[Ip-health] News: PharmaTimes- US Senators seek incentives for pharma R&D into rare childhood diseases

Terri - Louise Beswick Terri at haiweb.org
Mon Aug 9 05:18:39 PDT 2010

US Senators seek incentives for pharma R&D into rare childhood diseases

09 August 2010


New bipartisan legislation was introduced in the US Senate last week
which seeks to encourage innovative R&D by drugmakers aimed at treating
rare and neglected pediatric diseases.


The Creating Hope Act of 2010 builds on existing law to increase
incentives for the development of treatments for disabling and deadly
diseases, with a focus on rare conditions that may otherwise fail to
attract sufficient R&D funding.


"Seven thousand known rare or orphan diseases afflict nearly 30 million
Americans - approximately 50% of whom are children," said Democrat
Senator Sherrod Brown, who is co-sponsoring the bill with Republican Sam
Brownback and Democrat Al Franken.


The Senators note that while the US National Institutes of Health (NIH)
estimates that there are more than 6,000 rare diseases as defined by the
Orphan Drug Act - and others such as tuberculosis, malaria, and dengue
fever are neglected because they affect impoverished populations in
developing countries - fewer than 300 of these diseases are of interest
to the pharmaceutical industry. Because rare and neglected diseases
cannot guarantee the same return on investment as the more common
diseases that affect larger and often wealthier populations, there can
be little incentive for these companies to invest in the R&D required to
create new therapies, they add.


The Creating Hope Act amends provisions of 2007's Food and Drug
Administration (FDA) Amendments Act introduced by Sens Brown and
Brownback which established an incentive for pharmaceutical companies to
develop innovative therapeutics for neglected tropical diseases.


Under this law, companies which develop new drugs and biologics for
neglected tropical diseases are eligible for a "priority review voucher"
entitling them to expedited review of another drug produced by that
manufacturer. Because this voucher can be used to expedite the marketing
of a "blockbuster" or "me-too" drug, it provides a strong financial
incentive for the development of treatments for otherwise neglected


The proposed new legislation would improve upon this incentive not only
by increasing the commercial value of the priority review voucher by
making it transferable, but by expanding priority review voucher
eligibility to include rare pediatric diseases.


"Because of the unique set of circumstances that discourage innovation
in this field, thinking outside the box becomes a necessity," says Sen


By Lynne Taylor



More information about the Ip-health mailing list