When you have conditions that fall into the rare diseases classification, the recruitment of patients is challenging considering the small population in the Nordics. In order to advocate patient centricity, trial sponsors and CROs must explore where these patients are coming from regionally and geographically to support successful patient enrolment. As a trial sponsor, if you are simply looking at Sweden in terms of patient recruitment there will be ‘X’ amount of patients – but if you include all Nordic countries there is clearly a much greater population. In this case, you have to consider how you will reach those patients initially then also the operational and logistical aspects of getting them there. For example, while you may expand your reach to other Nordic countries, how will a Swedish patient be able to take part in a Danish trial? Again, it is important to truly consider the needs of the patient alongside your communication pathways to support patient retention.

Countries like Sweden have good registries and on an official level these are managed by physicians. As a trial sponsor you can advertise through: local newspapers, various social media channels, or through therapeutic-specific foundations and dedicated patient communities. The Swedish Association of Rare Disorders is one such organisation that delivers patient assistance programs to ensure the best outcome for patients, providers and companies committed to compassionate, patient-centred care. As a result patient recruitment is more streamlined and interaction with patients is improved through understanding the patients’ needs of them alongside the needs of their families.

Small/Virtual Pharma/Biotech Perspective

Patient recruitment is incredibly challenging for small/virtual companies who have all of their resources poured into one product and one clinical trial. If the trial does not move forward as expected this is a significant drain on resource and could have disastrous effects on the company and employees. For these companies, they must present results in any given time to private partners, hence effective patient recruitment and retention is essential. Recruiting and retaining rare disease patient populations based on immune status and genetic makeup is a challenging process, however identifying the right patients at the right time through the right channels is paramount. Tackling this challenge can include the use of biomarkers and genetic sequencing data. In this case, small/virtual companies can draw on a CRO’s scientific expertise and data team to plan for how they and trial sites might address such issues for the duration of a trial.

Measuring Patient Recruitment Study by Study

Patient recruitment is a complex area and difficult to measure unless you consider this study by study. The main challenge is that sites overestimate patient recruitment and trial sponsors tend to believe them. To minimise this it is important to carry out feasibility studies to estimate number of patients more accurately. Some companies operate on a ‘Lasagne Law’ where patient recruitment eventually drops to nothing. The implications of this mean studies get delayed and you have to terminate studies due to poor recruitment. When progressing to Phase II, a specific patient population is required, for example, for oncology patients with a particular mutation, you would need to recruit for that specific mutation. In this case you would go through a diagnosis process where you screen for mutations and markers to drive the trial forward. Additionally, you would compile a database for a specific mutation so you can go back to it again and again for reference; this is the foundation for effective and efficient patient recruitment. As soon as the patient has been screened the patient will know their condition and the trial sponsor can use networking to interact with them and keep them engaged.

In summary, patient recruitment for rare diseases has many facets to consider in terms of identifying recruitment channels, trial-size dependent factors, and study by study implementation. While challenges exist, supporting rare disease patients from the start and throughout the trial will make trials more accessible to this patient population. As a result, we expect to see more patients taking part in trials and consequently more treatments approved for use.

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