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Table 2 Barriers and solutions during the conduct of study

From: Clinical development innovation in rare diseases: overcoming barriers to successful delivery of a randomised clinical trial in alkaptonuria—a mini-review

Barrier

How addressed

Fixed time for completion of study

Focussed and organised consortium; effective and rapid communication with funder European Commission

Lack of recruitment from UK

Recruited from outside UK

Travel for disabled patients

Patient society support and engagement; motivated patients

Language issues for overseas patients

Interpreters used

Complex protocol and extensive assessments

Assessments spread out over 5 days; all travel arrangements facilitated to minimise harship

Heterogeneous study sites

Careful harmonisation of processes and procedures; site initiation visits key

Continued patient participation

Patient Societies involved

Timely recruitment

Patient Societies involved. Weekly recruitment telemeetings

Retaining patients to complete study

Patient Societies involved. Weekly retention telemeetings

Efficient and timely completion

Weekly project board telemeetings

Face to face yearly meetings