ADAPTING TO THE CHALLENGE- HOW ARE INNOVATIVE STUDY DESIGNS BEING USED IN RARE DISEASES?
Author(s)
Berry LJ, Eddowes LA
Costello Medical Consulting Ltd, Cambridge, UK
Presentation Documents
OBJECTIVES: To investigate the extent to which novel or innovative study designs have been used in clinical trials for rare diseases since 2012, and compare their use across different types of rare disease. METHODS: We used a recent review of innovative study designs in rare diseases as a stimulus. Searches were conducted in MEDLINE in May 2016 for articles published in 2012 or later. Bibliographies of systematic literature reviews and pooled analyses were hand-searched. Eligible studies reported prospective, interventional drug trials, or protocols for these, with only rare disease participants, and an ‘innovative’ design. However, ‘innovative’ was undefined in order to include as many studies as possible with an unconventional design that may help overcome a problem inherent in the study of rare diseases, eg. small populations or dispersed participants. Included articles were grouped by trial design, and rare disease subgroups that we defined. RESULTS: Our search yielded 566 unique records, of which 8 fulfilled the inclusion criteria. Two additional records were identified through hand-searches giving a total of 10 included records, each reporting an interventional study. The range of ‘innovative’ study designs comprised 6 crossover trials, 2 n-of-1 trials, a factorial design and a randomized withdrawal design. All the included studies concerned non-cancer conditions: 4 neuromuscular and 6 non-neuromuscular, including hereditary hemorrhagic telangiectasia, hereditary angioedema, cold urticaria, ANCA-associated vasculitis and trigeminal neuralgia. Crossover designs were used across a range of rare diseases. CONCLUSIONS: Crossover study designs are being used more commonly than other types of innovative study design in rare disease research. Our results did not reveal any association between types of rare disease and the choice of trial design. The lack of rare cancer studies suggests that innovative trial designs are not being widely used to study their treatment. REFERENCES:
Conference/Value in Health Info
2016-10, ISPOR Europe 2016, Vienna, Austria
Value in Health, Vol. 19, No. 7 (November 2016)
Code
PRM210
Topic
Study Approaches
Disease
Multiple Diseases, Rare and Orphan Diseases
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