How patient advocacy can lead to more successful and meaningful clinical trials

From the sponsor that designs the protocol and the regulatory body which approves it, to the patients recruited for experimental treatment and the trialists who evaluate them, a clinical trial is a vehicle with many moving parts that achieves optimal speed when each party plays its part. 

Patients and advocates play no small role in driving treatments to the market, with those voices lobbying for the US Food and Drug Administration (FDA) approval of Amylyx’s amyotrophic lateral sclerosis (ALS) treatment Relyvrio, citing the high unmet need of treatment options for patients despite the treatment’s modest efficacy data, safety risks, and negative FDA AdCom opinion. 

On Clinical Trials Day, Clinical Trials Arena sat down with patient advocate and Global Head – Growth & Insights at Savvy Cooperative Kelly Franchetti to discuss how patient advocacy has evolved to drive patient centric clinical trials and where the relationship between patients, advocates, industry, and agency needs to be to advance drug development.

Kelly Franchetti: Patient advocacy has played a more active role in clinical trials compared to 20 years ago, and many factors contribute to this evolution. Patients, as consumers, are becoming much more savvy, and advocacy recognises and is reinforcing that trend. It also helps that pharma and regulatory bodies are actively acknowledging the importance of bringing advocacy early and often into the conversation when it comes to clinical studies.

Kelly Franchetti: Absolutely. I think there’s a direct correlation with not only trial termination and dropout rates, but also with the number of trials that enter “rescue” situations. With patient advocacy at the periphery, trials can lose sight of patient recruitment and retention, and as a result, find themselves having to address these patient centric aspects of a trial much further down the line. The emphasis on the patient has improved over the years, getting patients and advocacy involved as early as possible in the life cycle of a trial. And it is important to distinguish patients from patient advocacy, both of which need seats at the table, because patient advocacy represents the greater voice of patients as a collective while a patient represents the individual. A clinical trial is an ecosystem that is optimal when advocacy and patients are the first line to discuss how a trial will be operationalised and what measures will be used for evaluation.

Kelly Franchetti: The commonality that holds all these groups together is the dedication towards stopping or changing the trajectory of a disease and bettering patient lives. Where the disconnect comes in is that each of these groups have different primary goals. Pharma’s primary goal is very different from the patient’s primary goal, which is different from agency’s primary goal, especially when it comes to the design of a clinical study. Endpoints, in particular, can be a source of commonality but also be the driver of dissonance. I don’t know that the regulatory bodies have caught up to where patient 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.

Kelly Franchetti: 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 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.

Kelly Franchetti: 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.