Business


Table 1: The most expensive specialty drugs (2017) Source: Drugs.com; https://www.drugs.com/slideshow/top-10-most-expensive-drugs-1274; October, 2017


DRUG NAME


Glybera (alipogene tiparvovec)


Ravicti (glycerol phenylbutyrate)


Brineura (cerliponase alfa)


Carbaglu (carglumic acid)


Lumizyme (alglucosidase alfa)


Actimmune (interferon gamma- 1b)


Soliris (eculizumab)


COMPANY uniQure


INDICATION


Lipoprotein lipase deficiency


Horizon Pharma Cycle Disorder BioMarin Pharmaceutical


Late infantile neuronal ceroid lipofuscinosis type 2 (CLN2) disease, a form of Batten disease


Orphan Europe N-acetylglutamate synthase deficiency


Genzyme Horizon Alexion Pompe disease.


Life-threatening osteopetrosis


Hemolytic uremic syndrome (aHUS)/ paroxysmal nocturnal hemoglobinuria (PNH)


Folotyn (pralatrexate) Allos Therapeutics Relapsed or refractory peripheral T-cell lymphoma (PTCL)


Demser, (metyrosine) Ilaris (canakinumab) Valeant


Pharmaceuticals Novartis


Pheochromocytoma


Periodic Fever Syndromes; Active Systemic Juvenile Idiopathic Arthritis


for these systems4. As our understanding of dis- eases has become more sophisticated and as dis- eases have, in some cases, become more complex and difficult to treat due to the combined influ- ences of unhealthy lifestyles, changing global envi- ronmental conditions and demographics, and con- stant exposure to harmful chemicals and infectious agents; and as important variables among the human population which affect both the nature and the experience of disease, such as gender-speci- ficity, ethnicity, age, among others, are taken into consideration for drug discovery and development, there is an ever greater need to find more effective


48


PRICE/Year $1.2 million


$793,000 $700,000


$419,000 - $790,000


$524,000 - $626,000


$244,000 - $572,000


$432,000 - $542,000


$96,000 - $472,000


$96,000 - $472,000


$379,000 - $462,000


ways to develop drugs. Unfortunately, achieving this goal is becoming more challenging every day given the increasing complexity of the process, the higher regulatory hurdles for drug approval, and the larger amount of information that needs to be taken into consideration and evaluated during the entire undertaking. In our consumerist and pharmaco-dependent


society, it is expected and demanded to have access to the best possible drugs with the lowest number of side-effects. However, like consumer goods, peo- ple get what they pay for. So, it is not surprising that access to the best drugs (for which more


Drug Discovery World Spring 2018


Page 1  |  Page 2  |  Page 3  |  Page 4  |  Page 5  |  Page 6  |  Page 7  |  Page 8  |  Page 9  |  Page 10  |  Page 11  |  Page 12  |  Page 13  |  Page 14  |  Page 15  |  Page 16  |  Page 17  |  Page 18  |  Page 19  |  Page 20  |  Page 21  |  Page 22  |  Page 23  |  Page 24  |  Page 25  |  Page 26  |  Page 27  |  Page 28  |  Page 29  |  Page 30  |  Page 31  |  Page 32  |  Page 33  |  Page 34  |  Page 35  |  Page 36  |  Page 37  |  Page 38  |  Page 39  |  Page 40  |  Page 41  |  Page 42  |  Page 43  |  Page 44  |  Page 45  |  Page 46  |  Page 47  |  Page 48  |  Page 49  |  Page 50  |  Page 51  |  Page 52  |  Page 53  |  Page 54  |  Page 55  |  Page 56  |  Page 57  |  Page 58  |  Page 59  |  Page 60  |  Page 61  |  Page 62  |  Page 63  |  Page 64  |  Page 65  |  Page 66  |  Page 67  |  Page 68  |  Page 69  |  Page 70  |  Page 71  |  Page 72