Centering the Patient in Plasma-Based Therapy Development at Takeda
Kristina Allikmets, MD, PhD, shares how Takeda is bringing the patient to the forefront as they develop therapeutics for chronic and rare diseases. She is SVP, Head of R&D, Plasma-Derived Therapies Business Unit, at Takeda Pharmaceuticals.
How are you centering the patient in your plasma-derived therapies work?
We put the patient at the center of the work we do on a daily basis. Delivering plasma-derived therapies, or PDTs, is so much more than making the product available for the patient. Any decision we make must always benefit the patient and help us serve them better, and to do this we include the patient voice early in the drug development process. This way, we learn about patient needs are from the patients themselves, which is a huge driver of much of the work we do within the PDT R&D organization.
How are you connecting to patients?
We have long-standing relationships with a number of patient groups, we reach out for input from individual patients, and we have patient advisory boards. These interactions are not just for patient stories, but to understand the patient journey, which informs how products are developed and what products are needed. For the drug development process, we learn by talking to patient organizations or individuals to understand more about challenges they face, for example, around side effects or administration.
PDTs typically treat chronic diseases; they are not usually a one-off therapy. Understanding where the need is, understanding exactly the burden of the disease for the patient, and hearing patients describe how they manage their disorders is important. Our interactions with patients not only impact our drug development, but they also remind us why we do the work we do in the first place: to improve the lives of people with rare and complex chronic disease.
"Understanding where the need is, understanding exactly the burden of the disease for the patient, and hearing patients describe how they manage their disorders is important."
Can you give an example of how patient feedback changed what you now do for patients?
Administration can be a challenge for our patients – particularly the volume some therapies require for treatment. But we heard from patients that at-home administration was important to them, as that would allow for more flexibility in their treatment schedule. To meet this need, we developed formulations for some of our therapies that could be used subcutaneously, which allows for at-home administration.
How else are you catering to specific needs of patients?
We learned that while patients and caregivers appreciate the opportunity for at-home administration, it can be complex logistically to do it yourself. Administering PDTs often requires needles, tubes, different vials of liquids – and all of these components require effort. We’ve been working to develop a device to simplify the process, by reducing the number of steps for patients or their caregivers to administer the therapy. It’s still a needle-stick and infusion, but we can create a special technical device that eases the process of getting the drug from the vials into the pump, which could ease some of the complexity.
"Our interactions with patients not only impact our drug development, but they also remind us why we do the work we do in the first place: to improve the lives of people with rare and complex chronic disease."
What is it about Takeda that drives patient preferences?
It comes from the guiding principle for us in Takeda and of course in PDT as well: we work in a sequence of patient, trust, reputation, business. Every time a decision is made, the first question we always ask ourselves is, “How would that meet the needs of our patients? Does it build trust with the patient community, the healthcare community?” We assume that if we do the right thing for our patients and have the trust of the community, then the business will follow.
So, with the example I gave of at-home administration: this was a very logical decision to make when asking ourselves how best to serve our patients. With lifelong therapy – and many patients having a diagnosis very early on in their lives – at-home administration is a very big need. We have heard from families where several of the children have primary immunodeficiency diagnoses, and we see it as our promise to patients and caregivers to development treatment options that support their ability to treat and manage their disorder while also leading active lives.
"For the drug development process, we learn by talking to patient organizations or individuals to understand more about challenges they face, for example, around side effects or administration."
What do you appreciate about working with patients in rare disease indications?
In rare diseases, it's not the knowledge from medical textbooks that matters. It is directly interacting with and learning from the patient community about specific disorders. Some diseases we work with are ultra-rare. It's impossible to know what the patient needs are and how the diagnosis is made without learning from patients and their caregivers. We also have to ensure that we put our utmost effort into raising awareness of particular rare diseases with healthcare professionals so they know to watch for symptoms when they occur.
Additionally, when emerging therapies become available for rare diseases, there isn’t always a clear regulatory pathway. To get to a registration, you need to first learn from patients to identify endpoints that are clinically relevant, and then work with regulators to ensure that the program meets the rigors of registration.
What is one thing about how patient engagement gets done in pharma that you’d like to see changed?
I would like to see more consistency and a formal process for patient engagement to become part of the DNA of an organization and implemented consistently across all departments. That is what Takeda has tried to do. We have a document called the Patient Engagement Plan, which captures the activities for a particular development program to follow. That includes when to reach out to patients, how to reach out and who to reach out to during the development of the drug.
We want this to start in the very first stages of drug development. I think having a more structured approach is what helps, because then there's no chance of forgetting this step. The patient voice is built into the process from day one, so every decision being made throughout the drug development journey considers the needs of the patient, as told to us by the patient.
What do you find valuable about participating in the Patients as Partners community/conference?
This is an authentic and meaningful opportunity to come together with other advocates for patients to learn not only from the patients, but from each other. The more we share about our experiences, our approaches and programs, the better integrated the patient voice will be in medical care overall.