FDA, Rare Diseases

Rare pediatric PRV program not linked to uptick in new drugs starting trials, study finds

The rare pediatric priority review voucher (PRV) program was not found to be associated with an increase in the number or rate of new rare pediatric disease drugs that began clinical trials, a Health Affairs study found.

But the researchers from Harvard Medical School, working on what they said is the first study on the impact of the rare disease PRV program on drug development, also found that their data do provide some encouraging news about rare pediatric disease drug development.

“Such [rare pediatric] drugs were more likely to advance from Phase I to Phase II trials, compared to drugs for rare adult diseases, after the PRV program’s creation, although the same difference was not observed for progress in later stages of development,” they write.

The authors also found that compared to drugs for rare adult diseases, drugs for rare pediatric diseases progressed more quickly through all phases of clinical testing and were more likely to be first-in-class.

The study’s findings seem to contradict the appearance that the rare pediatric disease PRV program is a success. From 2012 to today, the US Food and Drug Administration (FDA) has awarded more than a dozen rare pediatric disease PRVs, which have consistently sold for around $100 million each.

​But the authors remain cautious about both the rare pediatric and tropical disease PRV programs in general, noting, “The rapid proliferation of these programs must be considered carefully,” particularly as in 2016, the 21st Century Cures Act created another PRV program for medical countermeasures.

And given the large number of rare pediatric diseases still without treatment options (only an estimated 5% of rare pediatric diseases have a treatment), the authors make the case for additional policies outside the PRV program to bolster the development of new therapies.

“For example, new funding could be directed to the National Institutes of Health to expand a collaborative public-private development partnership for rare pediatric diseases,” they write.

The study’s findings come as President Donald Trump during the State of the Union signaled his intentions to renew efforts to develop new treatments for pediatric cancers, pledging $500 million over 10 years.


First published here and syndicated in partnership with RAPS. Email news@raps.org for more information.


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Recursion Pharmaceuticals Salt Lake City, UT
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