Orphan drug

[Guest guest]

I recently attended a seminar about global regulatory compliance and

thought that this may be useful at some point.

The Orphan drug legisltation is designed to encourage pharmaceutical

companies to research rare diseases. I am not sure if PSC would meet

these criteria or if it is now more common than this.

Does anyone know what the current estimate is for the number of

people in the US with PSC?

http://www.orphan-

europe.com/fe/orphan/jsp/orphan_druglegislations.html

The Orphan drug legislations

US: Orphan Drug Act 1983

Japan: Orphan Drug Legislation 1993

Australia: Orphan Drug Program 1998

EU: Orphan Drug Legislation 2000

Criteria for Orphan Designation (prevalence)

US: less than 200,000 patients/year corresponding to 7.5/10,000

inhabitants

Japan: less than 50,000 patients/year corresponding to 4.0/10,000

inhabitants

Australia: less than 2,000 patients/year corresponding to 1.1/10,000

inhabitants

EU: less than 5.0/10,000 inhabitants corresponding to 185,000

patients/year

Prevalence: less than 5 per 10,000 inhabitants

Economic or epidemiological criteria

10 years market exclusivity for the therapeutic indication

Creation of a Committee for Orphan Medicinal Products

Access to centralised procedure for licensing

Protocol assistance

http://rarediseases.about.com/gi/dynamic/offsite.htm?

zi=1/XJ & sdn=rarediseases & cdn=health & tm=50 & gps=138_1352_800_558 & f=10 & su

=p284.21.140.ip_p284.2.420.ip_ & tt=2 & bt=0 & bts=0 & zu=http%

3A//www.fda.gov/orphan/faq/index.htm

How much money is available for grants?

The current annual budget for funding grants is approximately $13

million. Clinical trials are awarded grants from $200,000 (Phase 1)

and $350,000 (Phase 2 and 3) per year in total costs for up to 3

years. The annual Request for Application (RFA), published in the

Federal Register will provide more up-to-date information on dollar

limits.

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How are awards made?

The number of grant awards varies each year depending on the

availability of funds. On-going studies are funded first with the

remainder of funds going to new studies. In recent years, OOPD has

funded approximately 12-15 new awards annually.

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Who may apply?

Academic institutions and other responsible organizations: public,

private, non-profit, or for-profit. Small businesses are encouraged

to apply.

Kind Regards,

Nichole Rowland

(wife of PSC & UC 08/2004)

[Guest guest]

Hi Nichole;

From: Bambha K, Kim WR, Talwalkar J, Torgerson H, Benson JT, Therneau

TM, Loftus EV Jr, Yawn BP, Dickson ER, Melton LJ 3rd 2003 Incidence,

clinical spectrum, and outcomes of primary sclerosing cholangitis in

a United States community. Gastroenterology 125: 1364-1369.

" The prevalence of PSC in 2000 was 20.9 per 100,000 men (95% CI, 9.5

to 32.4) and only 6.3 per 100,000 women (95% CI, 0.1 to 12.5). "

If there are 150 million men in the U.S. and IF this assessment of

Olmsted county, MN, applies to the entire U.S., then there would be

20.9 x 1500 = 31,350 men with PSC. Similarly if there are 150 million

women in the U.S. and IF this assessment of Olmsted county, MN,

applies to the entire U.S., then there would be 6.3 x 1500 = 9,450

women with PSC. This gives a total of 40,800 persons affected in the

U.S.

However, Arne has noted that the population in Olmsted county, MN,

may be mainly of Scandinavian origin/ancestry, and if this group has

a higher prevalence of PSC than other nationalities, then it may be

an overestimate. Certainly, one could say that there is likely to be

no more than 41,000 patients in the U.S., and so it would qualify as

an orphan disease.

Best regards,

Dave

(father of (21); PSC 07/03; UC 08/03)