[Pharm-policy] Firms spent $7.7 million on clinical research per orphan drug approval
in 1997
James Love
love@cptech.org
Thu Apr 5 22:34:07 2001
Michael Palmedo has tracked down the most recent year of available data
on the Orphan Drug Tax Credit. The US government reported spending
$61.4 million on the credit in 1997. In 1997, there were 16 orphan
products that received FDA marketing approval, assuming you count the
three Somatropin indications sold by three different companies as one
product, and tbe two Liposomal indications as one, or 19 if you count
them separately.
The credit is equal to "50 percent of the qualified clinical testing
expenses," for drugs and biologics that qualify under the US Orphan Drug
Act. The statute is here:
http://www4.law.cornell.edu/uscode/26/45C.html. The 1998 data should be
available soon.
Based upon the credit of $61.4 million, the companies must have made
expenditures of $122.8 million, before taxes, for clinical research on
products that qualified as orphans. Note here that the credit is given
not only for products that are successful, BUT ALSO FOR PRODUCTS THAT
FAIL, as long has they have received Orphan status. Indeed, as the
links below illustrate, there are many more products that receive the
credit than those that are approved for marketing. Thus, the
expenditures per approval should be understood as a risk adjusted
figure, because this includes failures too.
Depending upon how you count the drugs with mulitple indications, this
comes to $6 to $8 mii million per approval, before the tax credit. Not
every approval is for a new chemical entity. For example, the
Paclitaxel approval is for the use of Taxol, a drug already on the
market to treat breast and ovarian cancer, for a new use of AIDS-related
Kaposi's sarcoma.
Given the costs to consumers of the Orphan Drug seven year marketing
exclusivity, the amount of investment by the private sector seems pretty
small. Indeed, the 1997 industry wide investments in orphan product
clinical testing of $61.4 million (after the tax credit), is equal to
about two weeks of Taxol sales. The 1997 Taxol orphan designation
delayed competition for Taxol for more than 30 months.
Jamie Love
--------------
Orphan Tax credit/Orphan approvals: 1997
Date: Thu, 05 Apr 2001 19:29:54 -0400
From: Mike Palmedo <mpalmedo@cptech.org>
Orphan tax credit expenditures and approvals:
YEAR TAX CREDIT ORPHANS APPROVED
1997 $61.4 million 16 (19 indications)
The full IRS reports on tax returns to corporations can be found on this
IRS webpage, and Table 21 of each report is where the expenditures on
the orphan tax credit are found:
http://www.irs.gov/tax_stats/soi/corp-complete.html
The complete list of "Orphan Designations With Market Approvals"
(arranged in alphabetical order - not by year) is found on the FDA site:
http://www.fda.gov/orphan/designat/recent.htm
The complete list of "Orphan Product Designations," which includes drugs
approved and not approved for marketing can be found here:
http://www.fda.gov/orphan/designat/list.htm
Following is two lists:
1. a short list of just the names of the 1997 orphan designations.
2. Detail on the 1997 orphan designations.
1997 ORPHAN DESIGNATIONS APPROVED FOR MARKETING
Anagrelide
Coagulation Factor IX
Daclizumab
Diazepam Viscous Solution for Rectal Administration
Fomepizole
Liposomal Amphotericin B (for two indications)
Oprelvekin
Paclitaxel
Rituximab
Sermorelin Acetate
Somatropin (for three indications)
Sterile Tac Powder
Tobramycin for inhalation
Toremifene
Ursodiol
Zinc Acetate
LIST OF ORPHANS WITH MORE INFORMATION: 1997
Generic Name: Anagrelide Trade Name: Agrylin
Orphan Indication: Treatment of essential thrombocythemia.
Sponsor: Roberts Pharmaceutical Corp. Contact: Mr. Drew Karlan
Address: Eatontown, NJ Phone: (732) 389-1182 Fax: ( ) -
Status: Designated/Approved Designation Date: 27-Jan-88 Marketing
Approval Date: 14-Mar-97
--------------------------------------------------------------------------------
Generic Name: Coagulation Factor IX (recombinant) Trade Name: BeneFix
Orphan Indication: Treatment of hemophilia B.
Sponsor: Genetics Institute, Inc. Contact: Dr. Frederick Gates
Address: Cambridge, MA Phone: (617) 503-7332 Fax:
Status: Designated/Approved Designation Date: 03-Oct-94 Marketing
Approval Date: 11-Feb-97
--------------------------------------------------------------------------------
Generic Name: Daclizumab Trade Name: Zenapax
Orphan Indication: Prevention of acute renal allograft rejection.
Sponsor: Hoffmann-La Roche, Inc. Contact: Ms. Ileana Leon Brown
Address: Nutley, NJ Phone: (201) 812-3720 Fax: (201) 812-3700
Status: Designated/Approved Designation Date: 05-Mar-93 Marketing
Approval Date: 10-Dec-97
--------------------------------------------------------------------------------
Generic Name: Diazepam viscous solution for rectal administration
Trade Name:
Orphan Indication: For the management of selected, refractory, patients
with epilepsy, on stable regimens of antiepileptic drugs (AEDs), who
require intermittent use of diazepam to control bouts of increased
seizure activity.
Sponsor: Athena Neurosciences, Inc. Contact: Ms. Kathleen Dumas
Address: South San Francisco, CA Phone: (415) 877-0900 Fax: (415)
877-8370
Status: Designated/Approved Designation Date: 25-Feb-92 Marketing
Approval Date: 29-Jul-97
--------------------------------------------------------------------------------
Generic Name: Fomepizole Trade Name: Antizole
Orphan Indication: Treatment of methanol or ethylene glycol poisoning.
Sponsor: Orphan Medical, Inc. Contact: Ms. Carol Curme
Address: Minnetonka, MN Phone: 9525136974 Fax: 9525419209
Status: Designated/Approved Designation Date: 22-Dec-88 Marketing
Approval Date: 04-Dec-97
--------------------------------------------------------------------------------
Generic Name: Liposomal amphotericin B Trade Name: AmBisome
Orphan Indication: Treatment of visceral leishmaniasis.
Sponsor: Fujisawa USA, Inc. Contact: Dr. Jerry Johnson
Address: Deerfield, IL Phone: (847) 317-8800 Fax: (847) 317-7286
Status: Designated/Approved Designation Date: 06-Dec-96 Marketing
Approval Date: 11-Aug-97
--------------------------------------------------------------------------------
Generic Name: Liposomal amphotericin B Trade Name: AmBisome
Orphan Indication: Treatment of cryptococcal meningitis.
Sponsor: Fujisawa USA, Inc. Contact: Dr. Jerry Johnson
Address: Deerfield, IL Phone: (847) 317-8800 Fax: (847) 317-7286
Status: Designated/Approved Designation Date: 10-Dec-96 Marketing
Approval Date: 11-Aug-97
--------------------------------------------------------------------------------
Generic Name: Oprelvekin Trade Name: Neumega
Orphan Indication: Prevention of severe chemotherapy-induced
thrombocytopenia.
Sponsor: Genetics Institute, Inc. Contact: Ms. Ingride Bartinique
Address: Cambridge, MA Phone: (617) 498-8652 Fax: (617) 498-8876
Status: Designated/Approved Designation Date: 17-Dec-96 Marketing
Approval Date: 25-Nov-97
--------------------------------------------------------------------------------
Generic Name: Paclitaxel Trade Name: Taxol
Orphan Indication: Treatment of AIDS-related Kaposi's sarcoma.
Sponsor: Bristol-Myers Squibb Pharmaceutical Research Contact: Ms.
Cheryl Anderson Institute
Address: Wallingford, CT Phone: (203) 284-6083 Fax: (203) 284-7630
Status: Designated/Approved Designation Date: 25-Mar-97 Marketing
Approval Date: 04-Aug-97
--------------------------------------------------------------------------------
Generic Name: Rituximab Trade Name: Rituxan
Orphan Indication: Treatment of non-Hodgkin's B-cell lymphoma.
Sponsor: IDEC Pharmaceuticals Corporation Contact: Ms. Alice Wei
Address: San Diego, CA Phone: (858) 431-8920 Fax: (858) 431-8889
Status: Designated/Approved Designation Date: 13-Jun-94 Marketing
Approval Date: 26-Nov-97
--------------------------------------------------------------------------------
Generic Name: Sermorelin acetate Trade Name: Geref
Orphan Indication: Treatment of idiopathic or organic growth hormone
deficiency in children with growth failure.
Sponsor: Serono Laboratories, Inc. Contact: Mr. Thomas Lang
Address: Norwell, MA Phone: (617) 982-9000 Fax: (617) 871-6754
Status: Designated/Approved Designation Date: 14-Sep-88 Marketing
Approval Date: 26-Sep-97
--------------------------------------------------------------------------------
Generic Name: Somatropin Trade Name: Humatrope
Orphan Indication: Treatment of short stature associated with Turner
syndrome.
Sponsor: Eli Lilly and Company Contact: Dr. Timothy Franson
Address: Indianapolis, IN Phone: (317) 276-2000 Fax:
Status: Designated/Approved Designation Date: 08-May-90 Marketing
Approval Date: 11-Mar-97
--------------------------------------------------------------------------------
Generic Name: Somatropin Trade Name: Genotropin
Orphan Indication: Treatment of adults with growth hormone deficiency.
Sponsor: Pharmacia & Upjohn Contact: Mr. Gregory Shawaryn
Address: Kalamazoo, MI Phone: (616) 833-8239 Fax: (616) 833-8237
Status: Designated/Approved Designation Date: 06-Sep-94 Marketing
Approval Date: 31-Oct-97
--------------------------------------------------------------------------------
Generic Name: Somatropin for injection Trade Name: Nutropin
Orphan Indication: As replacement therapy for growth hormone deficiency
in adults after epiphyseal closure.
Sponsor: Genentech, Inc. Contact: Dr. M. David MacFarlane
Address: South San Francisco, CA Phone: (415) 225-1000 Fax: (415)
225-6000
Status: Designated/Approved Designation Date: 18-Nov-96 Marketing
Approval Date: 15-Dec-97
--------------------------------------------------------------------------------
Generic Name: Sterile talc powder Trade Name: Sclerosol Intrapleural
Aerosol
Orphan Indication: Treatment of malignant pleural effusion.
Sponsor: Bryan Corporation Contact: Mr. Frank Abrano
Address: Woburn, MA Phone: (781) 935-0004 Fax: (781) 935-7602
Status: Designated/Approved Designation Date: 18-Sep-95 Marketing
Approval Date: 24-Dec-97
--------------------------------------------------------------------------------
Generic Name: Tobramycin for inhalation Trade Name: TOBI
Orphan Indication: Treatment of bronchopulmonary infections of
Pseudomonas aeruginosa in cystic fibrosis patients.
Sponsor: Pathogenesis Corporation Contact: Dr. A. Bruce Montgomery
Address: Seattle, WA Phone: (206) 467-8100 Fax: (206) 282-5065
Status: Designated/Approved Designation Date: 13-Oct-94 Marketing
Approval Date: 22-Dec-97
--------------------------------------------------------------------------------
Generic Name: Toremifene Trade Name: Fareston
Orphan Indication: Hormonal therapy of metastatic carcinoma of the
breast.
Sponsor: Orion Corporation Contact: Dr. Daniel Azarnoff
Address: Finland, Phone: (415) 340-8222 Fax:
Status: Designated/Approved Designation Date: 19-Sep-91 Marketing
Approval Date: 29-May-97
--------------------------------------------------------------------------------
Generic Name: Ursodiol Trade Name: URSO
Orphan Indication: Treatment of patients with primary biliary cirrhosis.
Sponsor: Axcan Pharma Inc. Contact: Ms. Quyen Vinh
Address: Canada, CA Phone: 4504675138 Fax: (514) 669-5161
Status: Designated/Approved Designation Date: 20-Jun-91 Marketing
Approval Date: 10-Dec-97
--------------------------------------------------------------------------------
Generic Name: Zinc acetate Trade Name: Galzin
Orphan Indication: Treatment of Wilson's disease.
Sponsor: Lemmon Company Contact: Ms. Deborah Jaskot
Address: Kulpsville, PA Phone: (215) 256-8400 Fax: (215) 721-8796
Status: Designated/Approved Designation Date: 06-Nov-85 Marketing
Approval Date: 28-Jan-97
--
James Love
Consumer Project on Technology
http://www.cptech.org
1.202.380.3080 fax 1.202.234.5176
mailto:love@cptech.org