Dr. Kerry Cooper is currently the VP of U.S. Medical Affairs of Renal Cardio at AstraZeneca. He has been a nephrologist for 35 years, has extensive experience as a clinician and taking care of patients, and has been in the pharmaceutical industry for more than 10 years. What has always been a primary area of focus for Cooper is the proper care of patients.
“When it comes to clinical trials, pharmaceutical companies historically have focused on what the FDA needs to see to support approval,” says Cooper. “Registrational trials need to demonstrate that a treatment is safe and effective, and that it has an appropriate risk/benefit ratio. But we now know there can be a gap between what companies demonstrate to the FDA and how that transpires into the real-world setting. This is a challenge that pharmaceutical companies continue to look to overcome by continued evidence generation that can relate to their patient populations.”
Patients certainly care if a medicine is going to make them live longer or slow the progression of their disease. But they also care about how it will make them feel on a day-to-day basis. Consideration needs to be paid to how it will help with the symptoms of the disease, but also to how it will impact their ability to work, perform daily activities, and spend quality time with their families.
“Part of the challenge we face is going through the continuum of evidence generation to understand the impact on patients,” states Cooper. “Our major focus in the renal division of AstraZeneca is on chronic kidney disease. Our goal is both to manage the complications of chronic kidney disease today and ultimately evaluate the potential of treatments to slow the progression of the disease, keep patients off dialysis machines, and help them have a better quality of life.”
Think About The Patient Perspective
Two of the areas Cooper is focused on are anemia (low red blood count) and hyperkalemia (high potassium levels). AstraZeneca has a medicine for lowering high potassium levels, also known as hyperkalemia, called Lokelma. It does so effectively and has been proven to be safe to patients. Unfortunately, if you tell patients they need to manage their hyperkalemia, or lower their potassium, that does not really resonate with them. What they hear is they must go on a very restrictive diet which prohibits them from eating many foods they enjoy.
“For example, many people would consider a healthy diet to be one that includes fruits and vegetables,” notes Cooper. “Sadly, those foods can be very high in potassium. It can be very difficult to tell a patient they cannot eat them. When you talk to patients, especially those on dialysis, one of their biggest complaints is they can't eat in a way that makes them happy. Those eating restrictions can impact their day-to-day living as well as their social interactions.”
This presents a challenge to companies like AstraZeneca. They not only have to develop an effective treatment that lowers potassium, they also must help support patients, providing education and awareness of their disease and their diet overall. Learning how to do both involves reaching patients and activating them so they feel equipped to have discussions with their healthcare providers.
Those patient discussions also allow Cooper and his colleagues to better understand what types of outcomes are important to patients. Discussions with individual patients as well as patient advocacy groups are becoming an increasingly important part of designing research studies.
A New Approach
Anemia is a common consequence of chronic kidney disease and while there are available treatments, there has been very little innovation in recent years. AstraZeneca has partnered with FibroGen on an investigational medicine, roxadustat, which is part of a new class that treats anemia via a novel mechanism. Additionally, it provides a different delivery method compared to the current standard of care. Current therapies involve an injection that is given subcutaneously to non-dialysis patients or intravenously to patients on dialysis
“Roxadustat is an oral therapy that is under investigation for patients with anemia across the spectrum of chronic kidney disease,” says Cooper. “At the forefront, we are thinking about the needs of the patient. We are looking at how our medicine impacts the levels of red blood cells and how it can work naturally in the body to treat anemia different than it has been today but also asking ourselves what that means to patients.” If a patient is anemic, that generally means they will feel fatigued and tired. They will get short of breath easily and have little tolerance for exercise. Their ability to work and do daily activities may also be impaired.
For that reason, AstraZeneca is also looking at how their studies can quantify laboratory targets and endpoints while improving a patient’s quality of life. Cooper believes understanding patient perspectives is a critical component in evaluating the success of treatments.
“Patient feedback comes from many different sources,” says Cooper. “We get feedback from patients participating in trials, patients taking approved medicines, and patient advocacy groups. The best feedback we can receive from a patient relates to what we can incorporate into studies and strategies that will have the most meaningful impact on their lives. One of the best sources of that information comes from our patient advisory boards, which allow us to meet with groups of patients. We also perform market research with patients and through our regular interactions with advocacy groups, we uncover great insights that vary greatly across our respective disease areas to inform our plans.”
Understand What Patients Want
Patients taking part in a chronic kidney disease trial can also vary from those with a mild condition to those with more advanced symptoms. Some patients will have few (or no) symptoms while others will have severe symptoms and possibly even be on dialysis.
Some of the therapies being developed by AstraZeneca are being studied across the entire spectrum of chronic kidney disease. As a result, Cooper notes the therapies may mean very different things to patients at different levels of severity.
“We are very interested in things like how well patients tolerate the therapies,” says Cooper. “We want to understand their experience with it, the convenience of taking it, and even the taste and the palatability of the treatment. If it's an injection, we want to know if it stings and if it’s difficult to administer. We can’t only ask patients about their satisfaction with the therapy itself, we must also understand their experience using it.”
For example, AstraZeneca’s potassium therapy is not a pill. It is a powdered substance that comes in a small packet. It must be mixed with cold water in small amounts and ingested once per day over an extended period.
“Hyperkalemia is a chronic condition, so it’s important that patients prescribed this therapy take it on a regular basis,” states Cooper. “Therefore, we must understand its overall value for them, including right down to how they respond to taking it. If a therapy has a bad texture, bad taste, or is not palatable, they are not going to take it long-term.”
Real-world evidence gathered after a trial is also valuable to Cooper. He notes patients selected for a clinical trial tend to be from a specific population. This is due to the tendency of a clinician to select patients they know to be attentive and reliable. Those factors may make those patients more likely to adhere to the medicine than the general population.
Once a therapy is used outside the study, it is also possible to get feedback on the preference of one therapy over another, and how experiences differ with each treatment.
Monitor Patient Reported Outcomes
At the end of the day, what’s important to patients and physicians is how a therapy impacts patients, how patients access those therapies and how it makes them feel. For that reason, Cooper believes it is important to monitor patient reported outcomes. He also would like to continue to see payers and industry partners embrace patient centricity and innovative solutions.
“Pharmaceutical companies have embraced patient centricity, and one of the areas where we continue to see value is in how we partner with payers to help ensure access,” says Cooper. “For example, if there are two therapies approved for treatment of a disease, one might be markedly preferred by patients over the other for a variety of reasons. However, access to those medications may vary from payer to payer. We recognize the need to work with payers in this regard to identify how we can bring our therapies to patients. In many cases, this means identifying innovative strategies with payers that might share risk across both the payer and AstraZeneca to bring the greatest value and preference possible for our patients. This can help ensure that patient preference, or patient choice, does not factor into the decision-making process versus cost alone. That is something we see a great opportunity in across our therapy areas and in particular with treatments for renal conditions, especially if we want to ensure patients take their medicines regularly.”
When creating therapies, pharma companies must take care to ensure the treatment meets the needs of patients. For that reason, Cooper would like to see patients become more active decision-makers in the therapies they use.
“AstraZeneca has made a commitment to the unmet need that exists for patients with chronic kidney disease,” adds Cooper. “This is an epidemic in the U.S. and around the world. The morbidity and mortality rates are as high for chronic kidney disease as they are for cardiovascular disease and cancer. We are focused on innovating in this space with important developments in anemia, potassium management, and diabetes, which is the most common cause of kidney disease in U.S. Our hope is that our research and developments will be extremely impactful to patients with this disease.”