PBAC submissions and outcomes – 2014 in review

Michael Wonder – Wonder Drug Consulting Pty Ltd

www.maestrodatabase.com

Submissions

The PBAC considered 170 submissions of which 168 were prepared and lodged by the pharmaceutical industry (sponsors of medicines and medicinal preparations).

·      88 (52%) of the 133 submissions were major submissions and 82 (48%) were minor submissions

·      13 (77%) of the 133 submissions were initial submissions and 39 (23%) were resubmissions

Outcomes

The 170 submissions yielded 244 outcomes.  The number of PBAC outcomes from a given PBAC meeting or analysis period may be greater than the number of submissions for the same meeting or analysis period as some submissions may have contained multiple requests and/or multiple patient populations.

Of the 244 outcomes, 157 (64%) were recommendations, 57 (23%) were rejections and 30 (12%) were deferrals.

Submissions and outcomes for new medicines

The PBAC considered 47 submissions to list new medicines in 2014

·      32 (68%) of the 49 submissions were initial submissions and 15 (32%) were resubmissions

·      The 47 submissions yielded 48 outcomes: 24 (50%) recommendations, 18 (38%) rejections and 6 (12%) deferrals

The table below summarises the submissions for new listings and their related outcomes. The PBAC considered submissions for some medicines in the table prior to 2014.

© Wonder Drug Consulting Pty Ltd 2015.

New medicines considered by the PBAC in 2014

Medicine	Sponsor	Disease/condition	Number of submissions	PBAC outcomes	Comment
Aclidinium bromide (Bretaris Genuair)	Menarini	Chronic obstructive pulmonary disease	1	Recommendation	PBS listed – 1 Aug 2014
Alemtuzumab (Lemtrada)	Genzyme	Multiple sclerosis	2	Recommendation, rejection
Anakinra (Kineret)	Menarini	Cryopyrin-associated periodic syndrome	1	Recommendation
Axitinib (Inlyta)	Pfizer	Renal cell carcinoma	2	Rejection, recommendation
Betaine (Cystadane)	Emerge Health	Homocystinuria	1	Recommendation	PBS listed – 1 Aug 2014
Brentuximab vedotin (Adcetris)	Takeda	Non Hodgkin’s lymphoma	2	Recommendation, recommendation	PBS listed – 1 Dec 2014
Coal tar (Scytera)	Dr Reddys	Psoriasis	1	Recommendation
Collagenase clostridium histolyticum (Xiaflex)	Actelion	Dupuytren’s contracture	2	Rejection, recommendation
Crizotinib (Xalkori)	Pfizer	Non small-cell lung cancer	3	Deferral, deferral, recommendation
Elosulfase alfa (Vimizim)	BioMarin	Mucopolysaccharidosis type IVA	1	Rejection
Empagliflozin (Jardiance)	Boehringer Ingelheim	Type 2 diabetes mellitus	1	Recommendation	PBS listed – 1 Jan 2015
Enzalutamide (Xtandi)	Astellas	Prostate cancer	1	Recommendation	PBS listed – 1 Dec 2014
Fampridine (Fampyra)	Biogen Idec	Multiple sclerosis	2	Rejection, rejection
Febuxostat (Adenuric)	Menarini	Gout	1	Rejection	Resubmission to be considered by PBAC in Mar 2015
Ingenol mebutate (Picato)	Leo	Actinic keratosis	3	Rejection, rejection, rejection
Ivacaftor (Kalydeco)	Vertex	Cystic fibrosis	3	Deferral, recommendation, recommendation	PBS listed – 1 Dec 2014
Lisdexamfetamine dimesylate (Vyvanse)	Shire	Attention deficit hyperactivity disorder	2	Rejection, recommendation
Lixisenatide (Lyxumia)	GSK	Type 2 diabetes mellitus (dual combination)	1	Rejection
Lixisenatide (Lyxumia)	GSK	Type 2 diabetes mellitus (triple combination)	1	Rejection
Lurasidone hydrochloride (Latuda)	Dainippon Sumitomo	Schizophrenia	1	Rejection	Resubmission to be considered by PBAC in Mar 2015
Macitenan (Opsumit)	Actelion	Pulmonary arterial hypertension	1	Recommendation	PBS listed – 1 Sep 2014
Nitisinone (Orfadin)	Menarini	Tyrosinaemia	1	Deferral
Obinutuzumab (Gazyva)	Roche	Chronic lymphocytic leukaemia	1	Rejection	Resubmission to be considered by PBAC in Mar 2015
Ocriplasmin (Jetrea)	Alcon	Vitreomacular adhesion	1	Rejection
Ofatumumab acetate (Arzerra)	GSK	Chronic lymphocytic leukaemia	1	Recommendation
Olodaterol hydrochloride (Striverdi Respimat)	Boehringer Ingelheim	Chronic obstructive pulmonary disease	1	Rejection
Perampanel hemisesquihydrate (Fycompa)	Eisai	Epilepsy	1	Recommendation	PBS listed – 1 Nov 2014
Pertuzumab (Perjeta)	Roche	Breast cancer	2	Deferral, recommendation
Pomalidomide (Pomalyst)	Celgene	Multiple myeloma	2	Rejection, recommendation
Ponatinib hydrochloride (Iclusig)	Specialised Therapeutics	Chronic myeloid leukaemia	1	Deferral
Ponatinib hydrochloride (Iclusig)	Specialised Therapeutics	Acute lymphoblastic leukaemia	1	Deferral
Regorafenib monohydrate (Stivarga)	Bayer	Colorectal cancer	1	Rejection	Resubmission to be considered by PBAC in Mar 2015
Riociguat (Adempas)	Bayer	Pulmonary arterial hypertension	1	Recommendation
Ruxolitinib phosphate (Jakavi)	Novartis	Myelofibrosis	2	Rejection, deferral
Simeprevir sodium (Olysio)	Janssen-Cilag	Hepatitis C	1	Recommendation	PBS listed – 1 Dec 2014
Sofosbuvir (Sovaldi)	Gilead	Hepatitis C	1	Rejection	Resubmission to be considered by PBAC in Mar 2015
Sucroferric oxyhydroxide (Velphoro)	Vifor	Hyperphosphataemia	1	Recommendation
Taliglucerase alfa (Elelyso)	Pfizer	Gaucher disease	2	Rejection, rejection
Trametinib dimethyl sulfoxide (Mekinist)	GSK	Malignant melanoma	2	Rejection, recommendation
Umeclidinium bromide (Incruse Ellipta)	GSK	Chronic obstructive pulmonary disease	1	Recommendation	PBS listed – 1 Dec 2014
Vedolizumab (Entyvio)	Takeda	Ulcerative colitis	1	Rejection	Resubmission to be considered by PBAC in Mar 2015
Vortioxetine hydrobromide (Brintellix)	Lundbeck	Depression	1	Rejection