Experience

Controlled Human Infection study data review and significant outcomes reporting: A clinical stage pharmaceutical company required a full re-evaluation of human challenge data following a clinical study with a European provider. The report provided highlighted one significant clinical finding in particular, allowing the company to both make a positive announcement to investors and to raise significant capital on the back of publicising the result. Furthermore, the finding allowed for a clear path to market and the potential for accelerated approval. 

The use of expert oversight and analysis in specialist areas avoids both costly mistakes or even failure at the clinical and even pre-clinical stage of drug development and provides clearer insights into drug potential and pitfalls. 

The ever-increasing cost of drug development has, in many instances, unfortunately led to the employment of staff with limited  experience in the interpretation and management  of clinical safety data, at both clinical and pre-clinical stages. Additionally, drug safety regulation frameworks are, of necessity, inherently conservative, the default position being that ‘the drug is at fault’.

The challenge for drug developers in this environment is to identify which safety signals are genuine, which are minimal and/or manageable, which are serious and require immediate intervention and reporting and which signals may mandate a change of plan or even candidate. 

Consider a program that was nearly aborted due to a life threatening SAE that was attributed, dogmatically by default, to the study drug. Careful consideration of the entire clinical picture, study disease, and study protocol by clinical experts rescued the situation, with attribution ultimately clearly attributable  to co-medication. The programme was able to continue. Generating positive results.

Data analysis leads to positive stock exchange announcement: An Australian company developing an intranasal drug for seasonal viruses requested a reanalysis of RCT data from a community trial.  A lack of efficacy had been noted for the primary outcome causing concern in the company and for investors. Following an extensive data review, statistically significant findings were evidenced for disease correlates, including pivotal clinical outcomes (symptoms). Re-targetting the drug towards prophylaxis of commonly-occurring, long-term adverse events associated with the infection was made possible. Earlier engagement with the Pathogenus scientific and clinical team could have further boosted both market size and sales potential.

​On the basis of the findings the company was able to announce positive results on the ASX leading to share price rises, stability and potentiated a novel marketing and development program for the product. 

Full-service design and implementation package: A Portuguese firm specialising in therapeutics for diabetic foot ulcers (DFU) required a full-service team to assist in the definition, design, approvals and implementation program for their product/s. The team successfully refocused the product on infections (DFI), designing an early and late-stage development plan including all documentation (synopsis, protocol, regulatory package). Preclinical data was re-examined, and further studies were mandated to support efficacy claims and to optimise dosing. Targetting DFI considerably raised the chances of a successful MAA. Options for multiple country trials and MAA were considered and researched re feasibility and likely outcomes with a view to accessing accelerated approvals programs. The late-phase study is nearing completion with data being compiled for review by the Pathogenus team.

Accelerated development of cholera vaccine: A vaccine company developing a novel cholera vaccine for global release was looking for innovative approaches to speed up development. After assessment of the pre-clinical and early phase 2 immunogenicity data, we advised the company perform a Human Challenge Trial (HCT) to establish phase 2 proof of concept data. This would allow the company to have early efficacy data and to guide the design of the phase 3 pivotal trial. Our team prepared the US development strategy and assisted the client in the FDA meeting preparations.

The team also advised the company on the selection of the clinical sites and agents to best perform the HCT trial. 

 Operational review and revisions enable delivers increased capability and capacity at no extra cost: A clinical site was going through a period of strong growth, but was experiencing difficulties scaling up their operations to meet demand. 

An in depth review by an operations manager of the existing and required processes and performance KPIs coupled to an assessment of the company wide roles and responsibilities (R&R) of each department was performed. A number of significant changes were proposed and implemented:

· Review of R&R identifies cost-rich and inappropriate use of skillsets:

- Following a round of process review and interviews with staff, it became apparent that clinical study investigators were spending >60% of their paid time performing simple and repetitive administrative work. Whilst critical to project delivery, it was equally clear that capable, in-house clerical staff were able to perform these functions both accurately in a timely manner. Besides realising immediate cost savings, the enhanced efficiencies allowed for the throughput of additional clinical trials with the same number of investigators (> profit margin).

· Healthy Volunteer screening processes were critically reviewed: A reduction in redundant steps within the screening process allowed for the doubling of the number of volunteers screened for inclusion in clinical studies with no increase in staffing or time taken.

· Training: Each department had developed individualised and unique training processes and programmes. Establishing a centralised training department for the company provided focus and  oversight, improving roll-out times, programme compliance and the appropriate documentation training and competences. 

The above and other critical changes in infrastructure allowed the sites to more than double the number of studies they were able to process per annum, reduce costs both for the company and the customer and retain an increased percentage of revenue.