Patient Recruitment for Melanoma Immunotherapy Trial
An innovative biotech company that specializes in developing DNA-based intratumoral immunotherapies needed help in recruiting patients unresponsive to anti-PD-1 therapy into a stage III/IV melanoma trial.
Phesi worked with the CEO and Chief Clinical and Regulatory Officer over a 4-week period to conduct a competitive analysis, reviewing enrollment rates in top recruiting centers across 39 recent, relevant trials:
Phesi recommended extending the current number of trial centres (7) by an additional 10 sites from either Germany or the US, and identified a list of 45 investigators. Phesi also assessed sites in Australia, the US and Canada (nominated by the client and partner CRO). Phesi forecasted the enrollment duration to be 19 months to recruit 63 patients.
Phesi provided the following information in a client report:
- Identified opportunities to optimize trial design at the protocol level
- Detailed the competitive environment (e.g. historical development, competing programs, protocols and amendments)
- Identified PIs and leading countries
- Identified operational parameters (e.g., site numbers, countries, inclusion/exclusion criteria, site list)
- Forecasted operational deliverables (e.g., site activation timelines, enrollment cycle times globally)
- Identified the best site candidates by country
- Provided detailed site competition analysis to inform discussion with site candidate
Integrated Clinical Development Planning for Iron Overload Candidate
An exciting biotechnology company that develops gene therapeutic technology based on RNA wanted to develop a baseline, integrated development plan for a lead iron overload (IO) candidate. The client was keen to take a data led approach to understand and document the following:
- Competitor activity, dosing and development status, including relevant input for strategic business development activities and interactions with the regulatory agency
- Lead target indication(s)
- Leading countries, KOLs and trialists
- Protocol design, including baseline patient characteristics, standard of care, outcome measures and inclusion/ exclusion criteria
The Development program should capitalize on the coexistence of 10 patients and maximize ROI
Within three months Phesi successfully delivered to the Board a baseline, fully integrated development plan document covering:
- Product positioning: TPP and commercial potential, the potential candidate value increasing 5-fold as a result of the analysis
- Clinical development timelines and implementation planning
- Regulatory strategy
- NCD strategy
- Program timelines, resourcing and budget
- Leading authorities from a KOL and trialist stand point
Cardiovascular Study Protocol Feasibility
Phesi’s client was designing and planning a Phase II study for a unique cardiovascular (CV) indication. Its compound was first in class, and the first to target a particular CV dysfunction. The client requested insights from similar historical studies to consider what may impact trial operational performance.
Phesi selected 37 trials from its comprehensive clinical trial database, with comparable design and targeting similar patient populations (excluding observational, surgical, ancillary trials and prematurely terminated trials). Phesi experts found that screen-failure rates would be significant, and predicted how many patients should be screened to achieve target recruitment numbers. Phesi also assessed the inclusion and exclusion criteria to ensure the protocol was feasible, and to quantify the impact on enrollment.
Phesi identified a predictable relationship between the number of sites and the rate of enrollment, showing that 60 patients could be recruited from five sites over 28 months. Based on the homogeneity of the patient population, availability of patients and experienced investigators, competing trials, and quality medical records[, Phesi recommended conducting the trial in a single European country.
In just a few days, Phesi’s client received a clear and feasible trial design appraisal as well as operational guidance based on known issues from previous similar trials.
Troubleshooting an Under-Performing GI Trial
A global pharmaceutical company was experiencing significant recruitment challenges in a priority Phase III gastroenterology trial, as competitors were running concurrent studies in the same patient population. Site activation was significantly below expectations for this type of trial, and the CRO was proposing to add further sites – despite resources spread thin and with budgets under pressure. The company brought Phesi in to identify viable solutions.
Phesi used its real-time, comprehensive database to quantify the level of competition geographically. This confirmed that many of the chosen sites were in highly competitive locations for similar trials and patients. Phesi identified 17 comparable historical trials (mostly Phase III), with similar designs and number of patients. Using these as a benchmark, we agreed with the client on realistic and feasible site activation rates, patient enrollment rates, and enrollment cycle time. Our data guided a targeted action plan to get the trial on track.
Phesi counselled the client to close poor-performing sites and focus on fewer but higher performing sites, and not add new ones recommended by the CRO. Phesi worked with the trial sponsor and the CRO to align on an achievable plan to complete the trial. Enrollment completed on the exact date that Phesi predicted.
delivering strategic value to clients
Service providers are often asked to show a return on investment (ROI) or the quantifiable results of their service. This is especially so in clinical development where everyone is experimenting with various types of technologies, such as artificial intelligence (AI), machine learning and various types of predictive analytics. Although the ROI may be present, it is usually difficult to show it at the outset because a typical study in a clinical development plan (CDP) takes a long time to complete from design through enrollment.
We at Phesi have been delighted to see one of the most significant, positive financial impacts our work has had on the valuation of a company. We thought it would be of interest to people in the clinical development community, including those in the Boardroom, business development and venture capital communities, and especially those in the biotechnology space who have limited funds and need to maximize the value of the assets you are developing.
Phesi was asked to work on a biotech’s lead asset at a strategic level, defining the integrated development plan. The priority asset was about to enter first in man trials. Support was required to develop the target product profile (TPP), CDP and protocol and to select KOLs, investigators and sites as part of proactive planning activities.
Phesi identified opportunities to secure orphan drug status in 2 lead indications, increasing the potential peak sales by £1.6bn, leading to investment by a third party and a dramatic rise in stock value: