In short
- Based on a successful rescue of a sponsor’s phase 3 trial in patients with advanced neuroendocrine tumors, PSI were awarded another phase 3 trial in a similar patient cohort
- Both the rescue and the second trial involved top sites around the world
- During the enrollment phase of the second trial, the sponsor decided to open an additional geography: China, a relatively untapped and exciting market, with new patient populations, but comprehensive and complex legislation and logistical demands
- PSI performed feasibility analyses, and initiated startup
- The sponsor subsequently decided to launch a specific China cohort to enroll enough patients to satisfy regulatory legislation
- This trial is progressing according to milestones
The rescue trial
This story started when a biopharma sponsor contacted PSI for a rescue of their phase 3 trial in patients with advanced neuroendocrine tumors. The original CRO didn’t deliver on its enrollment projections, and when asked to expand after 12 months of slow recruitment, didn’t have the operational ability to support a study of this size.
The sponsor was able to lean on PSI’s experience to get recruitment back on track: recruiting a large global patient cohort and achieving their milestones as projected.
- A detailed feasibility analysis at nearly 400 sites across 25 countries evaluated their current competition, number of patients in their databases, preferred treatment approach, and overall interest in new clinical trials
- In addition, the sponsor drew on PSI’s network of oncology top-enrollers across Europe, North America, South Africa, and Australia, and harnessed PSI’s existing relationships with sites that enroll well in neuroendocrine and radiopharm trials
- The careful site selection led to a 45% increase in enrollment rate
The second trial
Consequently, the sponsor awarded PSI with another phase 3 NET study, set to enroll a large cohort of patients with advanced neuroendocrine tumors across tens of countries worldwide. Tactics used to ensure this trial enrolled on time included:
- Harnessing existing relationships and knowledge of how to work with larger-volume sites: Several top-enrolling PSI sites with over 100 patients were already identified and were keen to learn more
- Evaluating sites based on prior experience with radiopharmaceutical studies: PSI prioritized the sites where collaboration between medical oncologists and nuclear medicine departments was already standard
- Early development of eCOA and QoL PRO translation: Regulatory authorities require these early in the process, which helped save time
- Considering drug supply plan for SoC therapies: Early consideration for the handling requirements for the study drug and for the standard of care therapies ensured the best compliance with local practice and cost-effectiveness
These tactics were proven to work well, but the sponsor made the decision to also enroll in China. As an important and emerging radiopharmaceutical market, expanding into China offers access to additional patient populations, alongside new regulatory and logistical complexities.
Radiopharmaceutical trials in China
Radiopharmaceutical trials are on the rise in China [1], however significant challenges exist:
- Regulatory complexity – radiopharmaceuticals are classified as a drug and a radioactive product, meaning increased oversight is required
- Logistical challenges – such as limited access to the required isotopes and complexity in transporting them to sites
- Different economic priorities – for example, neuroendocrine tumor research has less funding than other types of cancer
While innovations (such as the “Mid- and Long-Term Development Plan (2021–2035) for Medical Isotopes”) have helped advance the field [1], the question remains: what does enrolling patients on a radiopharmaceutical trial in China actually mean?
China: Challenges and solutions
Regulatory timelines and complexity: Compared with Europe or North America, China has longer regulatory timelines, compounded by a more complex regulatory framework. PSI used local contacts, and years’ worth of APAC knowledge, to decrease the impact on the timelines.
Central approval dependency and delays: In this trial, approvals were dependent on processes managed by the sponsor’s local partners. Some challenges were faced due to the lengthy decision-making processes involved with this local partner. In many cases, local vendors were needed for processes or materials that typically would not be required in other regions – we used our worldwide vendor network to proactively overcome potential logistical barriers before they arose.
Site logistics: Complex site logistics required additional alignment with each site’s capabilities. PSI’s extensive PI and KOL network helped with lessons learned to smooth each site’s difficult logistical considerations.
Additional agreements: Extra internal agreements (e.g., transfer agreements) allocated a defined share of activities for planned patients. Explicit role assignment meant that each study team participant was allocated specific responsibilities, with measurable outcomes, to facilitate smooth trial execution.
The outcome
During the time that enrollment was open in China, one site enrolled at a rate more than double that of the next highest performing site worldwide. The sponsor decided to open a China cohort (to continue global enrollment for the main trial).
Both trials are proceeding according to milestones.
This is a very exciting time for radiopharmaceutical development in China. The first radioactive IP was recently granted marketing authorization. There are many early phase studies ongoing in China, with promising initial data. We welcome the opportunity to continue to share our expertise and discuss all the nuances of expanding to deliver globally in this specialized field.
Key takeaways
In China, the joint efforts of sites, radiopharmaceutical regulatory bodies, sponsors, and CROs have helped accelerate the adoption of therapies for patients with unmet medical needs. The rapid development of China’s capability to run radiopharmaceutical trials, coupled with proactive collaboration with sponsors and CROs, is leading to a revolution in which patients are able to receive unparalleled benefits.
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Abbreviations
eCOA, electronic Clinical Outcomes Assessment;
PRO, patient-reported outcomes;
QoL, quality of life;
SoC, standard of care.
References
1. An, S., et al., Pathway to Approval of Innovative Radiopharmaceuticals in China. Journal of Nuclear Medicine, 2024. 65(Supplement 1): p. 72S–76S.
