[Ip-health] News: PharmaTimes- EU orphan drug regulation "is a success"

Terri Beswick Terri at haieurope.org
Thu Dec 2 06:11:44 PST 2010

EU orphan drug regulation "is a success" 

World News | November 30, 2010 


Lynne Taylor 


Some 69 drugs for the treatment of rare diseases are now available to
patients in Europe, compared with just eight in 2000, the year in which
the European Union orphan drug regulation was adopted, a new study


Moreover, during 2000-2008, R&D investment in rare diseases in the EU
rose more than 200%, while the number of company employees working on
orphan drugs increased from about 2,000 to over 5,000, says the study,
which was commissioned by industry groups the European Biopharmaceutical
Enterprises (EBE) and the regional bioindustry association EuropaBio to
assess the impact of the EU Orphan Medicinal Products (OMP) Regulation
since its adoption. 


Related Links

EU moves to bring rare diseases "out of the shadows"

Over 40 approval recommendations issued for orphan drugs in EU

The Regulation has been "one of the most successful EU healthcare
policies overall," according to Office of Health Economics (OHE)
Consulting, which carried out the assessment for the industry groups. 


Incentives provided in the legislation have "greatly fostered innovation
and entry into market of therapies addressing hitherto unmet medical
needs," it says, also noting that the Regulation has not only provided
support to companies investing in such treatments but, importantly, has
helped to establish new companies in Europe focused on researching new
treatments for rare diseases.


Welcoming the findings, Erik Tambuyzer, chairman of the EBE-EuropaBio
joint task force which released the assessment, said that this "is a
clear example of how the right legislation can drive forward European
healthcare innovation, delivering numerous benefits to individuals, to
society and, at the same time, to the development of a sustainable and
competitive economy." 


Individual rare diseases - which include certain cancers, metabolic
conditions, diseases of the nervous system and musculoskeletal disorders
- affect fewer than five in every 10,000 people, but in combination they
may affect, directly or indirectly, around 30 million people in Europe,
say the industry groups.


The study finds that R&D focused on finding new treatments for rare
diseases represents an increasingly significant proportion of the
biopharmaceutical industry's total R&D investment. Moreover, for nearly
all of the companies which have been created to focus exclusively on
researching and developing OMPs since the Regulation was adopted, all
their R&D activities and staff are located in the EU.


"Given long lead times in biopharmaceutical R&D, we can expect that the
EU OMP Regulation will have an even greater impact over the next years,"
it adds.


Companies surveyed for the report primarily target Europe and the US to
seek orphan indications, and their products are predominantly launched
in the two regions. However, the research also found that some firms
focus these activities in Europe only, and that, for these companies,
the most important element of the EU legislation is market exclusivity,
followed by access to the centralised procedure.


However, the biggest challenge following centralised marketing approval
remains the continued inequality of access to OMPs for patients across
EU member states. These large differences can be linked to demographic
and economic factors but also to the application of health technology
assessment (HTA) methodologies which, it says, can lead to high rates of
rejection and significant delay to access to new orphan drugs. 


"The increasing demand for HTA to inform health care decisions will
therefore represent a major challenge in terms of access to OMPs, which
are unlikely to meet standard HTA requirements," says the report.
However, it cautions that: "ensuring access to orphan drugs in national
health care systems in a timely and effective way is important to
maintain the positive impact of the OMP developers on the economy and,
ultimately, to continue delivering life-saving therapies for patients."





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