Adequacy of safety data for regulatory approval of pediatric indication through extrapolation algorithm

BackgroundOptimal drug therapy in children relies on availability of pediatric-specific information. European and American legislative initiatives have resulted in advancement of pediatric pharmacotherapy data. We aim to describe the quality and quantity of pediatric information in drug monographs of New Active Substances (NASs) approved by Health Canada.Design/MethodsCanadian drug monographs of NASs approved by Health Canada, from January 2007 until December 2016, were systematically reviewed for pediatric-specific information. Pediatric-specific information defined as: pediatric indication, dosing, pediatric-friendly dosage forms, and pediatric safety data.ResultsOver the period of the study, Health Canada approved 281 NASs. Of all the non-biologic NASs (205, 74%), 39(19%) were approved for use in pediatric patients. The number of drugs with pediatric approval was lowest in 2008 (1, 8%) and highest in 2016 (8, 32%), following no specific pattern. Neonates had the lowest rate of drug approvals through all pediatric age groups (4, 2%). All drugs with pediatric approval had pediatric-specific dosing information with the majority of them presenting pediatric safety data (79%). Pediatric friendly formulation was only available in 20%(8) of drugs with pediatric approval. Studies in pediatric populations were the source of pediatric information in 59%(23) of drugs with pediatric approval.Conclusion(s)Less than 20% of the NASs approved by Health Canada for use in adults contain pediatric approval. Neonatal populations remain a therapeutic orphan, with severe lack of dosing and safety information. Safe and effective pediatric pharmacotherapy requires well-conducted pediatric research to enhance pediatric drug data. Canadian children are in need for legislative initiatives to promote pediatric drug development.Disclosure(s)Nothing to disclose

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Using real‐world safety data in regulatory approval decisions: Sotagliflozin and the risk of diabetic ketoacidosis

Pharmacoepidemiology and Drug Safety ◽

10.1002/pds.4994 ◽

2020 ◽

Vol 29 (10) ◽

pp. 1322-1324

Author(s):

Michael Fralick ◽

Aaron S Kesselheim

Keyword(s):

Diabetic Ketoacidosis ◽

Real World ◽

Safety Data ◽

Regulatory Approval

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MSF Access Campaign Drug-Resistant Tuberculosis Research and Development Case Study May 2020

10.31219/osf.io/rdc7v ◽

2020 ◽

Author(s):

Vivian Cox ◽

Christophe Perrin ◽

Katy Athersuch ◽

Greg Elder ◽

Manuel Martin ◽

...

Keyword(s):

Clinical Trials ◽

Research And Development ◽

Value Chain ◽

Safety Data ◽

Pharmaceutical Research ◽

Regulatory Approval ◽

Drug Resistant ◽

Drug Resistant Tuberculosis ◽

Tb Treatment ◽

Resistant Tuberculosis

Two novel drugs, bedaquiline and delamanid, have recently become available to treat drug resistant tuberculosis (DR-TB) after many decades of little innovation in the field of DR-TB treatment. Despite evidence of improved efficacy and reduced toxicity of multi-drug regimens including the two agents, access to bedaquiline and delamanid has been very limited in many settings with a high burden of DR-TB and consistently poor treatment outcomes. Aside from regulatory, logistic, and cost barriers at country level, uptake of the novel agents was complicated by gaps in knowledge for optimal use in clinical practice after initial market approval. The main incentives of the current pharmaceutical research and development paradigm are structured around obtaining regulatory approval, which in turn requires efficacy and safety data generated by clinical trials. Recently completed and ongoing clinical trials did not answer critical questions of how to provide shorter, less toxic treatment DR-TB treatment regimens containing bedaquiline and delamanid and improve patient outcomes. Voluntary generation of evidence that is not part of this process – yet essential from a clinical or policy perspective – has been left to non-sponsor partners and researchers, often without collaborative efforts to improve post-regulatory approval access to life saving drugs. Additionally, these efforts are currently not recognized in the value chain of the research and development process, and there are no incentives to make this critical research happen in a coordinated way.

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