Brandon Downey, 32, a nurse in Chicago with severe hemophilia A, has switched treatments several times over the last decade. He was previously on one medication but switched to something different, because it reduced the number of days he had to infuse. When a new drug crossed his radar, his doctor wrote him a prescription. But it didn’t work as well as he expected.
So when a friend told him about his experience with yet another new drug, Downey asked to try that. He—and his doctor—have been pleased with his health ever since.
“I like to weigh the pros and cons of every single product and come up with the very best decision I can,” says Downey, who sees his doctor as his “best practice advisory council.”
Downey is a case study in shared decision-making, an approach to treating diseases in which patients are partners in their own care. This concept is based on the idea that a patient’s lived experience, personal preferences and lifestyle are as important to plotting a course of treatment as a provider’s knowledge of the treatments themselves.
“In the past, the physician said, ‘Here’s the treatment, here’s what we’re going to do, I’ll give you a prescription, and you go fill it,’” says Leonard A. Valentino, MD, president and chief executive officer of the National Hemophilia Foundation. “Shared decision-making is a process by which a provider and an individual have a dialogue around what the therapeutic options are, including the risks and benefits of all of those. It’s the first step in a fully informed treatment process.”
“It’s taking into consideration the doctor’s insight, but also the patient’s input,” adds Maissaa Janbain, MD, MSCR, associate director of the Louisiana Center for Bleeding and Clotting Disorders and assistant professor of hematology/oncology at Tulane University School of Medicine. “We want to individualize therapy and tailor it to the patient’s needs and lifestyle as much as possible.”
So Many Treatment Options
The influx of game-changing treatments for hemophilia is responsible in part for this paradigm shift. Extended half-life products allow people to do well with fewer infusions, reducing doses from three to two a week for people with hemophilia A and from twice to once a week or less for people with hemophilia B. Monoclonal antibodies allow some people with hemophilia to transition from IV infusions to subcutaneous injections as infrequently as every four weeks, depending on a patient’s condition and needs.
“Patients now have choices,” says Mindy Simpson, MD, medical director at the Rush Hemophilia and Thrombophilia Center in Chicago and assistant professor of pediatric hematology/oncology at Rush University Medical Center. “It’s not one-size-fits-all anymore. We’ve really hit a world of personalized medicine.”
All of this requires collaboration. “What we’ve learned is that there are a lot of good options, but there’s not necessarily a perfect one for all patients,” Simpson says. “So we all need to be open to having conversations about what the options are, and what we think that might mean for the patient.” She routinely asks patients if they’re happy with what they’re on, if there are things they would like to do differently, and if they have products in mind.
“I want to know, ‘What does your daily life look like?’” says Brittany Savage, NP, a bleeding disorders transition nurse practitioner at the Indiana Hemophilia & Thrombosis Center in Indianapolis. “‘What do you do for work? Do you play sports? Are there certain days that you work out?’ We look at that and recommend certain treatment days or treatment options.”
Shared decision-making often comes into play with adolescent athletes, particularly elite high school athletes hoping to compete at the college level. Often they want to infuse less frequently, which extended half-life products allow them to do. Yet infusing more frequently with standard half-life products could help maintain their factor levels better, protecting them from bleeds due to the wear and tear of their sport.
“I recently met with a patient who plays water polo and is on every-14-day infusions, and he loves that schedule,” Simpson says. “I said, ‘Let’s think about a once-a-week schedule on a short-term basis during your season, then the rest of the year you can go back to your every-other-week schedule.’”
“He said he would think about it,” Simpson adds. “I took that as a win.”
The give-and-take between providers and patients—which is vital for all people with bleeding disorders—will be particularly important when innovations under investigation, such as gene therapy, hit the hemophilia market. This groundbreaking treatment, which holds the promise of a cure for hemophilia, is currently in phase 3 trials, the last step toward FDA approval.
“Now you’re potentially faced with a one-time opportunity,” Valentino says. “It’s a much bigger decision. And who are we to decide for a patient, if this is going to last five years or 10 years, maybe a lifetime? Who are we to decide for that individual when it’s the right time for them to do it?”
The Benefits of Shared Decision-Making
The payoff of this approach to care can be profound. “When a patient is able to explore and compare treatment options, to provide their input into what they value and what their preferences are, and ultimately reach a mutual decision with providers,” Valentino says, “it’s much more likely that they are going to adhere to that treatment and truly be satisfied with it and their outcomes.”
As Roshni Kulkarni, MD, professor emerita and director of the Center for Bleeding and Clotting Disorders at Michigan State University, puts it, “If you don’t have buy-in, you will get nowhere.” In fact, research suggests that patient involvement in the treatment process reduces bleeding and improves quality of life.
In the shared decision-making model, doctors offer information on treatments, including how they work, how often they’re administered, and the side effects. Ideally they leave their biases at the door. For instance, doctors may be more likely to recommend a particular therapy if they have more experience with it, or maybe even studied it in clinical trials.
“Hopefully we lay out in as unbiased a way possible those options for people and introduce new therapies as they become available,” Valentino says. “And we’ve even started to talk about some of the products that are currently in a development phase, so that people become more aware of them. Then it’s not a surprise when it becomes an approved product.”
Physicians try to provide a more balanced view of a product than a drug advertisement or another source does. “I want patients to hear about it, if they haven’t, or to get my impression apart from other sources of information,” Janbain says. “I feel like it’s my duty to share with them the scientific points of view, free from conflicts of interest.”
If a therapy’s cost is an issue—another factor in the decision-making process—providers may be able to recommend resources that patients and caregivers are unaware of.
The Patient’s Role
Engaging with the physician may be an unfamiliar role for some patients, but it can help them manage their condition more effectively. “I ask my patients to write down whatever they are concerned about or any other thoughts that they have. It will lead to a more structured and a more fruitful discussion,” Janbain says.
Patients will want to know how to store the product and other practical issues that may be involved. This helps prevent them from being surprised or worried, which could affect their adherence to the treatment, Janbain says.
Even if someone is doing fine with their current regimen, it can’t hurt to learn about all the new options that are opening up. “Some people with hemophilia get on something that works for them and they don’t want to switch because they don’t want to go through the ups and downs while switching,” Downey says. “But they should know that there are things out there that can improve their life.”
Overall, says Downey, “I feel like the wisest minds take information from multiple places and analyze it. And shared decision-making is definitely that.”