Choosing Trials as Treatment: Lessons from Parenting
We all want the latest, newest thing, right? In fact, as I am typing these words, I look at my iPhone 6, kind of wishing that I had the iPhone 7, and I don’t honestly even know why! But I know it’s newer, so it must be better. The newer version often offers a completely new feature or an upgrade to old features that we already like. The exploding Samsung Galaxy aside, new phones usually offer new benefits with little new risk. We tend to apply the same mentality to new drugs, but thankfully the clinical trial system is in place to protect us from drugs that are dangerous or just not that effective. (That Samsung Galaxy would never have gotten through the FDA!) While using a trial as a treatment strategy is becoming more common, especially with highly specific, targeted drugs, it is still a decision to be made with more consideration than an iPhone upgrade.
The FDA As a Watchful Parent of a Growing Child
The clinical trial system was established to make sure that new drugs aren’t brought to market until they have been proven both safe and effective. Initially, the FDA regulated the process in a very paternalistic manner, making sure that the system was designed with the highest level of patient safety in mind, and always careful to avoid giving any false hope to patients. I tend to equate the trials process to parenting. Like the FDA, I want to protect my kids, and the easiest way to keep them safe is to restrict everything. My toddlers were unlikely to choke on grapes if I cut them into quarters first; as elementary schoolers, they will be the safest if they’re not allowed to walk to the library without me. Being very restrictive is the simplest way to maintain a high level of safety.
As my kids grow, I want to keep them safe while being less restrictive, and it takes a lot more work! I have to teach them how to be safe and watch them from a distance as they begin to venture out on their own. I want my kids to have access to all the good things our neighborhood has to offer, so I have to do the work and give them the tools to stay safe and make smart decisions. It turns out, the FDA finds itself in much the same position. Keeping patients safe is easiest if the trials are very restricted. In cancer trials, while everyone has cancer and patients don’t get a placebo, it’s still easiest if only the “healthiest” of the cancer patients and those without any treatment options participate in a trial, and it’s easier to maintain rigid standards if all related procedures are done at one facility. To ensure that patients fully understand the risks, the consent language is heavily regulated so that any possible benefits are de-emphasized. That makes it harder and scarier to enroll, but it does keep more patients safe from unexpected adverse effects.
The unintentional effect of that practice, though, is to exclude patients who could actually derive real benefit. In fact, more and more trials are showing patient benefit much earlier, and allowing patients to choose them as a treatment strategy could make a real difference in the length and quality of a cancer patient’s life.
Venturing Out: Letting the Patient Take the Lead
Much like the strategies I established to give my kids more freedom to make their own decisions while still staying safe, the FDA is beginning to find ways to allow broader participation in trials without compromising the integrity of the system established to judge whether a drug is safe and effective. Without explicitly stating as much, programs like Breakthrough Designation and Accelerated Approval, as well as the hard look they are taking at modernizing eligibility criteria, show a recognition of the fact that there are many drugs in the clinical trial process that could provide substantial benefit to patients, and they need to increase access without all of traditional restrictions.
Though patient safety will always be of utmost concern to the regulators at the FDA, they are finding ways to do the work to make sure that including more patients in clinical trials is done safely, with the patient being a fully informed partner in the decision making process.
At some point, I had to take a deep breath, confident that I’d given my kids the tools they needed to make safe, smart decisions. I sent them off to the library without me, with a clear understanding of the risks and how to mitigate or respond to them, and they enjoyed the benefits of their new-found freedom.
Patients, too, can be armed with a clear understanding of the potential risks and benefits of a clinical trial and then given the freedom to choose their treatment strategy. Much like my tween-aged kids, patients are at an exciting crossroads where they must cautiously weigh the risks to reap the potential benefits of something new.
Jamie Holloway is a both a scientist and a survivor, earning her PhD in tumor biology from Georgetown University a few years before her own breast cancer diagnosis. Now living with no evidence of disease after treatment for early stage triple negative breast cancer, she bridges the gap between scientists and researchers as a Precision Medicine Advocate for Cure Forward and as the Patient Advocate for the Metastatic Breast Cancer Project at the Broad Institute. She works with researchers as part of the Georgetown Breast Cancer Advocates and writes about her personal experience with cancer on her blog, Run Lipstick Chemo, and as a contributor to the Cure Magazine community.