CLINICAL DEVELOPMENT IN ONCOLOGY
“Innovation will take strides when what is scientifically considered significant and meaningful change crosses paths with what patient populations deem as meaningful change”
populations need them to be when discussing endpoints. Pharma may recognise some patient needs within the endpoints, but we ultimately don’t really get movement towards addressing those needs. And this is especially visible in oncology and rare disease because regulators establish endpoints that pharma is obligated to meet but does not align with what is meaningful change for a patient living with that condition. Post regulatory environments are also
where individual patient perspectives are integral because a patient can shine a light on payors, insurance, and individual struggles in a way that advocacy, which addresses the broader grouping, cannot. Having all parties at the table creates a more comprehensive and well-rounded picture. The connection between patients, advocacy
groups, pharma, and regulatory bodies is currently disparate, and we haven’t quite figured out the perfect formula for this relationship. The common ground is there, but the conundrum is building a relationship that can address each party’s primary goals.
JR: How are patients and a patient centric approach driving innovation in pharma?
KR: Patients are driving innovation by constantly being involved, giving their opinions and feedback. Innovation on the pharma side is actively bringing those patients in to
36 | Clinical Trials in Oncology
incorporate patient feedback and develop methodologies that address patient needs. But moving the innovation needle is a work in progress that requires further momentum and goal alignment, especially by regulatory groups. For example, in a clinical trial for a rare disease like Duchenne muscular dystrophy (DMD), there are definitive endpoints that must go into studies, one of which is a five-minute walk test. Now if you talk to a parent of a child with DMD, they might say the biggest meaningful change resulting from treatment is that my child, who was not previously able to use a spoon, is now able to eat from a spoon. But this is not an endpoint that the US Food and Drug Administration (FDA) considers as meaningful change. Innovation will take strides when what is scientifically considered significant and meaningful change crosses paths with what patient populations deem as meaningful change. The desire to move towards this point is apparent, but we are definitely not there.
JR: What are some of the trends in patient advocacy in the US compared to outside the US?
KR: From a rare disease perspective, patient advocate and advocacy groups are very active both in the US and outside the US. The US does have more advocacy involvement in some of the bigger indications, but that seems to be a direct reflection of healthcare in the US versus abroad. Outside of oncology and rare disease indications, advocacy is less visual abroad in the sense that in the US, the primary reason for some of these big advocacy groups is policy change in the overarching health care system, with clinical trials simply being an aside. There are more offerings in Europe that drive US advocacy constituents to plug into advocacy in those areas, especially for rare disease and oncology communities. We have so many advocacy groups in the US, some small and other corporate. And these smaller groups, especially the ones advocating for orphan diseases that are continuously emerging, are not able to do much because they lack the resources and support. So support for these groups is something that needs to change.
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