From: The impact of early phase price agreements on prices of orphan drugs
Parameter | Scenario | Price | |||
---|---|---|---|---|---|
Spinraza | Orkambi | ||||
Actual price (BE price) | €240,000 | €169,386 | |||
Scenario | BE price | change | BE price | change | |
Global market | large market | €175,768 | -€ 64,232 | €117,030 | -€52,356 |
Incidence (per 100,000) | min. - 20% | €318,000 | € 78,000 | €211,733 | €42,347 |
min. − + 20% | €212,000 | -€ 28,000 | €141,155 | -€28,231 | |
Off-label use | min. 2.5% (assumption) | €248,195 | € 8195 | €167,837 | -€1549 |
max. 5.9% (assumption) | €242,286 | € 2286 | €170,963 | €1577 | |
Growth population | min. 0% (assumption) | €290,743 | € 50,743 | €176,719 | €7333 |
max. 2.5% (assumption) | €226,133 | -€ 13,867 | €163,301 | -€6085 | |
Cost of capital | Pharma −9% | €185,261 | -€ 54,739 | €123,352 | -€46,034 |
Assumption 18% | €463,042 | € 223,042 | €308,305 | €138,919 | |
Probability success phase I-II | min. - 20% | €301,970 | € 61,970 | €201,060 | €31,674 |
min. − + 20% | €222,686 | -€ 17,314 | €148,270 | -€21,116 | |
Probability success phase II -III3 | min. - 20% | €313,044 | € 73,044 | €208,433 | €39,047 |
min. − + 20% | €215,304 | -€ 24,696 | €143,355 | -€26,031 | |
Probability success phase III - registration | min. - 20% | €317,383 | € 77,383 | €211,322 | €41,936 |
min. − + 20% | €212,411 | -€ 27,589 | €141,429 | -€27,957 | |
Probability reimbursement | min. (assumption) - 80% | €321,975 | € 81,975 | €214,379 | €44,993 |
max. (assumption) -100% | €206,064 | -€ 33,936 | €190,559 | €21,173 | |
Eligible | min. (assumption) - 80% | €286,200 | € 46,200 | €190,559 | €21,173 |
max. (assumption) -100% | €228,960 | -€ 11,040 | €152,447 | -€16,939 | |
Cost marketing | min. - 20% | €218,057 | -€ 21,943 | €145,188 | -€24,198 |
min. − + 20% | €305,280 | € 65,280 | €203,263 | €33,877 | |
Costs R&D - preclinical | min. - 20% | €225,700 | -€ 14,300 | €150,277 | -€19,109 |
min. − + 20% | €283,100 | € 43,100 | €188,495 | €19,109 | |
Costs R&D - phase I | min. - 20% | €245,044 | € 5044 | €163,157 | -€6229 |
min. − + 20% | €263,756 | € 23,756 | €175,615 | €6229 | |
Costs R&D - phase II | min. - 20% | €245,541 | € 5541 | €163,487 | -€5899 |
min. − + 20% | €263,259 | € 23,259 | €175,285 | €5899 | |
Costs R&D - phase III | min. - 20% | €250,929 | € 10,929 | €167,075 | -€2311 |
min. − + 20% | €257,871 | € 17,871 | €171,697 | €2311 | |
Costs R&D - phase IV | min. - 20% | €253,906 | € 13,906 | €169,057 | -€329 |
min. − + 20% | €254,894 | € 14,894 | €169,715 | €329 | |
Uptake | min. (assumption) - 90% | €290,743 | € 50,743 | €193,584 | €24,198 |
max. (assumption) - 95% | €226,133 | -€ 13,867 | €150,565 | -€18,821 | |
Proportion in clinical trials | min. (assumption) - 5% | €267,789 | € 27,789 | €178,301 | €8915 |
max. (assumption) - 10% | €279,560 | € 39,560 | €188,207 | €18,821 |