his colleagues program patients’ immune T cells to target a particular molecule on lymphoma cells.

Lymphoma patients who otherwise may have died in a matter of weeks find themselves enjoying long remissions.

The treatment builds on an expanded understanding of how cancer works at the genetic and molecular levels, made possible by recent advances in gene sequencing.

The discovery of another molecular dance the immune system performs with cancer cells is driving the development of promising immunotherapy drugs to treat melanoma and other cancers. Keytruda, which jumpstarts the immune system’s own fight against cancer, won the approval of the Food and Drug Administration (FDA) last month. Other, similar drugs that act on the so-called PD-1 cellular pathway are expected to follow.


Earlier advances in cancer genetics have already entered the common lexicon about cancer. Breast cancer patients refer to their “triple-negative” and “HER2” cancers. These terms describe molecular particulars of tumors that can be used to pinpoint the best treatments. Doctors no longer see cancer as a single disease but as a growing number of related diseases.

However, many other discoveries have not yet been translated into treatments. Just a tiny fraction of new molecular targets identified in the lab have led to active research on human patients. Any of these could lead to a cure at least as effective as June’s.

As cancer patients get more and more precise diagnoses, researchers have to cast a wider net to fill their trials with patients whose cancer has the exact genetic features that their treatments are designed to target.

They haven’t widened their nets enough. Only 10 to 20 percent of cancer patients ever hear that clinical trials are an option. Women, seniors, and non-white patients are least likely to learn about trials.

Even at major cancer research centers where trials are conducted, not all patients learn that there may be new treatments available at no charge.

Patients don’t pay for treatments they receive as part of clinical trials. Uncle Sam does, as part of the money the government doles out for medical research. And just when researchers need more patients, there is much less money to go around.

section 2

Tighter Belts, Fewer Trials

While Congress was briefly in session this fall, researchers and patient advocates flooded the U.S. Capitol to sound the alarm that medical research as we know it is in jeopardy.

The remarkable progress in the fight against cancer in recent decades has come with a price tag, and the federal government is less willing to pay it now. The National Institutes of Health (NIH), the major source of public funds for cancer research, has seen its budget shrink since the early 2000s. In 2013, Congress siphoned off another $1.6 billion of its budget as part of the so-called sequester.

The odds of having a medical research grant application approved by the NIH now stands at 1 in 6. It costs upwards of $15,000 per patient to run even a short a clinical trial, and the cost is rising as cancer research becomes higher tech.


In 2009, nearly 30,000 patients were enrolled in NIH-funded clinical trials for cancer. Just four years later, that number had fallen by a third, according to the American Society of Clinical Oncology.

If funding cuts weeded out only the least promising studies, it wouldn’t be a problem. But that’s not what happens.

Researchers tend to play it safe to ensure they get funding when it’s scarce, according to Dr. Louis Weiner, director of Georgetown University’s Lombardi Comprehensive Cancer Center and a member of the American Association for Cancer Research.

To maximize the chances that their experiments will work, researchers often look for tweaks to existing treatments instead of exploring the kind of radically new approaches that have led to major advances like June’s.

June’s work is now making its way through the FDA pipeline as a potential breakthrough therapy. The pharmaceutical company Novartis is paying for its final trials. Companies often step in to fund research when there’s a commercial product that has a good chance of winning FDA approval.

But the work was once “a wild and crazy idea” that struggled to find support, said Weiner. “There wasn’t a pharmaceutical company in the galaxy that was willing to invest in it in the early stages because they couldn’t see a tractable path forward,” Weiner said. Only government funding kept the research going.

There wasn’t a pharmaceutical company in the galaxy that was willing to invest in it in the early stages because they couldn’t see a tractable path forward.
Dr. Louis Weiner, Georgetown University’s Lombardi Comprehensive Cancer Center (on why federal funding keeps early stage cancer research going)

Few trials produce results as dramatic as June’s, but success stories like his drive public interest in clinical trials. Patients are less willing to participate in trials if the treatment isn’t exciting, experts told Healthline. Doctors are less likely to send their patients off to researchers if they don’t see a clear benefit.

Weiner and other researchers are aggressively speaking out about federal research budget cuts. They say the pipeline of new developments may dry up, eventually depriving patients of the options clinical research has provided.

Patients could find themselves abruptly at the end of what they have come to see as an everlasting supply of new drugs to try when the cancer outsmarts their current regimen. This would affect people like Alice Gordon, a 62-year-old retired New Jersey teacher who has had metastatic breast cancer since 2002.

“People who are at stage 1 wonder, ‘If a trial drug fails, what if that makes things worse?’ They don’t understand that there are all these new drugs out there. There’s always another drug. But the only way these drugs come out is by people doing the trials,” Gordon said. 

section 2

Rethinking Research Fundamentals

Kathy Giusti was diagnosed with a type of blood cancer called multiple myeloma in 1998, at the age of 37. Doctors prescribed the same chemotherapy drugs her grandfather had received before he died of the same disease 30 years earlier.

In this case, a dry drug pipeline triggered a tsunami of changes. One could argue that Giusti has done more than anyone else to improve clinical cancer research. A Harvard Business School graduate who was then a pharmaceutical executive, Giusti didn’t get her affairs in order, as her doctors advised. Instead, she started the Multiple Myeloma Research Foundation (MMRF), which has gone on to become one of the most influential cancer advocacy groups in the country.

The foundation brought pharmaceutical companies and major cancer centers into a research consortium, setting the rules that allowed them to play well together. Then members of the group divided up research questions systematically to move through them faster.

They share access to a tissue bank of biopsied tumors. Genetic sequencing results are stored in a shared data portal that also houses patients’ medical data. Researchers can do a lot without ever seeing a patient.

Now led by Walter Capone, MMRF uses the patient support services it offers to encourage patients to join the research efforts. The nurses who staff its support line understand the science and, unlike many busy doctors, have the time to explain it.

We put the tools back in patients’ hands to regain the control that was taken away by this diagnosis.
Walter Capone, head of the Multiple Myeloma Research Foundation (MMRF)

“We put the tools back in patients’ hands to regain the control that was taken away by this diagnosis,” Capone told Healthline.

The results have been impressive. In the decade since the consortium launched, six new drugs have been approved to treat multiple myeloma. Median life expectancy has nearly tripled, from three years to eight. Giusti herself has lived five times as long as her doctors predicted and remains active on the MMRF board.

Other advocacy groups have adopted this model, more aggressively connecting patients with open studies. Researchers increasingly look to collaborative research models to answer big questions.

They’re also more interested in sharing data in real time. Earlier this year, the National Cancer Institute launched Lung-MAP. This public-private research collaboration matches patients who have particular cancer biomarkers with trials testing drugs to treat them.

Since 2010, the NIH also has been working to improve trial recruitment by laying out best practices. The recommendations include getting more input from patients and patient advocacy groups early in the process of trial design.

They also encourage major cancer centers to support smaller hospitals in their area that are willing to carry out clinical trials. Cancer centers including Vanderbilt, the University of Colorado, and Memorial Sloan Kettering have built such networks.


The focus on the best ways to design trials to appeal to patients has brought attention to the need for randomized studies to compare treatment options that are fairly similar. Imagine, for instance, a trial in which the standard of care was observation and the experimental protocol was radiation and surgery. It would be hard to find a patient who would be happy to be randomly assigned to either arm.

“Over the 20 years that I’ve been in practice, people have gotten better at figuring out how to spot a study that you just won’t get patients to agree to,” said Dr. Gregory Masters. He’s an oncologist at the Helen F. Graham Cancer Center in Newark, Delaware, a community cancer center that runs clinical trials.

What patients will do doesn’t always sync with how scientists would like to answer research questions, but they are getting better at meeting in the middle.

“If we’re not flexible, then we won’t get research done. I prefer to look at that in an optimistic way and say there are other ways to get things done. Can we make studies smaller, so they’re not as expensive? Can we answer more defined questions rather than the huge global question and still move forward?” Masters said.

section 2

Plugging In to New Technologies

The NIH’s efforts aim to make trials more appealing to the patients who hear about them. But what about the other 80 to 90 percent who don’t?

Currently, researchers rely on community oncologists to spend time introducing patients to clinical trials that would send them outside of the practice for care. Instead, doctors could schedule an online chat so that the researcher could speak directly to interested patients.

Dr. Matthew Galsky, an oncologist at The Mount Sinai Hospital in New York, is researching how to use technology to improve trial recruitment. He sees telemedicine as one way to expand access. In a current study, he’s monitoring chemotherapy patients via video conference. This technique could extend cutting-edge care to patients who live far from any trial site.

“What we cover during the visits is identical to what we’d cover if the patient was in the office,” he explained. “If the model is tested and demonstrates feasibility, then we could test more complicated interventions.” For example, technology already exists to alert doctors when a patient takes a pill.

The NIH is exploring a similar idea, based on research suggesting that the way doctors describe trials conveys their own bias about the promise of the treatment, even when they think they’re being impartial. A pre-recorded YouTube video could be vetted for traces of bias, said Andrea Denicoff, RN, who leads operations at the National Cancer Institute’s National Clinical Trials Network. The same video could then be shown to every interested patient, saving doctors time.


The NIH recommendations, which Denicoff helped to develop, also encourage researchers to use social media in the basic ways that most businesses already do. Many researchers express frustration that their institutions haven’t fully embraced the technology.

“Clinical research has to come up to the speed of social media and information technology and data communication as any other industry,” said Dr. Pamela Munster, director of the early phase clinical trial program at the University of California, San Francisco (UCSF).

It’s not just a question of setting up Facebook pages. Researchers are required to make certain disclosures and are forbidden from making others. These rules make it difficult to communicate in short tweets or status updates. There’s a patchwork of regulations covering how doctors handle even patients’ most basic information.  

Patient support groups are better positioned than researchers to put information in plain English out on the web, Munster said.

“Some of the support groups have a nationwide network and they blog about trials. All the things I can’t say they put on their website, and I get a lot of referrals from them. Our hands are tied with a lot of the privacy issues,” she said.

At least one company, Antidote, hopes to fill the gap. (Full disclosure: Healthline has an affiliate partnership with Antidote.)

“The big problem is that patients don’t have the information, and that causes a terrible disconnect between patients and the people doing the research,” said Sarah Kerruish, a former Silicon Valley entrepreneur who now heads development at Antidote.

The big problem is that patients don’t have the information, and that causes a terrible disconnect between patients and the people doing the research.
Sarah Kerruish, head of development at Antidote

Next spring, the company will organize a social media campaign to encourage 1 million people to participate in cancer research. Antidote is also building an online navigator that will make trials easier to find. This will translate information from the medical jargon found on, the website where the government publicizes medical research, into plain English.

An even more challenging problem is sharing data on trial eligibility, availability, and results in real time, while still protecting patient privacy.

Although UCSF sits in the beating heart of San Francisco’s technology industry, researchers there don’t have even simple real-time information-sharing software. Research funding often doesn’t cover administrative overhead.

“Clinical trials are in enormous flux. I may have five slots on a trial this week, but they are gone next week. In any other industry, we’d have a pretty much up-to-date website. We tried that, but we simply don’t have the support to do it. There’s not enough research money to fix that problem,” Munster said.

Lastly, many health technology companies (including Healthline) are vying to use aggregated data from electronic health records to offer a range of products and services. But patients and doctors have expressed concern that so much sensitive personal information will invite hackers keen to steal health data.

Both MMRF and Lung-MAP suggest that it can be done, and for-profit companies are working to make such data mining more secure and more routine.

section 2

Chipping Away at a Mountain of Problems

In the meantime, there are a few low-tech solutions that could help smooth out the bureaucratic bumps. Dr. Dan Theodorescu, who leads the Comprehensive Cancer Center at the University of Colorado, has a few ideas.

What about a cover sheet to introduce the detailed study consent forms, comparing the risks of the study treatment to the risks of standard treatment? Experts say that presenting patients with 50 pages or more on the risks of the new treatment and zero pages on the risks of the standard course, as is done now, stacks the deck against research.

“Patients are just seeing what the [trial] drug will do, and that’s scary,” Theodorescu said. But “you’re not comparing the side effects to doing nothing.”

He proposes a handful of simple graphs comparing the risks of the study treatment to those of the conventional treatment to simplify matters for patients. Because the cover sheet wouldn’t remove the required disclosures, the FDA would likely not object. Dr. Richard Pazdur, who leads the FDA’s Office of Oncology Drug Products, said of Theodorescu’s idea, “I’d see that as a positive point.”

Theodorescu also argues that when multiple institutions operate arms of the same study — which is good, since it gives researchers access to more patients — they should simplify the process by which they agree on patient protections. Every medical center that does clinical research has an institutional review board, as required by the FDA.

When several institutions cooperate on a study, each board independently vets the terms. The process results in “tweaking, changes, delays, and cost for physicians that really don’t have time to do this,” Theodorescu said. The paperwork puts even more pressure on smaller providers, who may not have dedicated administrative staff to handle it.

Only a fraction of the holdups that review boards initiate actually influence the final research design, according to the Vanderbilt study. Theodorescu argues that there should be one centralized board, like the one the National Cancer Institute has for its many hospitals around the country.

Whatever the cause, delays slow the research pipeline. They may also make it tougher to recruit patients.

“Many trials take so long to get started that they are asking questions that may not excite doctors and patients,” said Weiner at Georgetown.

There’s room for improvement at every stage of conducting a clinical trial, but scientists are nothing if not willing to whittle away at difficult problems.

We’re learning to do the best that we can with the resources that we have, whether those are our time, patients’ time, or money.
Dr. Gregory Masters, an oncologist at the Helen F. Graham Cancer Center in Newark, Delaware

“I’m not totally negative about it,” said Masters. “We’re learning to do the best that we can with the resources that we have, whether those are our time, patients’ time, or money.”

It will take dedication to keep cancer medicine moving forward and to get more patients involved, but it can be done.

“It’s a lot harder to discover a new drug for cancer than it is to [improve trials],” Theodorescu said. “It’s a question of political will.”

The previous story in this series examines why patients can’t always access the clinical trials that could save their lives. Read the previous article »