Most drug evidence is built on the wrong patients.

Sponsors need to understand how their therapy performs in the real world. But patients seen at academic centers and enrolled at research sites aren't representative of the people actually on the drug.

Patients managing complex chronic conditions are receiving care across multiple community providers, often for years, without generating a single data point that a site-based drug registry would capture. The specialist visit, the academic center, the enrolled site: these represent a fraction of where treatment actually happens.

This is not just an enrollment challenge. It is a population validity problem. If the patients who are unreachable through the recruitment model are systematically different from those who are reachable (in disease severity, comorbidity burden, adherence behavior, access barriers, or care patterns) then the evidence the study generates reflects a selected subset of the treatment population, not the population itself. That gap does not close with a larger site network. It closes with a different access model.

The disconnect is not random. For therapies distributed through specialty pharmacies (SP) or supported by manufacturer patient support programs (PSP), the channels where most patients engage with their care are precisely the channels that most evidence generation programs are not designed to access.

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Why SP and PSP channels are central to evidence generation

SP and PSP channels reach highly qualified patients who are already on therapy and representative of the full treatment population. For most complex chronic and rare disease indications, these are the channels where the treatment relationship actually lives. These channels educate and support patients throughout their care, and can invite them to participate in studies near therapy initiation.
That timing matters. A patient enrolled close to treatment initiation can provide accurate exposure capture from the start, a complete baseline, and the kind of longitudinal follow-up that retrospective assembly from claims or EHR data cannot reconstruct after the fact.

Additionally, patients who learn about a study through a trusted source are more likely to consider participation. Enrollment moves faster. Cohorts are more diverse. And the startup timelines and site contracting overhead that make traditional registries expensive to stand up are largely removed.

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How enrollment gaps undermine payer, HTA, and post-marketing submissions

A study that underrepresents community-treated patients produces evidence with a known structural limitation — one that payer reviewers, HTA bodies, and regulators will find. In its December 2023 finalized guidance on registries and regulatory decision-making, the FDA explicitly identifies participant representativeness as a critical factor for preventing bias, and requires sponsors to assess whether their enrollment methods may have compromised the representativeness of the study population before submitting that data in support of regulatory decisions.

Label expansion filings depend on characterizing patients who may be managed very differently than trial participants were. PMC and PMR fulfillment requires long-term safety and effectiveness data across the treatment population as a whole. In both contexts, a study built around site-based enrollment is generating evidence with a structural gap that reviewers are equipped to find.

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SP and PSP integration is an evidence generation design decision

One of the most consequential evidence generation decisions a sponsor makes is which enrollment channels the study is built around. The data systems, consent frameworks, and patient engagement infrastructure required to reach those channels have to be built into the study design, alongside digital outreach, advocacy communities, and site-based referrals.

That is the structural choice. And it determines not just how fast a study enrolls, but what questions the evidence it generates is capable of answering. A study designed to reach the full treatment population produces a decision-grade living data asset. A study designed around available sites produces evidence with gaps that become visible only when regulators, payers, or medical affairs teams ask the questions the data cannot answer.

PicnicResearch has built its drug evidence model around this need. By enrolling patients through multi-channel recruitment methods including SP and PSP channels, studies on the PicnicResearch platform have achieved on-time or faster-than-forecast enrollment in 72% of treatment evidence studies.

Once enrolled, patients are followed directly through the PicnicHealth app, with medical record retrieval across all treating providers and ongoing patient-reported data collection, removing the site dependency that causes traditional registries to lose patients over time. The result is decision-grade evidence across the full treatment population, not just the patients a site network can reach.

Let’s explore how a multi-channel recruitment strategy built around SP and PSP could work for your research objectives. Get in touch with the PicnicResearch team.