How CMOs can Champion Patient-Centric Drug Development
Panteli Theocharous, PhD, FRCPath, Chief Therapeutics Officer and Head of Medical and Scientific Affairs at Garuda Therapeutics, discusses how to build a patient centric clinical infrastructure by engaging patients early, collaborating with advocacy groups and appreciating the nuances of the patient journey.
How has your career journey helped you understand the needs of patients?
I came from a very strong clinical background working at the Royal Free Hospital in London, where I was part of the team that established the allogeneic stem cell transplant service. That gave me a lot of insight into how we really engage with patients: taking patients through a thorough and complex bone marrow and stem cell transplant procedure. There was a strong focus on building an infrastructure and understanding the needs of patients to be able to support them and place them at the forefront of our minds and practice. I’ve been able to extrapolate from those experiences and apply them to various roles I’ve held in biotech, big pharma and CROs.
What goes into building such an infrastructure?
It’s about understanding the needs of patients. There’s a level of intensity in how we think about supporting and guiding and influencing their journey by communicating and understanding their needs. It’s not only about the patient. We also think about the caregiver and the family unit – particularly in the pediatric setting where there’s a level of empathy that we need to showcase to patients in order to help them make the right decisions and provide each of them with a personalized and tailored approach.
What does patient-centric drug development mean to you?
Patient centricity is about tailoring our approach for patients across countries, regions and cultures. We need to listen to what patients are saying. We need to take that on board and refine and influence our own strategies from a very practical perspective by asking how we can optimize our approaches for patients. By developing the right tools, we can give the patient options and educate them accordingly about the journey they are going to embark on. By developing the tools to engage patients at the very early stage, we can support them through their journeys.
"It’s not only about the patient. We also think about the caregiver and the family unit – particularly in the pediatric setting where there’s a level of empathy that we need to showcase to patients in order to help them make the right decisions and provide each of them with a personalized and tailored approach."
How can CMOs best work with patient advocacy organizations?
It's about building relationships and having a roadmap whereby we can get buy-in. This is a partnership and it is about having the patient at the epicenter of everything we do and everything we say. We need to showcase that level of empathy and understand the hurdles and challenges patients face and integrate that into our development strategy.
Even if we have a clinical protocol, for example, it's great to be able to get the patient's voice integrated into the protocol. We want to understand their challenges and how we can mitigate those challenges from a practical perspective. We need to learn from the journey patients have already undertaken to refine and optimize our protocols. CMOs should engage patient advocacy groups to learn how to integrate their feedback to develop a strategy and protocol that makes sense and is practical and minimizes patient burden and site burden.
When are key times to engage with patient groups and how can you do that effectively?
In my experience, early engagement with a patient advocacy group can be highly impactful. In one instance, I had a strong relationship with the CEO of such a group an we refined a blueprint together on how we would partner. We presented it to my company’s board and the collaboration was endorsed. There are ways of thinking about the mutual recognition of how we work together, partner and collaborate and what is important for the patient advocacy groups and what is important for the sponsor side in terms of enrolling patients in clinical trials and getting them through the clinical trial process. By being methodical and highly collaborative in our approach, we can achieve a high attention to detail that supports everyone.
How is patient engagement different in cell therapies?
Within cell therapy clinical trials – like for an autologous CAR-T setting for example – it can be quite intense with procuring the cells through leukophoresis, having cells manufactured over the course of several weeks or months and then infusing the re-engineered cells. Patients can feel very anxious about waiting. We need to build engagement and support into the process to manage patient expectations and to try to mitigate that anxiety.
"Even if we have a clinical protocol, for example, it's great to be able to get the patient's voice integrated into the protocol. We want to understand their challenges and how we can mitigate those challenges from a practical perspective."
How can CMOs better put themselves into the shoes of patients and caregivers?
It all comes from exposure and interface with patients, investigators, key opinion leaders and other experts managing patients in the real world setting. We need to continue to educate ourselves and appreciate the nuances of the patient journey.
Then, we need to translate those experiences methodically into our own individual scenarios like in leading clinical development programs. We need to continue to raise the clinical bar and emphasize focus on the patient and take that journey together.
How is the culture of patient centricity different between the UK and US?
The patient journey in terms of accessing a clinical trial or even from a pricing and market access and reimbursement perspective are different. There are nuances around how we think about and appreciate and influence a process whereby a patient gets access to a therapy – whether it's standard of care or new technology.
Based on your CRO experience, what separated companies that excelled in patient centricity from those that did not?
Some companies engaged advocacy groups much earlier in their development paradigm in order to understand the patient journey, standard of care and where their particular asset or technology would fit within that paradigm. By asking the right questions of the right stakeholders at the right time, these companies were able to build a better clinical strategy. These companies had clinical protocols that were much more pragmatic and feasible because they integrated the voice of the patient.
"I would strongly encourage CMOs and companies not to wait for a challenge in enrollment to reach out but to rather engage early and preempt challenges to make the patient journey as smooth as possible. Patients are waiting and time is a critical factor for them."
Can you share an example from your experience?
I have been served well by building these relationships of credibility and openness and having clear goals and objectives around what we wanted to achieve through these partnerships. By really engaging with an advocacy group in the hematology space and building a blueprint around our partnership to raise awareness and be educated about patient journeys, we learned about and identified the hurdles and challenges for different patient populations early on and were able to circumvent them in a way that led to more efficient patient enrollment.
I would strongly encourage CMOs and companies not to wait for a challenge in enrollment to reach out but to rather engage early and preempt challenges to make the patient journey as smooth as possible. Patients are waiting and time is a critical factor for them. We need to get patients into a clinical trial as an option very quickly. We need to get treatments to patients as quickly as possible.
As a Director on a Board of a patient advocacy non-profit, how do you want biotech companies to engage with you?
Foundations and non-profit organizations allow us to be able to ask specific questions of patient communities and encourage a level of engagement that can open up doors for biotech companies. This allows biotechs to explore pragmatic and practical ways to help patients down the line. We all need to align around understanding what different approaches can mean for patients and how patients can potentially benefit.
Anything else?
As a community we really need to take advantage and come together to exchange ideas and learn and look for areas of collaboration and partnership to continue to raise the bar for patients.
