FDA issues draft guidance produced with the help of PPMD on developing drugs for Duchenne Muscular Dystrophy
FDA has issued a draft guidance for industry, “Duchenne Muscular Dystrophy and Related Dystrophinopathies: Developing Drugs for Treatment” to assist drug companies in the clinical development of drugs for the treatment of X linked Duchenne muscular dystrophy (DMD) and related diseases, including Becker muscular dystrophy, DMD associated dilated cardiomyopathy, and symptomatic carrier states in females. This draft guidance addresses FDA’s current thinking regarding the clinical development program and clinical trial designs for drugs to support an indication for the treatment of one or more dystrophinopathies.
The development of this document was preceded and guided by a proposed draft guidance independently prepared by an advocacy group, Parent Project Muscular Dystrophy (PPMD). Below is a summary of a paper describing how PPMD took up the challenge of drafting these guidelines.
FDA has released a press statement stating that they “value(s) PPMD’s effort and input and appreciates the insights provided by the DMD community.” FDA believes that this is an excellent example of collaboration between engaged stakeholders and a beneficial approach to get contributions from patients and caregivers on drug development.
Stakeholders and interested parties may view the Federal Register notice for information on how to submit comments to the public docket.
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4th meeting of the Health Technology Assesment (HTA) Network was held in Brussels on 23 March, 2015. All Member States (MS) and Norway, as well as the EUnetHTA and the European Medicines Agency (EMA) were present. The highlights of this meeting included a presentation by EFPIA of an internal survey on the usage of Joint Work in relation to Health Technology in a sample of European member states. The presentation addressed the issue of how HTA reports at a European level may contribute to the avoidance of duplication of work, and to the greater efficiency of HTA in a national context. 
The French National Authority for Health (Haute Autorite´ de Sante´, or HAS) is commissioned to produce ’health economic opinions’, that determine the most cost-effective therapeutic strategies, and edit the recommendations accordingly, which is carried out by Economic and Public Health Assessment Committee (Commission Evaluation Economique et de Sante´ Publique, or CEESP). This decision in part as part in response to the French Court of Auditors (Cours des Comptes), which regularly challenged the lack of economic evidence use in pricing decisions in medications. An article published in Market access and health policy describes the mission, structural and functional aspects of CEESP and its impact on health economic assessment of drugs available in France. 
A study published in Brain & Development explores the contributions of Japanese patients is helping Antisense oligonucleotide (AO)-mediated exon skipping therapy for Duchenne muscular dystrophy (DMD) to reach the stage of marketing authorisation. DMD - a fatal progressive muscle wasting disease is known to be caused by dystrophin gene mutation. According to the authors the first account of the deletion within dystrophin exon 19 was found in 1990 in a Japanese DMD patient. Based on these finding, the authors who also originate from Japan proposed an AO-mediated exon skipping therapy for DMD in 1995. The review emphasised that since then “more than 300 reports on AO-mediated exon skipping therapy for DMD have been published, including at least two a month during the last few years.” The authors believe that the remarkable contributions by Japanese patients and researchers should be recognised for their efforts towards the development of the exon skipping therapy for DMD. 
