Background
Methods
Data sources
Selection of diseases and medications
Survey and selection of medical facilities
Ethics
Measures and analysis
Availability
-
Absent, 0 % of facilities: these orphan drugs were not found in any facilities surveyed;
-
Very low, < 30 % of facilities: these orphan drugs were very difficult to find;
-
Low, 30–49 % of facilities: these orphan drugs were somewhat difficult to find;
-
Fairly high, 50–80 % of facilities: these orphan drugs were available in many facilities;
-
High, >80 % of facilities: these orphan drugs had good availability.
Affordability
Results
Market availability of orphan drugs in China
Regions (Time range) | Number of orphan drug designations | Number of orphan drug approvals for marketing | Number of unique orphan drugs approved for marketinga (A) | Number of unique orphan drugs marketed in Chinaa (B) | Market availability rate in China [(B)/(A) × 100 %] |
---|---|---|---|---|---|
U.S. (1983–2012.12.31) | 2741 | 425b
| 315 | 119 | 37.8 |
EU (2000–2012.12.31) | 1098 | 65 | 65 | 16 | 24.6 |
Japan (1993–2012.12.31) | 300 | 185c
| 145 | 76 | 52.4 |
Total | 4132 | 675 | 525 | 211 | 40.2 |
408a
| 165a
| 40.4 |
ATC Category | Orphan drugs of China/U.S. | Orphan drugs of China/EU | Orphan drugs of China/Japan | Orphan drugs of China/U.S. + EU + Japanb
| ||||
---|---|---|---|---|---|---|---|---|
N | % | N | % | N | % | N | % | |
A-Alimentary tract and metabolism | 6/42 | 14.3 | 1/12 | 8.3 | 6/19 | 31.6 | 10/43 | 23.3 |
B-Blood and blood forming organs | 7/40 | 17.5 | 1/3 | 33.3 | 4/11 | 36.4 | 9/31 | 29.0 |
C-Cardiovascular system | 6/9 | 66.7 | 1/5 | 20.0 | 4/7 | 57.1 | 6/13 | 46.2 |
D-Dermatologicals | 1/4 | 25.0 | 0/0 | - | 0/0 | - | 1/4 | 25 |
G-Genito-urinary system and sex hormones | 8/10 | 80.0 | 0/1 | 0 | 1/2 | 50 | 7/10 | 70 |
H- Systemic hormonal preparations, excluding sex hormones and insulins | 15/36 | 41.6 | 1/3 | 33.3 | 3/7 | 42.9 | 9/19 | 47.3 |
J- Anti-infectives for systemic use | 12/32 | 37.5 | 0/2 | 0 | 28/43 | 65.1 | 32/58 | 55.2 |
La- Antineoplastic and immunomodulating agents | 53/136 | 39.0 | 10/28 | 35.7 | 40/66 | 60.6 | 55/124 | 44.4 |
L1 for blood cancers | 20/45 | 44.4 | 6/13 | 46.2 | 12/18 | 66.7 | 23/45 | 51.1 |
L2 for other cancers | 23/43 | 53.5 | 4/9 | 44.4 | 6/12 | 50.0 | 16/35 | 45.7 |
M- Musculoskeletal system | 5/12 | 41.7 | 0/0 | - | 2/7 | 28.6 | 6/12 | 50.0 |
N- Nervous system | 9/31 | 29.0 | 1/6 | 16.7 | 3/11 | 27.3 | 10/33 | 30.3 |
P- Antiparasitic products, insecticides and repellents | 4/17 | 23.5 | 0/0 | - | 2/3 | 66.7 | 5/12 | 41.7 |
R- Respiratory system | 2/10 | 20 | 0/2 | 0 | 1/3 | 33.3 | 2/9 | 22.2 |
S- Sensory organs | 2/8 | 25 | 0/0 | - | 1/4 | 25 | 5/12 | 41.7 |
V- Various | 9/34 | 26.5 | 1/3 | 66.7 | 1/2 | 50 | 8/28 | 28.6 |
TOTAL | 139/421c
| 33.0 | 16/65 | 24.6 | 96/185 | 52.4 | 165/408 | 40.4 |
Coefficient of Variation(C.V.) | - | 53.7 | - | 84.5 | - | 33.8 | - | 35.1 |
ATC Code | Generic name (Brand name) | Indication | Year marketing authorization received | Delay of release in China (in years) Compared to the earliest time | |||
---|---|---|---|---|---|---|---|
China | U.S.a
| EUa
| Japana
| ||||
A16AB02 | Imiglucerase (Cerezyme)b P | GD | 2008 | 1994 |
d
| 1998 | 14 |
A16AX07 | Sapropterin Dihydrochloride (Kuvan)b P | PKU | 2010 | 2007 | 2008 | -- | 3 |
B02BD02 | Human coagulation factor VIIIe
| HEM | 2002 | -- D | -- | -- | -- |
B02BD02 | Recombinant coagulation factor VIII (Kogenate FS) | 2010 | -- D | -- D | -- | -- | |
-- | Human prothrombin complex concentrate (e, Kcentra) | 2002 | 2013 | -- | -- | −11 | |
B02BD04 | Coagulation factor IX ( Benefix)c
| 2012 | 1997 | D, d
| 2009 | 15 | |
B02BD05 | Coagulation factor VIIa ( Novoseven) | 2010 | 1999 | D, d
| 2000 | 11 | |
H01BA02 | Desmopressin acetate spray (e, Stimste) | 2001 | 1994 | -- | -- | 7 | |
C02KX01 | Bosentan (Tracleer)b
| PAH | 2011 | 2001 | 2002 | 2005 | 10 |
B01AC19 | Beraprost sodium (Dorner) | 2008 | -- D | -- D | 1999 | 9 | |
B01AC11 | Iloprost inhalational solution (Ventavis)b
| 2007 | 2004 | 2003 | -- | 5 | |
C02KX02 | Ambrisentan (Volibris/Letairis)b
| 2010 | 2007 | 2008 | 2010 | 3 | |
B01AC21 | Treprostinil (Remodulin)b P | 2014 | 2002 | -- | -- | 12 | |
G04BE08 | Tadalafil (Cialis® / Adcirca®) | 2009 | 2009 |
d
| -- | 0 | |
G03XA01 | Danazol e
| HAE | 2002 | -- | -- | -- | -- |
H01AC01 | RDNA origin somatropin (e,Norditropin SimpleXx/Norditropin) | GHD | 1999 | 2000 | -- | 1997 | 2 |
H01AC01 | Somatropin (Saizen) | 2000 | 1996 | -- | -- | 4 | |
H01AC01 | Somatropin (Genotropin) | 2000 | 1997 | -- | 1997 | 3 | |
H01AC01 | Somatropin (Humatrope) P | 2002 | 1987 | -- | -- | 15 | |
-- | Recombinant human growth hormonee
| 2006 | -- |
d
| -- | -- | |
L01AB01 | Busulfan (Busulfex/Busilvex ) P | CML | 2002 | 1999 | 2003 | 2006 | 3 |
L01XE01 | Imatinib (e, Glivec/Gleevec)b P | 2010 | 2001 |
d
| 2001 | 9 | |
L01XE06 | Dasatinib (e, Sprycel)b P | 2011 | 2006 | 2006 | 2009 | 5 | |
L01XE08 | Nilotinib (Tasigna)b
| 2009 | 2007 | 2007 | 2009 | 2 | |
-- | Meisoindigo | 2010 | -- | -- | -- | -- | |
L01BB02 | Mercaptopurine (e, Xaluprine) | ALL | 2002 | 2014 | 2012 | -- | - 10 |
L01AA01 | Cyclophosphamide (e, Endoxan) | 2001 | -- D | -- | 2003 | - 2 | |
L01CB02 | Teniposide (e, Vumon)b P | 1999 | 1992 | -- | -- | 7 | |
L01XX24 | Pegaspargase (e, Oncaspar)c
| 2013 | 1994 | -- | -- | 19 | |
L01XE01 | Imatinib (e, Gleevec / Glivec) | 2010 | 2006 |
d
| 2001 | 9 | |
L01XE06 | Dasatinib (e, Sprycel) | 2011 | 2006 | 2006 | 2009 | 5 | |
L01DB07 | Mitoxantrone HCl (e, Novantrone)b
| AML | 2001 | 1987 | -- | -- | 14 |
L01DB06 | Idarubicin HCl (e, Zavedos/Idamycin)b P | 2002 | 1990 | -- | -- | 12 | |
L01BC01 | Cytarabine (e, Cylocide N) | 2000 | -- | -- | 2000 | 0 | |
-- | Homoharringtonine | 2002 | -- D | -- D | -- | -- | |
-- | Arsenious Acid (Yitaida) | APL | 2010 | -- | -- | -- | -- |
L01XX27 | Arsenic Trioxide (e,Trisenox)b P | 2008 | 2000 |
d
| -- | 8 | |
L01XX14 | Tretinoin (e,Vesanoid) P | 2002 | 1995 | -- | 1995 | 7 | |
-- | Homoharringtonine | 2002 | -- D | -- D | -- | -- | |
L01XC02 | Rituximab (MabThera/Rituxan)c
| NHL | 2000 | 1997 |
d
| 2001 | 3 |
L01XE05 | Sorafenib (Nexavar)b P | RCC | 2011 | 2005 | 2006 | -- | 6 |
L01XC07 | Bevacizumab (Avastin) | 2012 | 2009 |
d
| -- | 3 | |
N07XX02 | Riluzole (e, Rilutek)b P | ALS | 2010 | 1995 |
d
| 1998 | 15 |
R07AA | Poractant alfa (Curosurf)b
| PIRDS | 2000 | 1999 | -- | -- | 1 |
-- | Calf pulmonary surfactant (Kelisu) | 2010 | -- | -- | -- | -- |
The availability of orphan drugs at a hospital level
Availability | Range | Originator Brands (Brand Name) | Generics |
---|---|---|---|
Absent | 0 | Ambrisentan (Volibris), Somatropin (Genotropin), Dasatinib (Sprycel), Meisoindigo | None |
Very low | <30 % | Imiglucerase (Cerezyme), Sapropterin dihydrochloride (Kuvan), Coagulation factor VIIa (Novoseven), Bosentan (Tracleer), Iloprost (Ventavis), Somatropin (Saizen), Somatropin (Humatrope), Busulfan(Busulfex), Imatinib Mesylate Cap (Glivec), Nilotinib (Tasigna), Riluzole ( Rilutek) | Teniposide, Riluzole taba, Riluzole capa
|
Low | 30–49 % | Recombinant coagulation factor VIII (Kogenate FS), Imatinib mesylate Tab (Glivec), Arsenious acid(Yitaida), Sorafenib (Nexavar) | Human coagulation factor VIII, Danazol |
Fairly high | 50–80 % | Teniposide (Vumon), Rituximab (MabThera), Poractant alfa (Curosurf) | Human prothrombin complex concentrate, Recombinant human growth hormone, Mitoxantrone, Homoharringtonine |
High | 80 % | None | None |
The affordability of orphan drugs
Generic name (Brand name) | Median Unit PriceA (USD) | Daily Dose | Duration of Treatment Course (days) | Total Course Cost (USD) | Days of per capita net income | Coverage of NBMI (Y/N) | Affordability (if 5 % OOP) | ||||||
---|---|---|---|---|---|---|---|---|---|---|---|---|---|
Urban Resident | Affordability | Rural Resident | Affordability | ||||||||||
Ave (Y/N) | Urban High (Y/N) | ||||||||||||
Ave3
| High4
| Y/N | Ave | High | Y/N | ||||||||
Recombinant human coagulation factor VII (Kogenate FS) | 0.85/IU | 1750 IU | 2 | 2961.5 | 309.1 | 114.6 | N | 967.8 | 401.8 | N | Y, Part B | N | N |
Recombinant human coagulation factor VIIa (NovoSeven)B
| 869.02/mg | 50.4 mg | 1 | 43798.7 | 4571.9 | 1695.0 | N | 14313.3 | 5942.8 | N | N | N | N |
Bosentan (Tracleer) | 0.75/mg | 250 mg | 28 | 5272.4 | 550.4 | 204.0 | N | 1723.0 | 715.4 | N | N | N | N |
Iloprost (Ventavis) | 4.38/ug | 50ug | 28 | 6129.5 | 639.8 | 237.2 | N | 2003.1 | 831.7 | N | N | N | N |
Somatropin (Saizen) | 10.89/IU | 7 IU | 28 | 2134.3 | 222.8 | 82.6 | N | 697.5 | 289.6 | N | N | N | N |
Busulfan (Busulfex) | 4.66/mg | 224 mg | 4 | 4178.5 | 436.2 | 161.7 | N | 1365.5 | 567.0 | N | Y, Part B | N | N |
Imatinib (Glivec) | 0.32/mg | 600 mg | 28 | 5384.6 | 562.1 | 208.4 | N | 1759.7 | 730.6 | N | N | N | N |
Imatinib (Glivec) | 0.32/mg | 600 mg | 28 | 5438.5 | 567.7 | 210.5 | N | 1777.3 | 737.9 | N | N | N | N |
Nilotinib (Tasigna) | 0.26/mg | 800 mg | 28 | 5779.5 | 603.3 | 223.7 | N | 1888.7 | 784.2 | N | N | N | N |
Teniposide (Vumon) | 0.56/mg | 102 mg | 5 | 286.1 | 29.9 | 11.1 | N | 93.5 | 38.8 | N | N | N |
Y
|
Arsenious acid (Yitaida) | 2.32/mg | 10 mg | 28 | 650.6 | 67.9 | 25.2 | N | 212.6 | 88.3 | N | Y, Part B#
| N | N |
Rituximab (MabThera) | 6.1/mg | 91.1 mg | 28 | 15558.2 | 1624.0 | 602.1 | N | 5084.4 | 2111.0 | N | N | N | N |
Sorafenib tosylate (Nexavar) | 0.33/mg | 800 mg | 28 | 7502.6 | 783.2 | 290.3 | N | 2451.8 | 1018.0 | N | N | N | N |
Poractant alfa (Curosurf)C
| 4.8/mg | 525 mg | 1 | 2456.7 | 256.4 | 95.1 | N | 802.8 | 333.3 | N | Y, Part B | N | N |
Human coagulation factor VII | 0.3/IU | 1750 IU | 2 | 1065.7 | 111.2 | 41.2 | N | 348.3 | 144.6 | N | Y, Part A | N | N |
Human prothrombin complex concentrate | 0.15 IU | 3150 IU | 2 | 969.2 | 101.2 | 37.5 | N | 316.7 | 131.5 | N | Y, Part B#
| N | N |
Danazol | 0.004/mg | 600 mg | 28 | 61.9 | 6.5 | 2.4 | N | 20.2 | 8.4 | N | Y, Part B | Y |
Y
|
Recombinant human growth hormone | 3.7/IU | 7 IU | 28 | 725.6 | 75.7 | 28.1 | N | 237.1 | 98.5 | N | Y, Part B | N | N |
Teniposide | 0.44/ mg | 102 mg | 5 | 223.1 | 23.3 | 8.6 | N | 72.9 | 30.3 | N | Y, Part B | N |
Y
|
Mitoxantrone | 1.24/mg | 10.2 mg | 4 | 50.6 | 5.3 | 2.0 | N | 16.5 | 6.9 | N | Y, Part B | Y |
Y
|
Riluzole | 0.16/mg | 100 mg | 28 | 453.2 | 47.3 | 17.5 | N | 148.1 | 61.5 | N | N | N |
Y
|
Riluzole | 0.11/mg | 100 mg | 28 | 309.6 | 32.3 | 12.0 | N | 101.1 | 42.0 | N | N | N |
Y
|
Homoharringtonine | 0.75/mg | 2.5 mg | 5 | 9.4 | 0.98 | 0.4 | Y | 3.1 | 1.3 | N | Y, Part A#
| Y |
Y
|
Average | -- | -- | 17.2 | 4843.5 | 505.6 | 187.4 | -- | 1582.8 | 657.2 | -- | -- | -- |
--
|