Orphan Medicines Regulation For Rare Diseases On The Road To Success

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Orphan Medicines Regulation For Rare Diseases On The Road To Success

http://www.medicalnewstoday.com/medicalnews.php?newsid=46288

The European Commission has published its final evaluation (1) -

following comments received from stakeholders (2) - on five years of

the EU's Orphan Medicines Regulation (EC 141/2000). The report

confirms the tangible benefits of the Regulation for patients with

rare diseases and its contribution to growth and jobs by stimulating

increased investment in R & D to meet a high unmet medical need,

which in turn has also led to new start-up companies.

The joint industry task force of EuropaBio - the European association

for bioindustries - and of EBE - European Biopharmaceutical

Enterprises - welcomes the Commission's Report because it increases

awareness about rare diseases in the EU and promotes the continuation

and further support of the Orphan Medicines Regulation, an important

piece of innovative legislation in Europe.

There are some 8000 rare diseases, many are life-threatening and/or

seriously debilitating, and for most of them, there is no access to

effective medical treatments. Given that 70-80% of these rare

diseases have a genetic origin, biotechnology is an important tool to

develop treatments for them. Another consequence of this genetic link

is that these diseases affect children, who can thus benefit from

more than half of the products developed.

So far the Regulation has delivered 22 approved therapies in the EU,

and the portfolio of products in the pipeline, which have been

designated as orphan medicines, may promise treatments for more than

200 different conditions (3). This Regulation is transforming the

public health environment to the benefit of rare disease patients.

The Commission's Report notes that the total cost of approved orphan

medicines today in any given country typically accounts for less than

1% of the national healthcare budgets earmarked for medicines, but

timely and equitable access for patients to the approved medicines

remains an issue. The Report further notes that some member states

are limiting patient access by delaying reimbursement. As of December

2004 only one Member State could demonstrate that all approved orphan

medicines were reimbursed and available to patients. Eurordis, the

umbrella patient organisation for rare disease patients in Europe,

has published a report on the issue of access (4).

" The need for equitable and timely access for patients to innovative

therapies, coupled with the need for more incentives by Member States

to stimulate research, development and marketing of orphan medicines

should be combined. " says Tambuyzer, Chair of the Joint

EuropaBio / EBE Task Force. " By generating a new set of incentives to

tackle access issues to benefit patients, Member States would at the

same time help the EU move towards the so-called Lisbon knowledge

economy targets. " The industry is happy with the Comments from the

Public Consultation published by the Commission (2), but misses

reference to the access issue and to the suggestion above.

According to the Commission report in the absence of policies at

Member State level to stimulate private sector research, the 10 years

market exclusivity for an orphan medicine in respect of similar

products remains the main industry incentive. However, industry is

concerned that the delay in reimbursement and late access for

patients cuts broadly into the 10 year market exclusivity, eroding

the incentive quite a bit.

One year ago, the Joint EuropaBio / EBE Task Force on Orphan

Medicines published a White paper (4) putting forward 9 suggestions

for the full implementation of the Orphan Medicines framework in

Europe. " We call on the Commission and the Member States to look

again at these suggestions, and to consider them, " says

Tambuyzer. " The full implementation of the Orphan Medicines Framework

should include Member States resolving the issues around patient

access, and a predictable orphan medicines environment that will

foster further R & D in the field, with the typical collaboration

between researchers, clinicians, industry and patients which is so

typical for this field. Industry is taking up its part of this

responsibility, and is listening to other stakeholders as well. "

(1) The Commission Report - Review of five years of Orphan Medicines

Regulation

http://ec.europa.eu/enterprise/pharmaceuticals/orphanmp/doc/orphan_en_

06-2006.pdf

Commission Press Release.

(2) Commission Report on the comments received for their Working

Document: Commission's 5 year review of Orphan Medicines Regulation.

http://pharmacos.eudra.org/F2/orphanmp/doc/2006/summarycomments.pdf

(3) Register of designated Orphan Medicinal Products (by number)

http://pharmacos.eudra.org/F2/register/orphreg.htm

(4) Eurordis - Improving patient access to orphan drugs in Europe

http://www.eurordis.org/article.php3?id_article=1013

(5) Joint EuropaBio / EBE White paper on Orphan Medicines

http://www.europabio.org/documents/Orphans_WhitePaper_printedversion.p

df

More about Rare Diseases

EuropaBio web page resource on Orphan Medicines

http://www.europabio.org/healthcare.htm#orphan

EBE web page resource

http://www.ebe-biopharma.org/Topics/Orphans.htm

About EuropaBio

EuropaBio, the European Association for Bioindustries, has 70

corporate members operating worldwide, 11 associate members, 5

BioRegions and 24 national biotechnology associations representing

some 1500 small and medium sized enterprises involved in research and

development, testing, manufacturing and distribution of biotechnology

products. http://www.europabio.org