Why you should care

A faster drug development process could mean a more democratic one. 

Bonnie Addario didn’t see it coming. A “perfectly happy” mother of three and president of a San Francisco Bay Area oil company, she suspected something was wrong when she began feeling a constant, shooting pain in her chest. After a year of seeing countless specialists, two doctors finally diagnosed her a week before Christmas: advanced-stage lung cancer. The tumor had migrated from her lungs and engulfed her aorta. Without chemotherapy, she would die in three weeks.

Addario learned that lung cancer survival rates had barely budged since the 1970s. Stunned, she began searching for “holes in the system” of drug development. For one, it moved at a lumbering pace, with most drugs taking 10 to 15 years to develop and only 3 to 5 percent of adults participating in clinical trials. As an IV dripped chemo drugs into her veins, she vowed that if she survived, she “would do something to change this crazy, crazy process.”

Whether you are gravely ill or not, who isn’t frustrated with the way Big Pharma and the government control the rollout of crucial health care treatments? But it turns out that frustration may actually be helping to streamline the most Kafkaesque of systems — and it’s the patients who are behind it. The No. 1 strategy is decentralizing the whole thing, so you and I can be a part of the process. Think open-access, collaborative approaches — to make it easier, say, for patients to enroll in trials or academics to work with industry scientists — something Addario has been involved in since being declared cancer-free.

Clinical trials stem from a 1962 amendment to the Federal Food, Drug and Cosmetic Act that required drugs to be proven not only safe, but also efficacious, as demonstrated in a “well-controlled trial.” Researchers randomly assign participants to receive either the drug or a placebo and compare the results. “It’s a concern that other methodologies are not as rigorous,” said Scott Podolsky, director of Harvard University’s Center for the History of Medicine.

Not that the system doesn’t have flaws. Clinical cancer trials are typically conducted in states with large, well-funded, comprehensive cancer centers — and patients need to complete consent paperwork at the trial site to determine whether they even qualify. This could mean traveling thousands of miles. So the Addario Lung Cancer Medical Institute launched an online consent system as part of the Genomics of Young Lung Cancer Study. Interested individuals sent an online form to the lead researchers, who offered or denied consent to participate. Addario envisions clinical trials relying on a similar system.

Some also blame the current clinical trial model, which tests only one drug at a time. Addario’s organization has begun enrolling patients for the four-arm Lung-MAP study, which will include 500 to 1,000 advanced-stage squamous cell lung cancer patients. Scientists will screen their tumors for modifications in 200 cancer-associated genes and assign them to the trial arm investigating the drug developed to target them. And rather than test all the patients at one cancer center, Lung-MAP will distribute them among 200 community hospitals.

Scott Johnson committed to streamlining drug development after his own struggle with an incurable disease. When he went blind in one eye while in college, a doctor diagnosed him with multiple sclerosis — when myelin, the protective tissue that encases the nerves, breaks down over time. After 25 years of ignoring the numbness and memory lapses, he stumbled on an article about scientists’ efforts to repair myelin, rather than prevent the immune system from destroying it. Maybe this new approach held the key to a cure.

a photo of cultured lung cancer

Cultured lung cancer.

Source Anne Weston/Corbis

But then Johnson learned about the lack of coordination among academia, pharmaceutical companies and funding agencies, slowing drug development. So in 2004, he started the Myelin Repair Foundation, a nonprofit organization that fosters collaboration among these stakeholders. To help academics fast-track their discoveries to cures, an advisory board of pharma researchers reviews the potential drug targets they’re studying, identifying the most promising candidates. The experiments are then repeated thousands of times using sophisticated equipment so that companies trust their discoveries enough to develop them into drugs.

Free the Data allows patients to set their own sharing preferences.

Still other patients have literally opened up the system. After Andrea Downing learned that she carried a BRCA1 gene mutation, linked to ovarian and breast cancer, she kept her status secret for years — until she saw a friend suffer through stage 4 breast cancer. She then began blogging about her double mastectomy and became a spokeswoman for a plaintiff in the Supreme Court case deciding whether genes could be patented.

Myriad Genetics had patented its discovery of BRCA1 and BRCA2, preventing other researchers from studying, analyzing or examining them. (A representative for Myriad said the company has always encouraged and supported research around these genes.) Shortly after the Supreme Court ruled against Myriad in 2013, though, a group of medical institutions launched the Free the Data initiative, allowing patients to upload their BRCA reports to a database. Although some, like Podolsky, worry about patient privacy, Free the Data allows patients to set their own sharing preferences. As a Free the Data steering committee member, Downing questions how patients “can drive the agenda of how that data gets used,” she said.

Patients have taken the reins to make drug development about them again. “Patients are a natural resource in this whole equation,” Downing said. “We have to have a seat at that table.”

*Correction: An earlier version of this article misstated the states where clinical cancer trials are conducted.

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