G-BA benefit assessment of new orphan drugs in Germany: the first five years

Figures & data

Table 1. G-BA extent of additional benefit for orphan and non-orphan drugs in the period 2011–2015.

	Orphan drugs	Non-orphandrugs
Additional benefit	28 (100%)	54 (46.2%)
Major	0	2 (1.7%)
Considerable	3 (10.7%)	27 (23.1%)
Minor	13 (46.4%)	21 (17.9%)
Non-quantifiable	12 (42.9%)	4 (3.4%)
Additional benefit not proved	0	63 (53.8%)
No additional benefit	Not applicable^a	59 (50.4%)
Reference price group	0	4 (3.4%)
Less benefit than appropriate comparator	Not applicable^a	0
Total assessments	28	117

^a For orphan drugs below €50 million.

Table 2. Chronologic list of AMNOG benefit assessment procedures for orphan drugs in the period 2011–2015.

Active ingredient	Brand name	Therapeutic indication(s)	Resolution date	Additional benefit	Estimated GKV patients
Pirfenidone	Esbriet^®	Idiopathic pulmonary fibrosis	15 March 2012	Non-quantifiable	6000
Tafamidis meglumine	Vyndaqel^®	Familial amyloid polyneuropathy	7 June 2012	Minor	40–104
Pasireotide	Signifor^®	Cushing’s disease	6 December 2012	Minor	160–360
Ivacaftor	Kalydeco™	Cystic fibrosis	7 February 2013	Considerable	170
Ruxolitinib	Jakavi^®	Myeloproliferative disorders	7 March 2013	Minor	1600
Decitabine	Dacogen^®	Myeloid leukemia	2 May 2013	Minor	300–780
Brentuximab vedotin	Adcetris^®	Hodgkin and non-Hodgkin lymphoma	16 May 2013	Non-quantifiable	60–260; 15–160
Bosutinib^a	Bosulif^®	Myeloid leukemia	17 October 2013	Non-quantifiable	380–500
Ponatinib^a	Iclusig^®	Myeloid and acute lymphoid leukemia	23 January 2014	Non-quantifiable	500–940; 25–195
Pomalidomide	Imnovid^®	Multiple myeloma	20 February 2014	Considerable	1900
Macitentan	Opsumit^®	Pulmonary hypertension	17 July 2014	Minor	580–7850
Riociguat	Adempas^®	Pulmonary hypertension	16 October 2014	Minor	580–7850; 920–5460
Cholic acid	Orphacol^®	Inborn errors in primary bile acid synthesis	6 November 2014	Non-quantifiable	10–25
Ruxolitinib (>€50 million)^b	Jakavi^®	Myeloproliferative disorders	6 November 2014	Considerable	1600–5000
Elosulfase alfa	Vimizim^®	Mucopolysaccharidosis IVA	20 November 2014	Minor	15–95
Siltuximab	Sylvant^®	Giant lymph node hyperplasia	4 December 2014	Non-quantifiable	130–1460
Cabozantinib^a	Cometriq^®	Thyroid cancer	22 January 2015	Minor	60–500
Obinutuzumab	Gazyvaro™	Chronic lymphocytic leukemia	5 February 2015	Non-quantifiable	818–1477
Teduglutide	Revestive^®	Malabsorption syndromes	19 February 2015	Minor	1100–2400
Ivacaftor (new indication)	Kalydeco™	Cystic fibrosis	19 February 2015	Minor	10–11
Ibrutinib	Imbruvica^®	Mantle-cell lymphoma; chronic lymphocytic leukemia	16 April 2015	Non-quantifiable	700–950; 2200–7800
Alipogene tiparvovec^a	Glybera^®	Lipoprotein lipase deficiency	21 May 2015	Non-quantifiable	17–35
Ataluren^a	Translarna™	Duchenne muscular dystrophy	21 May 2015	Minor	82–110
Pasireotide (new indication)	Signifor^®	Acromegaly	18 June 2015	Minor	265–1133
Ramucirumab	Cyramza^®	Stomach cancer	16 July 2015	Minor	5900–7900
Nintedanib	Ofev^®	Idiopathic pulmonary fibrosis	3 September 2015	Minor	2080–19,700
Eliglustat	Cerdelga^®	Gaucher’s disease	1 October 2015	Non-quantifiable	150–500
Olaparib^a	Lynparza™	Ovarial cancer	27 November 2015	Non-quantifiable	818–1477
Lenvatinib	Lenvima^®	Thyroid cancer	17 December 2015	Non-quantifiable	600–815

^a Time-limited resolution.

^b This resolution replaced the first one.