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PRESS RELEASE
HIGH DRUG COSTS DESPITE PUBLIC R&D
FUNDING IN THE MILLIONS
Ludwig Boltzmann Institute for
Health Technology Assessment (Vienna, Austria) identifies public
funding for the development of high-priced drugs. New
investigational search strategy aims at contributing to greater
transparency. Three pilot studies were conducted, more will
follow.
Vienna, Austria, 26 September 2019 - EUR 165
million in public and private R&D grants for the discovery and
development of a drug that generates millions in revenue per
patient (!) for a pharmaceutical company. Numerous public
research grants were also identified for the development of two
other high-priced drugs. This was possible thanks to a search
strategy developed by LBI-HTA, intending to create more
transparency about the societal value of public investments in
the health care system and therefore supports the demand for a
„public return on public investment“.
Newly developed
drugs are increasingly becoming extremely expensive. This poses
new challenges for healthcare systems. The OECD has been
addressing this problem for some time, as it restricts “access to
medicines”. In May of this year, the World Health Organization
WHO adopted a resolution calling for greater transparency in the
pricing of medicines. The Ludwig Boltzmann Institute for Health
Technology Assessment (LBI-HTA) in Vienna has now developed
methods precisely for this purpose. They allow the identification
of public and philanthropic funds that contribute to the
discovery and development of an approved drug.
LOTS OF MONEY. POOR TRANSPARENCY.
"
For the time being, we have
piloted our method on three drugs and have been able to prove
that all three drugs have received grants to the value of
millions of Euros in several digits" says Priv. Doz. Dr. phil.
Claudia Wild, head of the LBI-HTA, representing the quintessence
of the study. "These funds were provided at different stages of
discovery and development of the drug by public or philanthropic
sources. All three drugs are sold by pharmaceutical companies.
"The three compounds studied are: Nusinersen (against spinal cord
atrophy, a rare neuromuscular disorder), Cerliponase alfa (to
treat a hereditary disease in children that leads to progressive
brain damage) and Burosumab (against a genetic disease in
children that can lead to weak bones and lifelong physical
disabilities). All three drugs are therapies against rare
diseases in children.
"High R&D costs are often cited by
manufacturers as a reason for the high costs of their drugs,
therefore transparency about public research funding
contributions should be very important. However, this is
precisely not the case," explains Dr. Wild. In fact, basic
research - publicly and philanthropically financed - often
provides the discovery or brilliant idea that later finds
application in a drug - and at the same time is associated with a
high degree of uncertainty regarding results and high expenditure
of resources.
THE TRANSPARENCY PUZZLE
The
LBI-HTA sought to shed light on the question of public funding
and contribute to creating transparency around this issue. . "In
fact, we had to develop our own search methodology in order to
assemble puzzle pieces of information from numerous public
sources, which in the end resulted in enormous sums of funding,"
stresses Dr. Wild. The search strategy is structured in three
stages:
Stage 1: All generic and molecular names and terms
of the medicinal product or its active ingredient are identified.
This is done for the entire R&D process and in several relevant
databases (e.g. DrugBank, ChEMBL, Therapeutic Target Database).
Stage 2: The drug's development path up to approval is
determined using further trial and patent databases (e.g.
Orphanet, WHO international trial register, US Clinical
Trials.Gov, EU clinical trials registry, FDA orange book,
Espacenet, Health Canada Patent, PubMed and many more).
Stage 3: A systematic search for research grants based on the
identified names and development pathways in other databases
(e.g. NIH-RePORTER, CORDIS, IMI, EDCTP and various philanthropic
data sources) is finally conducted.
However, even with
this structured method and despite a high level of commitment,
the picture of funding in the development of the three drugs
remained incomplete. For instance, although more than 20 publicly
or philanthropically financed projects were found for the
discovery and development of Cerliponase alfa, the exact funding
amounts were often not available. Nevertheless, we could estimate
that EUR 31 million in funding had been allocated to projects
that directly served product development alone. The picture was
similar with Burosumab: numerous public and some philanthropic
projects supported the discovery and development of the drug, but
only funding amounts totalling EUR 26 million could be
quantified.
There were several difficulties in the
analysis. The biggest challenge was in distinguishing between
basic research (which can aid an entire product class as well as
knowledge about the disease itself) and research for the
development of a specific product.
Despite all the
difficulties, the method developed by the LBI-HTA enables a
significant increase in transparency about the use of public and
philanthropic funds in the discovery of new active substances and
the drug development based on them. Thus, the project makes a
significant contribution to the current and international
discussion on "Return on Investment of Public Investment".
Reference:
Schmidt L., Wild C. Public & philanthropic
financial contribution to the development of new drugs.
LBI-HTA Project Report No.: 120; Year 2019. Vienna: Ludwig
Boltzmann Institute for Health Technology Assessment.
http://eprints.hta.lbg.ac.at/1214/
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