FDA DCT guidance Sept 2024 – BENEFITS
This is number 2 in a series of newsletters based on the FDA guidance on DCTs, issued as final in September 2024 and is all about the benefits of DCT.
The guidance recognises the benefits of decentralized trials.? It is hard to summarise any better than their own statement, so these are two direct quotes:
“DCTs have the potential to expand access to more representative patient populations and improve trial efficiencies.? Advances in using electronic communications and information technology to interact with trial participants in different locations (i.e., telehealth) allow for fewer in-person visits to traditional clinical trial sites. Digital health technologies (DHTs), for example, have expanded the types of trial-related data that can be obtained remotely from trial participants. By enabling remote participation, DCTs may enhance convenience for trial participants, reduce the burden on caregivers, and facilitate research on rare diseases and diseases affecting populations with limited mobility or limited access to traditional clinical trial sites. This may help improve trial participant engagement, recruitment, enrolment, and retention of a more representative trial participant population to improve the strength and generalizability of the evidence produced by the trial.”
“Outreach through local health care institutions (e.g., pharmacies, clinics) may facilitate recruitment of participants with diverse demographic characteristics more reflective of the intended patient population in areas where there are limited or no traditional clinical trial sites.
Bringing trial-related activities to participants’ homes may reduce the need for travel and improve engagement, recruitment, and retention amongst potential participants who have challenges accessing traditional clinical trial sites. The use of local HCPs close to potential participants’ homes may further improve engagement, recruitment, and retention of a more representative participant population and reduce cultural or linguistic barriers to participation in clinical trials.”
From my perspective this is a great statement, identifying the potential benefits of DCT.? The trick is in ensuring the potential will actually be delivered.
For example – consider a study that:
·????? Has 10 monthly visits.
·????? All such visits are conducted at the site.
In scenario 1 ePRO is introduced to record the patient data directly at various intervals, making this a DCT.
The benefit of the ePRO is not felt by the patient.? The data COULD be collected at the site visits, but paper records are less accurate, so the benefit here is data quality.? But the patient still has to visit the site the same number of times, has to learn how to use an ePRO system / device and needs technology support in case the device fails (and the site has the same issues).? This is more work for the patient, not less, and will not lead to improved recruitment.? Indeed, it may well lead to poor retention (and a poor reaction from sites, as we have seen quite dramatically post pandemic).
In scenario 2, consider the same study, but now two visits are reduced to blood sampling only, to be conducted at a local clinic. This is more convenient, however two visits out of 10 has a fairly limited impact on the patient’s life, and they still have to get in the car or public transport and potentially have a carer accompany them.? Improvement in recruitment and retention will be mild. In addition, outside of urban USA, these clinics are not actually that common. The patient pool increases marginally as the trial is indeed more convenient, improving recruitment somewhat.
In scenario 3, 50% of the visits are conducted in the patients home by a nurse based at the site, who works there 3 days a week (the commonest shift pattern in western hospitals).? These will need to be more than just blood sampling, so are more complex, but are still highly suitable for a nurse to conduct. ?The impact of the trial on the patient’s life is more meaningfully reduced and if presented as an option at consenting will improve recruitment and retention more effectively.? However, the increase in the pool of possible patients is modest, as they still need to live near the site to ebable the nurse can get to them in a cost effective time frame and they still have to arrange visits at times when the nurse is working.? If the nurses travel further, because of the cost of their time and travel, the ROI for the in home care will start to reduce significantly (MRN in house data).
In scenario 4, patients are seen by nurses provided by a global or national provider, trained in the protocol as required. Patients can be seen who live much further away from the site (up to 3X further according to MRN in house data) but still live close to the nurse, keeping costs controlled.? They can also be seen in the evening or at weekends (unlikely with site nurses travelling from the site).? The pool of interested patients expands significantly to include people who can’t take time out because of work or family commitments – anything that means that they can only be seen out of hours or at a time convenient to them, not the nurse.? This also impacts diversity as these are often poorly served communities who do not participate readily in clinical research, and as such are the focus of regulatory efforts to increase diversity in trials. Recruitment rates are maximally impacted.
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These comparisons are summarised in the attached diagram.
Other mixes of these scenarios exist of course, this just serves to demonstrate the point - the value of DCT is dependent on reducing the impact on the patient’s life and extending the reach of the site geographically.? This is not guaranteed in a DCT – it all depends how you set it up.? The impact of a service should be assessed in terms of the reduction in time commitment of the patient, the activities required of the patient, the flexibility in out of hours visits and the distance from site that will be made accessible, whilst remaining convenient to the patient.
I write on anything I think is patient centric and interesting, based on my 18 years leading MRN, the oldest patient centric company in the world...(probably).
For more info on how to manage community based trials, go to the MRN web site