Global Spending on Orphan Drugs in France, Germany, the UK, Italy and Spain during 2007

By Orofino, Javier; Soto, Javier et al. | Applied Health Economics and Health Policy, September 2010 | Go to article overview

Global Spending on Orphan Drugs in France, Germany, the UK, Italy and Spain during 2007


Orofino, Javier, Soto, Javier, Casado, Miguel A., Oyagüez, Itziar, Applied Health Economics and Health Policy


Background

An orphan drug is a product indicated for the treatment of a 'rare disease' (i.e. for which few cases are reported). The exact definition of 'rare disease' varies between countries and is determined by its prevalence, that is, the number of patients with the disease at a given time. The WHO defines a disease as rare when the number of patients affected is less than 6.5-10 individuals per 10 000 inhabitants.[1] In the US, Japan and Australia, a rare disease is, therefore, one that affects fewer than 200 000, 50 000 and 2000 individuals, respectively.[2] In the EU, rare diseases are defined as those that affect fewer than 246 000 individuals in the 27 EU member states, that is, those with a prevalence of <5 per 10 000 inhabitants.[3]

Rare diseases are normally processes that shorten survival and affect the health-related quality of life (HR-QOL) of the patients.[4] They are often difficult to diagnose and most do not have any effective treatments available.[5] Moreover, in some cases, information and scientific knowledge on the disease are lacking.[6]

According to the European Organisation for Rare Diseases, there are currently between 5000 and 7000 rare diseases identified,[6] and approximately 250 new rare diseases are described each year.[5] Overall, 80% of rare diseases are of genetic origin and 75% affect children, thereby representing a substantial cause of premature deaths. Data from the EU and the US reveal that 35% of deaths in children aged <1 year are the result of rare diseases.[4]

Traditionally, pharmaceutical companies have not invested sufficiently in research into rare diseases.[7] To overcome this shortcoming, some countries have passed legislation to make research into these diseases more economically appealing. The first country to do so was the US, which passed the Orphan Drug Act in 1983.[8] Other countries have since followed: Singapore (1991), Japan (1993), Australia (1998),[5] and the EU (2000 - when the Commission Regulation on Orphan Medicinal Products was passed[9]). In general, these provide market exclusivity (up to 10 years in the EU and Japan, 7 in the US and 5 in Australia),[10] lower fees and accelerated authorization processes, as well as technical and scientific support.[8,9]

The Committee of Orphan Medicinal Products,[11] under the auspices of the European Medicines Agency, is responsible for examining applications for designation of orphan drug status for EU countries. The drug must meet three requirements:[9] (i) it should be indicated for the diagnosis, prevention or treatment of conditions that are life threatening or very serious and that do not affect more than 5 in every 10 000 individuals in the EU; (ii) it is unlikely that the estimated sales will cover the R&D investment in the drug; and (iii) there is no other effective and safe treatment available on the market for treating the target rare disease. Despite the benefits that designation of orphan drug status brings to these types of products, orphan drugs must follow the same regulatory formalities as any other drug. They must, therefore, show the same standards of efficacy, safety and quality as other pharmaceutical products, with the additional problem of obtaining statistically significant results in clinical trials with smaller sample sizes.[12]

Given the low number of patients with a rare disease, the associated low sales volumes and the risk associated with the clinical development of orphan drugs, the final price of these drugs is usually high in an attempt to offset the resources invested in production and clinical development. However, this same low number of patients who will be treated with these drugs means the contribution of orphan drugs to overall drug expenditure will not be particularly high.

The objectives of the present study were to (i) describe the status of orphan drugs in 2007 in the five countries in the EU with the greatest drug expenditure (France, Germany, the UK, Italy and Spain); (ii) estimate the mean annual cost per patient and indication of these orphan drugs; and (iii) determine the percentage contribution of these drugs to overall spending on drugs in each of these five countries in 2007. …

The rest of this article is only available to active members of Questia

Already a member? Log in now.

Notes for this article

Add a new note
If you are trying to select text to create highlights or citations, remember that you must now click or tap on the first word, and then click or tap on the last word.
One moment ...
Default project is now your active project.
Project items
Notes
Cite this article

Cited article

Style
Citations are available only to our active members.
Buy instant access to cite pages or passages in MLA 8, MLA 7, APA and Chicago citation styles.

(Einhorn, 1992, p. 25)

(Einhorn 25)

(Einhorn 25)

1. Lois J. Einhorn, Abraham Lincoln, the Orator: Penetrating the Lincoln Legend (Westport, CT: Greenwood Press, 1992), 25, http://www.questia.com/read/27419298.

Note: primary sources have slightly different requirements for citation. Please see these guidelines for more information.

Cited article

Global Spending on Orphan Drugs in France, Germany, the UK, Italy and Spain during 2007
Settings

Settings

Typeface
Text size Smaller Larger Reset View mode
Search within

Search within this article

Look up

Look up a word

  • Dictionary
  • Thesaurus
Please submit a word or phrase above.
Print this page

Print this page

Why can't I print more than one page at a time?

Help
Full screen
Items saved from this article
  • Highlights & Notes
  • Citations
Some of your highlights are legacy items.

Highlights saved before July 30, 2012 will not be displayed on their respective source pages.

You can easily re-create the highlights by opening the book page or article, selecting the text, and clicking “Highlight.”

matching results for page

    Questia reader help

    How to highlight and cite specific passages

    1. Click or tap the first word you want to select.
    2. Click or tap the last word you want to select, and you’ll see everything in between get selected.
    3. You’ll then get a menu of options like creating a highlight or a citation from that passage of text.

    OK, got it!

    Cited passage

    Style
    Citations are available only to our active members.
    Buy instant access to cite pages or passages in MLA 8, MLA 7, APA and Chicago citation styles.

    "Portraying himself as an honest, ordinary person helped Lincoln identify with his audiences." (Einhorn, 1992, p. 25).

    "Portraying himself as an honest, ordinary person helped Lincoln identify with his audiences." (Einhorn 25)

    "Portraying himself as an honest, ordinary person helped Lincoln identify with his audiences." (Einhorn 25)

    "Portraying himself as an honest, ordinary person helped Lincoln identify with his audiences."1

    1. Lois J. Einhorn, Abraham Lincoln, the Orator: Penetrating the Lincoln Legend (Westport, CT: Greenwood Press, 1992), 25, http://www.questia.com/read/27419298.

    Cited passage

    Thanks for trying Questia!

    Please continue trying out our research tools, but please note, full functionality is available only to our active members.

    Your work will be lost once you leave this Web page.

    Buy instant access to save your work.

    Already a member? Log in now.

    Search by... Author
    Show... All Results Primary Sources Peer-reviewed

    Oops!

    An unknown error has occurred. Please click the button below to reload the page. If the problem persists, please try again in a little while.