Designing your study, a worked example

The previous two editorials have been largely theoretical; a frame within which to view what is possible in pharmaceutical research. The following attempts to apply the theory to a real-world scenario.

Scenario: A new treatment aimed at a currently un-serviced portion of the market is currently going through its Phase III trials. It looks promising. It is now time to look at framing the market position of the treatment ready for launch.

First off, we want to characterise the patient cohort and current treatments on the market for this area. While there may not currently be a treatment indicated for this condition, there will almost certainly be some off label use, or smudging of the guidelines by physicians catering to this cohort (lets just be honest, if a doctor has a sick patient, they are going to try and find a treatment for them).

This is probably best served by a Retrospective Study utilising the available data in a patient’s EHR. It is ‘relatively’ cheap and quick to execute, allowing for the results and findings to be incorporated to the on-going Phase III trials. From this you will be able to:

  • Characterise the patient cohort
    • Age and geographic distribution
    • Comorbidities and current medications
  • Mapping of patient pathway
    • Current medication being used
    • Indication of effectiveness of treatments
    • Indication of safety of treatments
  • Definable search query for finding patient cohort using EHR (Code lists)
    • To be deploying in any current or future studies to increase patient recruitment
  • Mapping of code coverage within EHR
    • Indication of how easy/reliable research using EHRs will be for this cohort

Once this has been defined, a more extensive, prospective study into the effectiveness and safety of current treatments might be a good next step. This has two major advantages.

  • Frames the failings of the current market competition ready for your treatment to ‘ride in and save the day’
  • Starts to identify individual patients that may benefit from your treatment in the near future

A study like this could be run either as an interventional or observational prospective study, tracking the patients for a protracted period of time on the currently available treatments, allowing for detailed collection of data. Dependent on the treatment area, or more precisely, the normal variables collected through standard care and the number of interactions with the healthcare system in a given period of time, will indicate whether suitable data can be collected with an observational methodology (or if you are going to have to get your hands dirty and actually see patients). This will obviously have significant impact cost.

A purely observational study can even potentially be consented by the patient’s doctor and have the patient monitored purely through their standard care and the information collected through their EHR (in an anonymised form). If this will not collect the desired level of information, a more interventional methodology may be required, whether that be through patient visits to a clinic to collect particular variables, or standardise the treatments being taken to make analysis more meaningful.

Note: There is scope within certain disease areas for a middle ground where separate to the study, the sponsor can fund ‘best practice’ clinics for patients with the condition at certain practices. This is run in parallel to a prospective, observational study. However this can be difficult to engineer in an ethical and above board manner, so will not work in every situation.

This style of study can then be extended/expanded after authorisation into a safety study, collecting longitudinal data on patients using your treatment by the same methodology.

A couple of additional options to consider around this style of application are Patient Powered Research Networks (PPRNs), Registries and disease awareness campaigns.

PPRNs are rapidly growing into a buzz word around pharmaceutical research, however rather than being drawn into the hype, focus on the real benefits and opportunities. By investing in a PPRN (whether existing or creating a new one) at this phase of a drugs life cycle, you are creating a network and voice of patients you can listen to as a barometer for the wider market. This network can also be leveraged to help support patient recruitment for future studies.

Registries have fallen out of favour in some circles, largely due to the rise of the EHR databases, however this misses the point of a registry. A registry should be a snap shot of a patient at a given moment in time with a certain condition. Particularly identified (or pseudo-anonymised) registries are very useful for feasibilities, and as a starting point for patient recruitment.

Finally, disease awareness campaigns are an exceptionally cost effective way to increase patient recruitment over the life a treatment. Invest the money at this point and you can push patients towards PPRNs, increase general awareness of the condition and even start to influence prescribing patterns from the patient side.

As a final note on this series of editorials as a whole, I am again drawn to a section of Paul Simms’ (Chairman of eyeforpharma) introduction to ‘Real-World Revolution’ quoted in part one;

“Nowhere do we see the move from volume to value more clearly than in the incredible growth in the use and importance of real-world evidence. Randomised Clinical Trials remain the gold standard for clinical evidence but only through the collection and analysis of real-world evidence data can we paint a full picture of a medicine’s impact on the lives of a patient population.”

In other words, look broadly at the question and work out how you can best answer the question with the data available.

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