Table 3 Barriers and solutions after completion of study
Barrier | How addressed |
|---|---|
Timely data analysis | Efficient CRO and statistical support |
Assessing study results | Seasoned experts from Sobi and efficient consortium including clinical experts |
Dissemination | High profile publication prepared within 6 months of completion of data analysis |
Regulatory approvals | Sobi undertook production of documents and communications with the European Medicines Agency |
Further dissemination | More than 10 publications have already ensued from study |
Patient access to study drug nitisinone | Sobi, Lead coordinator and AKU Society UK have achieved near Pan-European National HTA approvals of study drug |