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09/10/20081
Doctoralseminar (9-10-2008)Thomas De Rijdt, PharmD
Orphandrugs
•Definitions•Legislation•Prevalenceand evolution•Slicing•Cost•Dispensing•Rawmaterials•Rare diseases: capita selecta•Conclusions
Definition: “Orphandisease”
•The term orphan disease implies two separate but relatedconcepts.
–To describe diseases that are overlooked by doctors, and has been', x, , ,renal cancer, high myopia, and even some common conditions, such asendometrial cancer and tobacco addiction. –
However, more specifically the term orphan disease is used todesignate diseases that affect only small numbers of individuals (so-called health orphans).
Aronson, Br J ClinPharmacol. 2006 March; 61(3): 243–245
Definition: “Orphandisease”
•Prevalence less than …
–USA: 1/200,000 –Japan: 1/50,000 –Australia: 1/2,000 –
Europe: 5/10,000
–WHO: 6.5-10/10,000
Aronson, Br J ClinPharmacol. 2006 March; 61(3): 243–245
Definition: “Orphandisease”
•A disease which has not been "adopted" by the pharmaceuticalindustry because it provides little financial incentive for theprivate sector to make and market new medications to treat orprevent it.
www.medicinenet.com accessed29-09-2008
•Listsof orphandiseases
–5000 –8000 knownrare disorders –Different organisations
•NORD : National organisationof rare diseases•NIH : National institutesof health•Eurordis: Europeanorganisationof rare diseases•Orphanet: Orphandrugs network•…
–Affecting6-8 % of the population
(EU : 25 millionpatients)
Definitionof “orphandrugs”
EMEA/290072/2007
•The term "orphan drug" refers to a product that treats a raredisease affecting fewer than 1 per200,000 Americans
www.fda.gov , accessed 29-09-2008
09/10/20082
Wheredo theycomefrom?
TaretMarket
•Intentionallydeveloped(eg Tasigna)•Unintentionallydeveloped(eg Thalidomide)
Designer drugsShelfdrugs
RIP Recreational/malafide use (e.g. PCP) Orphan drugs …
Legislation: orphandrug status
•USA : Orphandrug act (ODA)
–SinceJanuary1983 –
•Taxreductions•Extendedmarketing exclusivity(+ 7 years)
–Success?
•‘83 –’04 : 250 drugs authorised•‘73 –’83 : 10 suchdrugs onmarket
Haffner, N EnglJ Med. 2006 Feb2;354(5):445-7
Legislation: orphandrug status
•Europe: EMEA –COMP
–Committeeonorphanmedicinalproducts –
(
Verordening (EG) 141/2000 dd16-12-1999)
–Incentivesto encouragecompanies
•Protocol assistance•Financial benefits(fee reduction)•Marketing exclusivity(10 years)•Europeanregistration(all countries)
Procedure EMEA/COMP
•EMEA protocol assistance•Start at day1•StudybyCOMP•AdviceCOMP at day90
•Advicetot EC•Conclusionof EC
–Within30 days
•Publication
–A neworphandrug is born
–
Notyetauthorisedformarketing fordesired indication(otherprocedure)
OrphanEvolution2001-2007 : ATC
42%34%
ATC/Autorisation date 2001-2003
33%44%
ATC/Authoristion date 2001-2004
33%38%5%
ATC/authoristaion date 2001-2005
30%3%7%
ATC/authoristaion date 2001-2006
27%5%2%5%5%
ATC/authorisation date 2001-2007
8%8%8%
EnzymeAntithromboticAntihypertensiveHypothalamicAntineoplastic
5%6%6%6%
EnzymeAntithromboticCardiacAntihypertensiveHypothalamicAntineoplastic
4%5%5%5%5%
EnzymeAntithromboticCardiacAntihypertensiveUrologicalHypothalamicAntineoplasticAnalgesic
4%3%7%3%3%40%
EnzymeAntithromboticCardiacAntihypertensiveUrologicHypothalamicAntineoplasticAnalgesicOther
3%2%5%2%2%42%
EnzymeAntithromboticCardiacAntihypertensiveUrologicHypothalamicAntineoplasticImmunosuppressiveAnalgesicAntiepilepticaOther
•Itstartedwithenzymetherapy•Shift towardantineoplasticdrugs
09/10/20083
Authorisationper administrationroute
51%49%
OraalParenteraal
•No specialisedadministrationnecessaryin all cases.•Treatmentin ambulatorycare is possible.
Prevalence…
3.03.54.0
Prevalence/10.000
0.00.51.01.52.02.5
… slicingto orphandiseases
•Possibleto slice a diseaseintotypes•Ifprevalencesmallenough…orpansease.
–Leukemia
•Startedas 1 disease•AML, CML, ALL, CLL : commonknown•Now18 subtypes defined
–Different types recognizedas orphandiseaseand treatedwithorphandrugs (eg. chloretazine, oxaliplatin, …)
USA
Y. Waknine, 2004
Rocketscienceat high cost?
•Mostlyacademicresearch
–SubsidizedbyEC
(EMEA)
•
–Multinational, multicentricresearch –Lowerlevel of evidence(p<0.05 ??) –Case reports >> RCT
•Financial risk whennotprotected
Rocketscienceat high cost?
•High-techsynthetisation
–Enzymes: Aldurazyme, Myozyme, …
•Olderproducts, newname
–Thalidomide (Softenon)
•Existingmolecule, different route
–Pedea(= ibuprofen)
Rocketscienceat high cost?
•Simplemolecule, sameadministration
–Wilzin (= zincacetatecapsules)
•Treatmentof Wilson’sdisease
•Existingproduct, different name
–Hydroxycarbamide
•Hydreaversus Siklos
–Sildenafil
•Viagraversus Revatio
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