IDEA Pharma’s goal is to help more great medicines reach more patients and, to do this, we must unlock the potential of each molecule through better decision making and collaboration.
But, what does this really mean in practice?
In my past life as a community-based physical therapist, I treated patients across the oncologic, autoimmune, and neurologic spectrum, and I was also fortunate to care for countless caregivers, parents, and family members also experiencing physical and emotional hardship. This lens colours my viewpoint on efficacy in many of the ways Mike pushes us to understand and improve.
There was “Monica,” a great grandmother with twice-relapsed, triple negative metastatic breast cancer. To her, efficacy reflected the love she felt for her husband, himself an 88-year-old war veteran who dedicated himself to her, night and day, doing what he could to ease her pain and suffering. To Monica, efficacy was the ability to sit long enough to have dinner with her soulmate, for she came of age as a nurse during war and knew the realities of her relapsed/refractory TNBC. Efficacy was not measured in days or months, but opportunities to return the love she received each and every day over 60 years of marriage to the man who tirelessly devoted himself to her throughout their youth, adulthood, and old age.
On the other end of the spectrum, was “William,” a three-year-old child fighting a form of pediatric leukemia. To his mother, efficacy was the opportunity at leading a normal childhood – the hope that, one day, this child would grow to meet his own soul mate and have a family of his own, a family that she and her husband struggled for years to conceive, only to be faced with the harsh reality of caring for a sick child so soon after experiencing the joy of finally beginning a family in the face of adversity.
How can we best align endpoints to reflect the diversity of strength shown through the most difficult struggles of those we care for?
The dichotomy of “efficacy,” I think, can be best explained through a simple comparison of two recently approved products in hematology. For the sake of comparison, both products were approved within 1 year of each other, and both products selected patients who progressed after at least four prior lines of therapy, with nearly identical descriptions of these lines within each label.
Product A: ORR: 61.8% | DoR (6mo): 90.6% | DoR (9mo): 66.5% | CRS (Grade 3): 0.6%
Product B: ORR: 97.9% | DoR: 21.8mo | CRS (Grade 3 or higher) 5%
Which product shows superior efficacy? Product B is the obvious choice. But what if we apply another filter?
Product A: can be given at any community hospital or oncology practice (1000+)
Product B: can only be given at a specialty academic centre (150+ in the US)
At this point, it should be obvious that, while Product B shows superior “efficacy,” it is bottlenecked by the system and environment of care, among other technical challenges. It should also start to be obvious that Product B is a CAR-T therapy, whose development should not be understated – CAR-T is a game-changer for medicine, both in terms of what it can provide to patients, as well as the attention it draws towards medical innovation as a whole. What is interesting, however, is that Product A is in the same portfolio, developed by the same company, and offers a different form of efficacy – the efficacy of access, the freedom to choose your oncologist, and the flexibility to stay closer to home, if that is your wish. In my opinion, this company understood something very early through the relentless search for optionality – how do we get more great medicines to more patients?
But, really, I ask – what is efficacy in human terms?
At IDEA, we’d love to hear what you think.
Jonathon Lee is a Consultant and Global Medical Strategist with IDEA Pharma, operating out of London, UK. He is also a US-trained Doctor of Physical Therapy, Board-Certified Orthopedic Clinical Specialist, and a Fellow of the American Academy of Orthopedic Manual Physical Therapists.
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