President Obama today signed the legislation by Texan Rep. Michael McCaul into a law that offers drug companies incentives worth millions and possible billions for developing medication for rare childhood diseases. The legislation came up as an apparent effort to regulate the mismatch in drug development between adults and paediatrics (only 1 drug approved for childhood cancers since 1980- collar).
The FDA currently has schemes such as fast track programs, accelerated approval and priority review to speed up drug development in areas with high unmet need. However the additional law to reduce FDA review times by 40% (from 10 months to 6 months) and the professed savings of $500 M looks largely ambiguous. Additionally the FDAMA of 1997 also grants paediatric exclusivity to drugs that are proven in trials to be effective in children. This is for six months.
The real motive behind this move could be a shift in strategy of drug developers to move towards beginning to develop drugs in pediatrics first and subsequently move to adults. It won’t be a surprise if more and more companies pan out their clinical development life cycle beginning with paediatrics followed by or concurrently with adults.
Currently the privileges for fast drug development rolled out by the FDA are based on a relative scale of unmet need. In 2011, only 3 of the 30 NMEs registered came through the accelerated approval route which is underwhelming. 84% of the 91 applications for fast track status of drugs were granted which suggesting a high percentage of utilisation of this pathway. 21 out of 92 approvals in 2011 were from priority review again suggesting high acceptance and utilisation. Amidst these privileges to develop drugs for rare conditions, a new legislation at this juncture to promote paediatric drug development is seemingly unnecessary.
It can also been seen as an intelligent move by Obama towards his campaign for the polls as a supplement to the recent healthcare act.