Background
Methods
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Publications on neglected and orphan diseases
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Participants in the Conference on Neglected Infectious Diseases, organized by the DG Research of the European Commission, Brussels, November 8 and 9, 2006, which AF attended http://ec.europa.eu/research/health/infectious-diseases/neglected-diseases/pdf/nid-conference-final-report052007_en.pdf
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Contributions to two online hearings (November 1-15, 2006 and August 15 - September 30, 2007) of the WHO Intergovernmental Working Group (WHO-IGWG) to develop a global strategy and plan of action for neglected diseases http://www.who.int/phi/public_hearings/en/
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A letter signed by 162 scientists, public health experts, lawyers, economists, government representatives and parliamentarians to accompany the submission of the Medical Research and Development Treaty draft to the WHO Commission on Intellectual Property, Innovation and Public Health in 2005.
Results
Participation, attrition, demographic data
Causes for the treatment deficit for neglected diseases
% (n) | % (n) | % (n) | % (n) | % (n) | ||||
---|---|---|---|---|---|---|---|---|
most important | important | unimportant | least important | no judgment | Total valid % (n) | Total N (Missing) | ||
No or insufficient sustainability of public funding for R&D for neglected diseases | Round I | 40,3% (48) | 47,9% (57) | 7,6% (9) | 0,8% (1) | 3,4% (4) | 100,0% (119) | 159 (40) |
Round II
|
54,1% (33)
|
41,0% (25)
|
3,3% (2)
|
1,6% (1)
|
0,0% (0)
|
100% (61)
|
77 (16)
| |
No or inadequate direct public funding for research and development (R&D) for neglected diseases | Round I | 35,2% (44) | 60,0% (75) | 3,2% (4) | 0,0% (0) | 1,6% (2) | 100,0% (125) | 159 (34) |
Round II
|
54,8% (34)
|
40,3% (25)
|
4,8% (3)
|
0,0% (0)
|
0,0% (0)
|
100% (62)
|
77 (15)
| |
No or inadequate incentives for the private sector to invest into R&D for neglected diseases | Round I | 33,9% (42) | 49,2% (61) | 8,9% (11) | 4,8% (6) | 3,2% (4) | 100,0% (124) | 159 (35) |
Round II
|
41,9% (26)
|
45,2% (28)
|
8,1% (5)
|
3,2% (2)
|
1,6% (1)
|
100% (62)
|
77 (15)
| |
No or inadequate private sector investment into R&D for neglected diseases | Round I | 33,6% (42) | 58,4% (73) | 4,8% (6) | 0,8% (1) | 2,4% (3) | 100,0% (125) | 159 (34) |
Round II
|
45,2% (28)
|
40,3% (25)
|
4,8% (3)
|
8,1% (5)
|
1,6% (1)
|
100% (62)
|
77 (15)
| |
No or inadequate access to effective drugs for neglected diseases | Round I | 33,1% (40) | 47,1% (57) | 9,1% (11) | 8,3% (10) | 2,5% (3) | 100,0% (121) | 159 (38) |
Round II
|
41,0% (25)
|
47,5% (29)
|
4,9% (3)
|
6,6% (4)
|
0,0% (0)
|
100% (61)
|
77 (16)
| |
No or inadequate research infrastructure in countries with neglected diseases | Round I | 29,5% (36) | 55,7% (68) | 7,4% (9) | 6,6% (8) | 0,8% (1) | 100,0% (122) | 159 (37) |
Round II
|
27,9% (17)
|
60,7% (37)
|
8,2% (5)
|
3,3% (2)
|
0,0% (0)
|
100% (61)
|
77 (16)
| |
No or ineffective drugs for neglected diseases | Round I | 20,5% (24) | 48,7% (57) | 15,4% (18) | 8,5% (10) | 6,8% (8) | 100,0% (117) | 159 (42) |
Round II
|
30,0% (18)
|
58,3% (35)
|
8,3% (5)
|
3,3% (2)
|
0,0% (0)
|
100% (60)
|
77 (17)
| |
Disease-specific research difficulties (unknown etiology, lack of research material) |
Round II
|
4,9% (3)
|
62,3% (38)
|
18,0% (11)
|
11,5% (7)
|
3,3% (2)
|
100% (61)
|
77 (16)
|
No or inadequate research coordination |
Round II
|
8,2% (5)
|
49,2% (30)
|
32,8% (20)
|
6,6% (4)
|
3,3% (2)
|
100% (61)
|
77 (16)
|
Lack of awareness/visibility of neglected diseases |
Round II
|
32,8% (20)
|
52,5% (32)
|
13,1% (8)
|
1,6% (1)
|
0,0% (0)
|
100% (61)
|
77 (16)
|
Lack of health-needs driven priority setting in public funding |
Round II
|
44,3% (27)
|
50,8% (31)
|
4,9% (3)
|
0,0% (0)
|
0,0% (0)
|
100% (61)
|
77 (16)
|
No or inadequate health delivery infrastructure and staff in developing countries |
Round II
|
45,9% (28)
|
41,0% (25)
|
9,8% (6)
|
1,6% (1)
|
1,6% (1)
|
100% (61)
|
77 (16)
|
Inadequate research priorities in private sector R&D |
Round II
|
48,4% (30)
|
37,1% (23)
|
11,3% (7)
|
3,2% (2)
|
0,0% (0)
|
100% (62)
|
77 (15)
|
Poverty as reason for market failure (perception of no market for drugs, insufficient R&D) |
Round II
|
55,0% (33)
|
35,0% (21)
|
5,0% (3)
|
3,3% (2)
|
1,7% (1)
|
100% (60)
|
77 (17)
|
Poverty as disease-proliferating factor (i.a. inadequate prevention, inadequate housing, lack of clean water) in endemic countries |
Round II
|
57,4% (35)
|
32,8% (20)
|
6,6% (4)
|
3,3% (2)
|
0,0% (0)
|
100%(61)
|
77 (16)
|
Orphan drug regulations for rare diseases
very effective | effective | ineffective | very ineffective | no judgment | |
---|---|---|---|---|---|
Orphan drug laws | 7.1% | 54.3% | 17.1% | 0% | 21.4% |
Market exclusivity | 22.1% | 33.8% | 17.6% | 4.4% | 22.1% |
Tax credits | 14.5% | 47.8% | 10.1% | 1.4% | 26.1% |
Protocol assistance | 13.2% | 41.2% | 14.7% | 0% | 30.9% |
Fee reduction/Fee waivers | 8.8% | 41.2% | 23.5% | 1.5% | 25.0% |
Measures to promote R&D for neglected diseases
% (n) | % (n) | % (n) | % (n) | % (n) | ||||
---|---|---|---|---|---|---|---|---|
very desirable | desirable | undesirable | very undesirable | no judgment | Total valid % (n) | Total N (Missing) | ||
Market exclusivity | Round I | 7,8% (9) | 19,1% (22) | 26,1% (30) | 20,9% (24) | 26,1% (30) | 100% (115) | 159 (44) |
Round II
|
3,6% (2)
|
20,0% (11)
|
36,4% (20)
|
21,8% (12)
|
18,2% (10)
|
100% (55)
|
77 (22)
| |
Tax credits | Round I | 14,2% (16) | 48,7% (55) | 6,2% (7) | 1,8% (2) | 29,2% (33) | 100% (113) | 159 (46) |
Round II
|
20,4% (11)
|
46,3% (25)
|
11,1% (6)
|
3,7% (2)
|
18,5% (10)
|
100% (54)
|
77 (23)
| |
Protocol assistance | Round I | 25,9% (30) | 44,0% (51) | 4,3% (5) | 0,0% (0) | 25,9% (30) | 100% (116) | 159 (43) |
Round II
|
33,9% (19)
|
55,4% (31)
|
3,6% (2)
|
0,0% (0)
|
7,1% (4)
|
100% (56)
|
77 (21)
| |
Fee reduction/Fee waivers (e.g. for marketing approval, scientific advice) | Round I | 27,0% (31) | 53,9% (62) | 3,5% (4) | 0,9% (1) | 14,8% (17) | 100% (115) | 159 (44) |
Round II
|
28,1% (16)
|
56,1% (32)
|
7,0% (4)
|
0,0% (0)
|
8,8% (5)
|
100% (57)
|
77 (20)
|
% (n) | % (n) | % (n) | % (n) | % (n) | ||||
---|---|---|---|---|---|---|---|---|
very (definitely) feasible | feasible | unfeasible | definitely unfeasible | no judgment | Total valid % (n) | Total N (Missing) | ||
Market exclusivity | Round I | 10% (11) | 28,2% (31) | 20,9% (23) | 2,7% (3) | 38,2% (42) | 110 | 159 (49) |
Round II
|
13.7% (7)
|
27.5% (14)
|
27.5% (14)
|
2.0% (1)
|
29.4% (15)
|
51
|
77 (26)
| |
Tax credits | Round I | 21.8% (24) | 40,0% (44) | 4,5% (5) | 2,7% (3) | 30,9% (34) | 110 | 159 (49) |
Round II
|
35.3% (18)
|
37.3% (19)
|
5.9% (3)
|
2.0 (1)
|
19.6% (10)
|
51
|
77 (26)
| |
Protocol assistance | Round I | 32.7% (36) | 44,5% (49) | 1.8% (2) | 0,0% (0) | 20.9% (23) | 110 | 159 (50) |
Round II
|
35,3% (18)
|
52.9% (27)
|
3.9% (2)
|
0.0% (0)
|
7.8% (4)
|
51
|
77 (26)
| |
Fee reduction/Fee waivers (e.g. for marketing approval, scientific advice) | Round I | 26,6% (29) | 51.4% (56) | 4.6% (5) | 0,9% (1) | 16.5% (18) | 110 | 159 (49) |
Round II
|
23.1% (12)
|
50.0% (26)
|
11.5% (6)
|
0.0% (0)
|
15.4% (8)
|
52
|
77 (25)
|
% (n) | % (n) | % (n) | % (n) | % (n) | ||||
---|---|---|---|---|---|---|---|---|
very desirable | desirable | undesirable | very undesirable | no judgment | Total valid % (n) | Total N (Missing) | ||
Prize funds with prizes awarded based on degree of innovation | Round I | 35.6% (42) | 44.1% (52) | 6.8% (8) | 3.4% (4) | 10.2% (12) | 118 | 159 (41) |
Round II
|
33.9% (19)
|
35.7% (20)
|
12.5% (7)
|
3.6% (2)
|
14.3% (8)
|
56
|
77 (21)
| |
Obligation for national governments to invest into neglected disease R&D | Round I | 48.3% (56) | 38.8% (45) | 6.9% (8) | 1.7% (2) | 4.3% (5) | 116 | 159 (43) |
Round II
|
40.4% (23)
|
36.8% (21)
|
14.0% (8)
|
1.8% (1)
|
7.0% (4)
|
57
|
77 (20)
| |
Separation of innovation incentives from drug prices | Round I | 38.8% (45) | 30.2% (35) | 4.3% (5) | 4.3% (5) | 22.4% (26) | 116 | 159 (43) |
Round II
|
38.2% (21)
|
27.3% (15)
|
12.7%(7)
|
5.5% (3)
|
16.4% (9)
|
55
|
77 (22)
|
% (n) | % (n) | % (n) | % (n) | % (n) | ||||
---|---|---|---|---|---|---|---|---|
definitely feasible | feasible | unfeasible | definitely unfeasible | no judgment | Total valid % (n) | Total N | ||
Prize funds with prizes awarded based on degree of innovation | Round I | 20.9% (23) | 60.0% (23) | 4.5% (5) | 0.9% (1) | 13.6% (15) | 110 | 159 |
Round II
|
30.8% (16)
|
48.1% (25)
|
11.5% (6)
|
1.9% (1)
|
7.7% (4)
|
52
|
77 (25)
| |
Obligation for national governments to invest into neglected disease R&D | Round I | 28.2% (31) | 41.8% (46) | 20.0% (22) | 5.5% (6) | 4.5% (5) | 110 | 159 (49) |
Round II
|
11.3% (6)
|
43.4% (23)
|
35.8% (19)
|
3.8% (2)
|
5.7% (3)
|
53
|
77 (24)
| |
Separation of innovation incentives from drug prices | Round I | 12.6% (14) | 39.6% (44) | 11.7% (13) | 6.3% (7) | 29.7% (33) | 111 | 159 (48) |
Round II
|
15.1% (8)
|
41.5% (22)
|
11.3% (6)
|
9.4% (5)
|
22.6% (12)
|
53
|
77 (24)
|
A regulatory instrument to promote R&D for neglected diseases
very desirable | desirable | undesirable | very undesirable | no judgment | n = | missing** | Median*** | |
---|---|---|---|---|---|---|---|---|
Desirability* | 44.2% (49.1%) | 44.2% (37.7%) | 7.1% (3.8%) | 1.8% (3.8%) | 2.7% (5.7%) | 113 (53) | 46 (24) | 2.00 (1.00) |
very (definitely) feasible | feasible | unfeasible | definitely unfeasible | no judgment | n | missing** | Median*** | |
Feasibility* | 14.2% (18.9%) | 63.7% (60.4%) | 8.0% (15.1%) | 3.5% (5.7%) | 10.6% (0.0%) | 113 (53) | 46 (24) | 2.00 (2.00) |