The importance of patients in the development of new medicines

Jun 9, 2017

As our knowledge of the way the human body works in a normal and disease state continues to evolve, medicines are becoming increasingly intricate and precise in their design. Thus, medicine is turning toward a more patient-centric approach putting the patient at the heart of clinical trials and therapy development.

Dr. Salim Mujais, Vice President and Medical Head of Immunology, Transplant and Infectious Disease Therapeutic Area at Astellas, spoke to Change Together about the importance of the patient in the development of new treatments.

Silent heroes

Patients hold the key to driving the development of new medicines – a role Dr. Mujais heralds as the defining factor in advancing healthcare:

“Patients are the silent heroes of our work. Without their participation, we cannot make our drug development real or with consequence and results,” he explains.

Dr. Mujais described the actions of patients who take part in clinical trials as “an extreme form of altruism” that supersedes the needs and wants of an individual to aid the wider population. In this regard, clinical research could not serve patients if they did not become a part of the overall development process.

“Patients contribute themselves to the process. We contribute work, hours and thinking, but ultimately, they contribute themselves to the research. This makes their contribution, from a humane perspective, the most important part of everything we do.” Drug development proceeds with patient and for patients. They are not mere subjects of drug development, but also have a motivational role. “We are all patients at certain times of our lives. We are bound together by the beautiful imperfections of life and as we alternate between the roles of drug developers and patients, we are inspired by the tenacity of the human spirit, and we are driven by the suffering of the collective that is us.”

Dr. Mujais stressed the importance of balancing the technical with the humanistic aspects of the work of medical scientists: “there is always a risk that our technical prowess may outpace our humanistic approach, and this reflects itself sometimes in the terms we use. One unfortunate consequence may be the blurring of the line around the concept of “personalized medicine”. It should mean that the patient, as a person, is the focus of our care, the individual with their hopes and life plan. Instead, it is currently used to refer to the genetic signature of the tumor that is targeted by therapy. The DNA signature of the tumor risks replacing the signature of the person.”

Evolution, not revolution

The term ‘personalized medicine’ has been at the fore of healthcare in the U.S. and around the world for some time now. It refers to a new wave of care designed to take into consideration all aspects of a person when treating and caring for them.

The phrase gained popularity at the turn of the century as the next step in healthcare. Yet Dr. Mujais doesn’t quite see it like that – instead, he believes it has been ever-present in medicine.

“The change is more evolutionary than revolutionary,” he explains. “Researchers have always designed trials based on stratification and enrichment, meaning only those patients who will benefit from the treatment will be chosen.”

In this new environment, clinical trials are now designed to couple the reality of the patient world with the optimum design and protocol to ensure the relevant scientific questions are answered, he adds. “If it doesn’t work in the patient world, then you start to get dropouts, deviations and incomplete data.”

According to Dr. Mujais, the key difference is the presence of “more refined techniques and better technologies, giving us the opportunity to better implement the concept of ‘personalized’ medicine.”

The importance of advocacy groups

Hand in hand with the patients themselves are the supporting advocacy organizations. While many consider patient advocacy groups and those involved in developing new medicines as distinct entities with polarizing ambitions, Dr. Mujais believes each group overlap in their interests relating to improving care and saving lives.

“By their very nature, advocacy groups are committed to the same end goal as researchers. Although they also defend patient rights on more fronts than just drug development, there are areas of overlap in which collaboration between us and them can be enhanced.”

Both groups’ end goal is to improve the number of people surviving their disease, added Dr. Mujais, and both play a part in achieving that objective but from different perspectives.

“Researchers want to create a drug that eliminates a disease and patient advocacy groups essentially want the same thing. From a human perspective, that’s what really matters.

“We greatly appreciate advocacy groups because we are not just researchers – we are also patients. Advocacy groups are advocating for us and in that regard, the communication between advocacy groups and pharma should be natural and expected.”

“We all play a role in the edifice of humanity, and enriching human lives is our common creed. Everyone working in the field of transplantation, for example, can ponder the intriguing possibilities that a kidney transplant could have prolonged Mozart’s life and a liver transplant would have lessened Beethoven’s suffering!”

The importance of patients in the development of new medicines

Astellas Patient Advocacy is a function within Corporate Affairs at Astellas that focuses on creating, building and maintaining third-party relationships. We serve as a conduit between Astellas and external stakeholders to help improve patient outcomes, improve access issues and address patients’ unmet needs head on.