We often prescribe new medications that were proven to be effective in rigorous clinical trials and were approved by the Food and Drug Adminstration only to find that our patients don’t get better.

Amazon tells us every day which books would interest us, and Google can chart the fastest routes to our destinations, because these companies have mountains of data on which to base their predictions. We could provide better care for our patients if we had information that would allow us to target treatments to individual patients. But we don’t. Doctors and researchers need clinical trial data to make this happen — and that is hard to come by.

Take gastroenterology, which two of us (A.K.W. and S.D.S.) specialize in, as an example. Some of the newest medications for ulcerative colitis — a debilitating, lifelong illness — are miracle cures for some patients, have no effect for others, and even harm some people. To find the right medication, gastroenterologists must run their patients through a list of costly medications, in essence treating them like guinea pigs. They do this because they simply don’t have access to the data needed to help identify the right medicine that might work for a specific patient.

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Why not? When researchers conduct clinical trials and publish their results in peer-reviewed journals, doctors have faith that those results are credible and accurate. Yet such studies traditionally provide only a summary estimate of benefits, such as “30 percent of patient improved with drug A.” What this information does not tell us is which 30 percent responded. Information about individual patients, like their age, gender, or lab results, can sometimes help predict which patients will benefit from a drug. Sadly, pharmaceutical companies have few incentives to release this type of information and may even have business reasons to keep the data generated by their research investments secret.

Pharmaceutical companies have recently begun releasing data like this to researchers under a movement called Open Science or Open Data, but the process is slow and seemingly designed to frustrate. You first submit a request that makes a case for the data. Then you wait. And wait. And wait some more. The request goes through a regulatory review process and negotiations between your university and the pharmaceutical company before it’s given a thumbs up or down.

Third-party organizations like the Yale University Open Data Access (YODA) project, launched in 2011, have become additional platforms to access clinical trial data. As groundbreaking as this platform has been, it has miles to go. Currently, only a very small number — 31 — of drugs have clinical trial data available on YODA. To put this in context, more than 200 drugs have been approved by the FDA’s Center for Drug Evaluation and Research since 2013.

In short, there are roadblocks every step of the way for getting access to the data that doctors like us need to move from population-focused medicine, which relies on the average estimate of benefits, to precision medicine, which aims to use patients’ genes and traits to match them with drugs that will benefit them. We need more information, we need it for more medicines, and we need to make it simpler for doctors and researchers to access. Doing so will allow us to tailor therapies for our patients.

Tailoring therapy is good medicine on a variety of levels. It limits costs, which are skyrocketing for pharmaceuticals across the U.S. One year of the newer drugs for treating ulcerative colitis, for example, can cost upward of $5,000 per month. Some patients pay most of this out of their own pockets. Yet these drugs aren’t effective for many who take them. Imagine taking a medication that costs $60,000 a year just to find out it doesn’t work for you.

Tailoring therapies also limits harmful side effects. Some of these are relatively minor, such as nausea, itching, and rash. But others, such as liver problems or cancer, are serious. Some of the newer gastroenterology drugs produce unwanted side effects in almost a third of patients. If doctors had full clinical trial data at their fingertips, they could look at which patients were most likely to experience a particular side effect, and what characteristics made that more likely to happen.

Most important, tailoring therapy helps patients return to health as quickly as possible, medicine’s main goal.

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Clinical trial data can improve everyone’s health. Pharmaceutical companies should share this information because federal tax dollars often underwrite a portion of their study costs, and because there are clear ways to reveal the data while protecting the anonymity of patients.

But if pharmaceutical companies won’t widely share their data and methodologies because it’s the right thing to do, federal mandates should require them to do it.

Akbar K. Waljee, M.D., is an associate professor of gastroenterology, Sameer D. Saini, M.D., is an associate professor of gastroenterology, and Brahmajee Nallamothu, M.D., is a professor of cardiology at Michigan Medicine. All are members of Michigan Medicine’s Institute for Healthcare Policy and Innovation, the VA Center for Clinical Management Research, and staff physicians at the VA Ann Arbor Healthcare System.

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  • I read the article above with the greatest interest. I would like to make two comments:
    -RCTs are the gold standard to establish inference between a treatment and an effect. They are regulatory driven and are not designed to help decision making for a particular patient
    -On the other hand of the spectrum is real world evidence (registries, comlections of medical records, claims data). These data are currently underused by clinicians for questions of accessibility and costs. This reasource is however the key to achieving targetted treatment and patient-centric medicine because they are able to point out the determinants of a particular treatment scheme success while computing its probability.

  • The Healthcare Industry has promoting a false narrative about data, data driven research, and digitized medicine. No one is asking what happened. The Tech Industry has harvested trillions of data points on patients to use for nefarious purposes, like profiteering and misleading the public. The data is inconvenient, it could lead to “liability” or cut into drug, medical or insurance industry profits. Lobbyists told Congress that even collecting Data on Medicare, the largest pool of Americans was “government overreach.” The Lobbyists are hard at work, misleading the public, Doctors, and policy makers on what data they collect and how it is used. Any data that could interfere with profits, is not collected or available. They have infiltrated academia too. No one noticed how pharma funded Science, proprietary laws, corporate lobbyists and the tech industry redefined data. In healthcare the only readily available data, is gleaned from billing records. There are different, reporting requirements and schemes designed to keep us all in the dark. For example the “Opiate Epidemic” a fourth of those deaths are due to other pharmaceuticals, yet they are attributed to opiates. People that die in hospital have their deaths attributed to underlying conditions. No one is looking at how the data is collected and why.
    The Fix is In, people. Anyone who exposes adverse events from a pharmaceutical is subject to a Lawsuit, so no research will be done. They are Gaslighting us all. WHy are they drugging those immigrant children in those Foster Homes? They have been doing it to low income and minority American kids for decades. There is no research there. It was inconvenient for the drug companies to do long term studies, or even collect data. Physicians went blindly along, as long as it was advertised, or profitable.

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