Reducing Study Complexity To Minimise Cost And Burden Of Clinical Trials

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Reducing Study Complexity To Minimise Cost And Burden Of Clinical Trials

It’s no secret. Clinical trials are getting more complicated and this is driving up the cost of developing new drugs. As recently reported by the Tufts Center for the Study of Drug Development (CSDD)^a, this complexity is negatively impacting on clinical trial efficiency, the burden on those executing the studies, and participant recruitment and retention. But, it is a trend that is only set to continue, with more difficult to treat and rare diseases being targeted, more stratified and unique populations being enrolled, and more diverse and voluminous data being collected.

It is very tempting to think you are de-risking a project by performing as many tests and collecting as much data as possible early on. But, when up to 40% of that data may never be used in the new drug application, can you really afford the luxury of extra time and cost required at both site and sponsor level, and the extra burden it adds to the participants of your studies?

There’s a balance between getting everything, and getting studies done in a timely, cost-effective way. But how do you figure out early on what you won’t need later? The key to that is working with your external partners to weed out what procedures and data are unnecessary. Regulatory authorities have minimum expectations regarding the safety and efficacy data they expect. Clinicians will understand the current landscape and medical practice. Patients can help you understand how they access care (recruitment pathways), what are they willing to do, and what will help/hinder their participation. If you don’t have the established networks, then crowdsourcing help from those that do is another possibility^b.

If you are looking to minimise study complexity (and hence reducing clinical trial cost), considering the following questions may help:

Study design: What trial design will most efficiently answer your question? How many study arms do you need? What endpoints do you really need? Which data/tests and how often are they really required, will the results support your endpoint analysis? Are standard-of-care tests/procedures suitable or are bespoke testing/procedures required? How long does the study need to be?

Study population: What are the essential eligibility criteria that will create a uniform enough sample without creating recruitment barriers (e.g. Do you really need those age or comorbidity restrictions?) What are the minimum tests and procedures required to confirm eligibility? (e.g. Do you really need another MRI/CT scan if the patient had one recently?) What is the process for collecting and documenting informed consent? (i.e. is it a short paper form in lay language, or a complex series of steps involving paper and online documentation, that will require additional security and privacy controls?) How many sites/countries will you need to achieve the recruitment and ethnic diversity you require?

Randomisation: What process will sites use to randomise/track a patient? (e.g. can sites randomise with an envelope or will data entry into online/phone systems be required?).

The nature of your intervention: How and when will your intervention be administered, how does that compare with currently available competitors, and will this impact recruitment/retention? Will participants require training and regular follow-up in order to maintain compliance, and how might this need to be supported with site training/time/follow-up? What are the supply/storage/accountability requirements for your product?

Study sites: What is the nature of the site teams that you need (e.g. Do you need a single department or multidisciplinary/multidepartment team?); What specialised training and resources will a site need to comply with the protocol? (e.g. Will you need to certify specialised raters? Are separate blinded and unblinded team members required? Do the sites have the equipment you need, or will it need to be provided/outsourced?) Which site staff are required to use which electronic systems when, and is specialised training required? Which site staff will require GCP training?

Data collection: What data is required from what sources, how often, and how will this be data be integrated, monitored, analysed? (e.g. Will patients need to enter diary information electronically, download or transmit wearable data, etc? Will quality of life data be required in addition to efficacy/safety data? Will site data need to be uploaded manually to eCRFs or other data repositories? What systems will be used to collect, report and distribute safety information? Who will be monitoring data completeness and accuracy, and when? What are expectations regarding timing of data entry and query resolution and do sites have the resourcing to comply? Will interim analyses be required?).

Follow-up: How long will participants need to be followed subsequently to the treatment phase of the protocol for safety and efficacy reasons? (e.g. will a 48hr phone follow-up suffice, or will 3 monthly in person visits for a year be required?).

Practical issues: How will the project be managed and by whom? When, how and how often will your internal team and external partners meet/communicate? What are the issue escalation/resolution and decision-making authority structures within the team/organisation? What electronic systems are internal and external partners expected to use? What reporting is required by whom, to whom, how and how often? What skills and training do different team members require, and when?

Assessing your protocol complexity^c will help identify where there may be non-core requirements that can be stripped out, helping you achieve a cheaper, faster study, without loss of quality. These questions are just some of those that can help you do that. By taking the time upfront to plan and think strategically through the minimum requirements you need to achieve your study aims, you also stand a better chance of avoiding subsequent protocol amendments which only add further clinical trial cost and delays^d.

Southern Star Research project managers excel at helping our clients streamline their study conduct, stretching those valuable financial resources all that bit further. Call us today to find out how we can help your organisation successfully achieve its clinical development goals while reducing clinical trial cost.