Avrupa İlaç Ajansının yeni Kas Distrofisi Rehberi (İng)

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New EMA Guidance on muscular dystrophy

EMA has published a new draft guideline titled “Guideline on the clinical investigation of medicinal products for the treatment of Duchenne and Becker muscular dystrophy”. The scope of the guideline is limited to the X-linked recessive dystrophinopathy Duchenne (DMD), the most common and severe form of muscular dystrophy, and its milder version -Becker (BMD) muscular dystrophy. Other neuromuscular diseases are presently not within the scope of this guideline. 

Recent advances in basic and clinical research have opened new perspectives for future therapeutic options in Duchenne and Becker muscular dystrophy (DBMD). The increasing number of clinical trials that recruit a rather small number of patients for these progressive disorders has raised several issues, including the study design, the choice of appropriate efficacy endpoints in general and the definition of reliable surrogate outcome measures as well as the need of subgroup analyses with respect to the heterogeneous patient population and the duration of the trials (e.g. long-term treatment goals 3). As most of the cases of Duchenne muscular dystrophy (DMD) have an onset in early childhood, while the onset of Becker muscular dystrophy (BMD) covers a broader age spectrum, specific difficulties have been identified that pertain to diagnostic criteria, age- and stage related clinical relevance and different safety aspects.

This Guideline is intended to provide guidance for the evaluation of medicinal products in the treatment of DMD and BMD; it is acknowledged that for several aspects the present document cannot give definite guidance due to the heterogeneity in phenotypes of both diseases and the expected treatment goals that also may vary according to disease status.

The present document should be conceived as general guidance and should be read in conjunction with other relevant EMA and ICH guidelines