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Medicines Milestones A decade of persistence, prevention and promise MEDICINES MILESTONES 2 A decade of persistence, prevention and promise

Table of Contents

1 Foreword 3 2 Executive summary 4 2.1 Breakthrough medicines make their mark 4 2.2 Biological medicines and targeted medicines – the way of the future 5 3 Decade trends 6 3.1 Number of medicines 6 3.2 Number of new molecules ...... 12 3.3 New medicines by areas and conditions ...... 12 3.4 Number of prevention (including ) 16 4 National Health Priority Areas 17 4.1 Number of TGA approvals 17 4.2 Cancer focus areas ...... 18 4.2.1 Number of approvals each year over 10 years 18 4.2.2 Split between focus areas ...... 18 4.2.3 Focus vs non-focus areas ...... 19 4.3 Arthritis and musculoskeletal conditions ...... 20 4.3.1 Number of approvals each year over 10 years 20 4.3.2 Split between focus areas ...... 21 4.4 Cardiovascular health 21 4.4.1 Number of approvals each year over 10 years 21 4.4.2 Split between focus areas ...... 22 4.5 Diabetes focus areas 23 4.5.1 Number of approvals each year over 10 years 23 4.6 Depression (this is the only mental health National Health Priority Area) ...... 24 4.6.1 Number of approvals each year over 10 years 24 4.6.2 Focus vs non-focus areas ...... 25 4.7 Asthma 25 4.7.1 Number of approvals each year over 10 years 25

5 Vaccines 27 6 The way forward 28 7 Background and methods 32 7.1 Project background ...... 32 7.1.1 Pretium’s Tracker database ...... 32 7.1.2 National Health Priority Areas ...... 32 7.2 Objectives ...... 33 7.3 Methods 33 7.3.1 Disease coding 33 MEDICINES MILESTONES A decade of persistence, prevention and promise 3

1 Foreword

To see the enormous value that health and medical research and development add to our health and our economy, you don’t have to look much further than the medicines industry. This comprehensive report Medicines Milestones finds that the Australian medicines industry brought 795 new medicines to market in Australia in the last decade. Health and medical research and innovation brings not only better health to so many Australians, it lifts the quality of the healthcare services and builds our global competitive advantage. Indeed, Australia’s reputation for excellence in research attracts $1 billion a year in R&D investment and sponsors more than 700 clinical trials, involving 18,000 Australians. Reviewing the achievements of the last 10 years has shown that health and medical research has built a vibrant, high-value medicines industry. It generates employment for a highly skilled workforce, helps retain our own talent in Australia, and attract talent from offshore. In all these ways, health and medical research continues to generate increases in Australian’s productivity and in our GDP – not to mention our better health. And while this report identifies the medicines industry’s achievements in the 21st century, health and medical researchers in Australia continue the quest for better understanding of health and disease prevention, develop more effective diagnostics and treatments, and find better ways to deliver healthcare. This vital work deserves the support of governments, investors and the broad community. Innovation is alive and well and living in Australia as celebrated in this report on some of our progress to date.

Dr Christine Bennett Chair, Research Australia MEDICINES MILESTONES 4 A decade of persistence, prevention and promise

2 executive Summary

In the ten years to 2011, some 795 medicines were approved for use in Australia. These medicines are extending life expectancy, improving quality of life, and even preventing some diseases altogether. The Medicines Milestones report, commissioned by The Australian Medicines Industry and carried out by research firm Pretium, found that treatments were approved for a total of 93 different conditions. These ranged from common conditions such as asthma and hypertension, to the very rare, such as Niemann-Pick disease Type C. The report found that treatments for cancer, cardiovascular diseases and vaccines led approvals in Australia over the last decade. Cancer treatments accounted for 115 of total approvals, reflecting the burden of the disease (one in two Australians can expect to be diagnosed with cancer at some point in their lives1). The Therapeutic Goods Administration (TGA) also approved 82 medicines for cardiovascular disease, a leading cause of death in Australia. Prevention of disease also featured prominently with 57 new approvals. Vaccines that became available in the past 10 years include childhood vaccines on the National Immunisation Program such as measles, mumps, rubella, polio, diphtheria and whooping cough. Others included meningococcal and meningococcal meningitis, cervical cancer, influenza, tetanus, smallpox and cholera. Most of the Federal Government’s National Health Priority Areas were strongly represented, with 184 TGA approvals, led by cancer control with 56 new approvals in identified focus areas, arthritis and musculoskeletal conditions (53), cardiovascular health (29) and diabetes (27). However, when it came to depression, there were just six approvals for new medicines within the mental health National Health Priority area over the last decade. Nevertheless, depression treatments are currently being trialled, with over 60 Phase III trials focusing on depression recorded on the global clinical trials register, www.clinicaltrials.gov

2.1 breakthrough medicines make their mark

A significant finding was that one in four medicines approved was a medical breakthrough using a newly discovered molecule, unique to any other currently used to combat disease. It takes the testing of 10,000 new molecules, 15 years and $1.4 billion to get one medical breakthrough from microscope to medicine. These 224 medical breakthroughs represent a $313 billion investment in research and development and have changed the way 52 different diseases are treated.

1 http://www.cancer.org.au/Newsmedia/factsfigures.htm last viewed 15 June 2012 MEDICINES MILESTONES A decade of persistence, prevention and promise 5

2.2 biological medicines and targeted medicines – the way of the future

There currently are more than 3,000 medicines and vaccines in various stages of development globally with the potential to help millions. There are more than 800 medicines in development to treat various forms of cancer, around 300 to treat rare diseases such as Addison’s disease and cystic fibrosis, and over 250 to treat cardiovascular disease and nearly as many to treat diabetes. One of the most important trends over the next decade is likely to be a sharp rise in the number of new biological medicines in development. These are medicines developed using biological process, with living cells or organisms. Biological medicines marshal the body’s own defences to treat disease. Patients can expect to see the increasing emergence of ‘targeted’ therapies for people with specific gene sequences, so treatment can be targeted to the patients whose genetic make-up indicates that they are likely to have a better response. Importantly, Australia is playing a significant role in this quest to develop new medicines and vaccines. The Australian medicines industry invests more than $1 billion a year in R&D and funds more than 700 clinical trials.

The Medicines Milestones report was commissioned by The Australian Medicines Industry to provide an overview of the last ten years of TGA approvals in Australia, noting changes and trends and focusing on national health priority areas. The research was carried out by Pretium using its proprietary Drug Tracker database, which contains information on regulatory approvals, PBAC outcomes and PBS listings. MEDICINES MILESTONES 6 A decade of persistence, prevention and promise

3 decade trends

3.1 number of medicines

During the last 10 years, Pretium’s Drug Tracker database indicates that 795 new medicines have been registered for use in Australia by the Therapeutic Goods Administration (TGA). The number of these new registrations by year ranges from a high of 103 in 2002 to 58 in 2011 (Figure 1). A sharp decline in TGA registrations is apparent in 2011. This may be explained by a change in the way the TGA processes evaluations. It may also be the case that there are some medicine approvals pending. The actual number of medicines registered by the TGA decreased steadily between 2004 and 2007, before a sharp rise of 32 per cent in 2009. Medicines receiving registration were for conditions ranging from common diseases such as asthma and hypertension to orphan diseases such as Niemann-Pick disease Type-C and Fabry disease.

REGULATORY APPROVALS

S 120

AL 103

OV 100 93 90 84 82 79 PR 74 80 68

AP 64 60 58 OF 40 20

NUMB ER 0 2002 2003 2004 2005 2006 2007 2008 2009 2010 2011

YEAR OF REGULATORY APPROVAL

Figure 1 Number of TGA registrations, 2002-2011 (n=795)

Table 1 presents the medicines registered by the TGA by condition and year. The coding of the conditions is described in Section 7 (Background and methods). Where possible medicines were left in their ATC codes, where the codes clearly corresponded to conditions. However, additional coding was required for many of the medicines based on their approved indication. MEDICINES MILESTONES A decade of persistence, prevention and promise 7

Table 1 Medicines approved by TGA by condition and year

Condition* 2002 2003 2004 2005 2006 2007 2008 2009 2010 2011 Total Acne 1 2 1 4 ADHD 1 2 1 4 Addictive disorders 2 1 3 AIDS 1 1 2 Allergies 1 1 1 2 1 1 7 Alzheimer’s disease 1 1 1 3 Aged related macular 1 1 1 2 5 degeneration Anaemia 1 1 2 1 2 7 Anaesthetic 1 2 1 1 2 1 8 Anaplastic astrocytoma 1 1 Anthrax 1 1 1 3 Anti-bacterial 1 2 1 2 2 2 10 Anti-malarials 2 1 3 Anti-mycotic/fungal 1 2 1 2 1 7 Arthritis 2 5 4 4 1 3 3 8 1 3 34 Asthma 3 1 1 2 2 2 1 1 13 Behavioural disorders 1 1 2 Bipolar 2 2 1 2 4 2 13 disorders 2 1 1 2 1 2 4 13 Blood pressure 9 2 4 6 4 2 4 5 4 2 42 disease 1 1 2 Benign prostatic hyperplasia 2 2 4 Cancer 9 10 12 11 15 10 13 14 12 9 115 Cardiovascular 1 1 1 1 2 1 7 Cystic fibrosis 1 2 2 5 Cholesterol 2 5 5 1 1 2 1 1 18 Chronic kidney disease 1 1 2 1 5 COPD 1 1 2 4 1 1 2 Dermatological 1 1 2 1 5 Dysmenorrhea 1 1 Drug overdose 1 1 Dialysis 1 1 Diabetic foot 1 1 Diabetes 1 1 7 3 4 3 4 2 2 27 Endometriosis 1 1 Erectile dysfunction 2 2 1 1 1 3 10 Eczema 1 1 Epilepsy 1 1 Eye disorders 2 1 3 1 1 1 9 Generalised anxiety disorder 1 2 3 Gastrointestinal 3 3 8 5 3 3 7 3 3 2 40

* An explanation for the coding can be found in Section 7 (Background and methods). MEDICINES MILESTONES 8 A decade of persistence, prevention and promise

Condition* 2002 2003 2004 2005 2006 2007 2008 2009 2010 2011 Total Genetic disorders 2 1 2 4 9 Glioma 1 1 Growth disorders 3 1 1 2 6 1 14 Hepatitis B 1 1 2 1 5 Hepatitis C 4 2 2 1 9 Herpes 1 1 HIV 3 3 3 3 2 3 1 18 Hormone replacement therapy 2 2 4 Immunodeficiency 1 2 2 1 1 1 8 Imaging 2 5 1 3 2 1 14 Insomnia 1 1 2 Influenza 1 1 1 1 4 IVF 3 1 1 1 6 Mental illness 1 1 Major depressive disorder 1 2 1 1 1 6 Multiple sclerosis 1 1 1 1 4 Nasal indications 1 1 2 4 Neurological 1 2 3 1 2 2 11 Nutritional supplements 1 2 2 1 6 Oral contraceptive 2 3 1 2 2 1 2 13 Osteoporosis 2 3 2 2 2 3 4 2 20 Otitis media 1 1 2 Overactive bladder 2 1 1 1 5 Panic disorder 1 1 Pain 3 2 1 1 1 1 4 1 14 Parkinson’s disease 1 1 1 2 1 1 7 Premenstrual dysphoric disorder 1 1 2 Premature ejaculation 1 1 Pneumonia 2 2 Pulmonary hypertension 2 1 3 Prevention of blood loss 2 1 4 1 3 11 Prevention of clots 3 2 2 1 1 4 3 3 3 22 3 2 1 1 1 1 1 10 PTSD 1 1 Respiratory distress syndrome 1 1 2 Respiratory 1 1 2 Sedation 1 1 Schizophrenia 2 1 3 2 1 1 10 Schizoaffective 1 1 Seizures 1 1 1 2 1 6 Sleep disorder 1 1 2 Social phobia 1 1

* An explanation for the coding can be found in Section 7 (Background and methods). MEDICINES MILESTONES A decade of persistence, prevention and promise 9

Condition* 2002 2003 2004 2005 2006 2007 2008 2009 2010 2011 Total Stem cell transplantation 1 1 Social anxiety 1 1 1 3 Surgical bleeding 1 2 2 5 Testosterone deficiency 1 1 2 Thyroid dysfunction 1 1 2 Thrush 1 1 Transplant 1 1 1 1 4 8 Urological 2 1 3 Vaccine 11 2 2 7 8 2 5 7 8 5 57 103 84 93 82 74 64 68 90 79 58 795

* An explanation for the coding can be found in Section 7 (Background and methods).

To display the information presented in Table 1 graphically, the data is presented based on the number of medicines approved over the time period. Cancer conditions had the highest number of medicines approved (n=116) over the 10-year period. Prevention of disease through the development of vaccines was also a high priority with 57 medicines approved. Medicines associated with cardiovascular disease, (blood pressure n=42; cholesterol n=18; prevention of blood clots n=22) were also shown to be a focus with a total of 82 medicines approved in this category.

140 120 115 100 80 57 60 42 40 40 34 27 18 18 20 22 20 10 13 13 13 10 14 14 11 13 14 11 10 10 0 e ol HIV osis Oral enia Pain ders ders owth ectile essur Bipolar Cancer Er Gr Arthritis ological accines Asthma Imaging of clots eventing evention Diabetes V Psoriasis disor Pr Pr blood loss Cholester ointestional dysfunction Antibacterial Neur Osteopor Schizophr contraception Blood pr Blood disor Gastr

Figure 2 Conditions where 10 or more medicines were approved for use by the TGA between 2002 and 2011 (n=549) MEDICINES MILESTONES 10 A decade of persistence, prevention and promise

Figure 3 presents the conditions where between five and nine medicines were approved by the TGA for the 2002-2011 time period. Medicines for eye disorders (excluding age-related macular degeneration), hepatitis C and genetic disorders all had nine medicines approved. The category of genetic disorders includes disorders such as Niemann-Pick Type C disease, Pompe’s disease and Fabry’s disease, all of which are rare diseases with small numbers of patients in Australia.

10 99 9 9 8 8 8 8 7 7 7 7 7 7 666 6 6 5 555 5 5 5 5 4 3 2 1 0 es IVF der Eye osis ders essive disease Allergies Seizur Anaemia disor ransplant s Disease T Anti-fungal Hepatitis B Hepatitis C diovascula r onic kidney Anaesthetic Dermatology degeneration Cystic fibr elated macular Car Chr Major depr Surgical bleeding Genetic disor Nutritional support Immunodeficiency Overactive bladder Parkinson’ A ge-r

Figure 3 Conditions where 5-9 medicines were approved for use by the TGA between 2002 and 2011 (n=145)

Figure 4 presents the additional conditions for which medicines were approved by the TGA. Some recoding was completed to collapse categories to make the information more manageable (Table 2). Recoding was completed on all conditions where there were fewer than four medicines approved over the time period.

4.5 4 4 4 44 4444 4 4 3.5 3 33 3 3 3 2.5 2 1.5 1 0.5 0 oid osis Acne Nasal ostate ADHD COPD Thyr ological Multiple Anthrax therapy Influenza scler Hormone Ur Pulmonary conditions indications Respiratory eplacement hyperplasia Antimalarials r hypertension Gynaecological Benign pr

Figure 4 Other conditions where medicines were approved by the TGA between 2002 and 2011 (recoded n=55) MEDICINES MILESTONES A decade of persistence, prevention and promise 11

When the conditions were recoded, mental health became a major focus with 26 medicines included in the analysis (Figure 5). This was made up of medicines specifically indicated for social anxiety disorders (n=3), addictive disorders (n=3) and generalised anxiety disorder (n=3). 3.5 3 33 3 3 2.5 2 22 2 2 1.5 1 1 11 11 1 0.5 0 der der der der der der ders dose fective disor disor Addictive Dementia Alzheimer disor Behavioral dysphoric emenstrual Generalised ess disor Pr Mental illness Social phobia Social anxiety Panic disor Sleep disor Drug over Post-traumatic Schizoaf str anxiety disor

Figure 5 Mental health conditions where medicines were approved by the TGA between 2003 and 2011 (recoded n=26)

Table 2 Recoding of medicines with smaller number for graphical representation

Condition* Recoding Condition* Recoding Acne Dermatological HRT HRT ADHD ADHD Insomnia Other Addictive disorders Mental health Influenza Influenza AIDS Antiviral Mental Illness Mental health Alzheimer’s disease Mental health Multiple sclerosis Multiple sclerosis Anaplastic astrocytoma Other Nasal indications Nasal indications Anthrax Anthrax Otitis media Other Anti-malarials Anti-malarials Panic disorder Mental health Behavioural disorders Mental health PMDD Mental health Blood clotting Other Premature ejaculation Urological Bone disease Other Pneumonia Antiviral Benign prostate hyperplasia Benign prostate hyperplasia Pulmonary hypertension Pulmonary hypertension COPD COPD PTSD Mental health Dementia Mental health Respiratory distress syndrome Respiratory Dysmenorrhea Gynaecological Respiratory Respiratory Drug overdose Mental health Sedation Other Dialysis Other Schizoaffective disorder Mental health Diabetic foot Other Sleep disorder Mental health Endometriosis Gynaecological Social phobia Mental health Eczema Dermatological Stem cell transplantation Other Epilepsy Other Social anxiety Mental health Generalised anxiety disorder Mental health Surgical Bleeding Surgical bleeding Glioma Other Testosterone deficiency Other Haemorrhoids Other Thyroid conditions Thyroid conditions Hepatitis B Hepatitis B Thrush Gynaecological Herpes Antiviral Urological Urological

* An explanation for the coding can be found in Section 7 (Background and methods). MEDICINES MILESTONES 12 A decade of persistence, prevention and promise

Within this group, a small number of conditions could not be recoded and have been included below as part of an ‘other’ category. These conditions had one or two medicines registered over the 10-year period (Figure 6).

2.5 2 2 2 2

1.5 1 1111 11 1

0.5

0 one Glioma Dialysis Epilepsy ocytoma Sedation infection Stem cell Anaplastic stoster deficiency Otitis Media Diabetic foot Te astr Bone disease tranplantation

Figure 6 Conditions with medicines registered between 2002-2011 which were recoded as ‘other’ (recoded n=13)

3.2 number of new molecules

Between 2002 and 2011, there were 224 new molecules registered by the TGA (Figure 7). A new molecule is defined as a medicine that is unique compared to those already registered in Australia. As such, this list does not include submissions put to the TGA for generic medicines or when new indications are requested. The frequency of these registrations per year ranged from 15 in 2003 to 30 in 2004 and 2006. There are many hurdles to be overcome during the journey of medicines discovery and development. The complexity of this process means that while a significant number of new medicines may become available in some years, in other years there may be fewer.

TGA NEW MOLECULES (INCL VACCINATIONS)

S 35 30 30 AL 30 27

OV 25 25 22 PR 20 20 19 19

AP 17 15 15 OF

10 5

NUMB ER 0 2002 2003 2004 2005 2006 2007 2008 2009 2010 2011

YEAR OF TGA APPROVAL

Figure 7 Number of TGA new molecules registered, 2002-2011(n=224)

3.3 new medicines by areas and conditions

A total of 224 new molecules were registered by the TGA between 2002 and 2011 (Table 3). Cancer treatments (n=36), vaccines (n=23) and diabetes (n=10) had the most new molecules over the period of interest. MEDICINES MILESTONES A decade of persistence, prevention and promise 13

Table 3 New molecules 2002-2011

Condition* 2002 2003 2004 2005 2006 2007 2008 2009 2010 2011 Total Addictive disorder 1 1 Allergies 1 1 2 1 5 Aged related macular 1 1 2 degeneration Anaemia 1 2 3 Anaesthetic 1 1 Anti-bacterial 1 1 2 Anti-fungal 1 1 1 3 Arthritis 2 1 1 5 9 Asthma 1 1 ADHD 1 1 Bleeding – surgery 1 2 3 Blood disorders 1 1 2 2 1 7 Blood pressure 1 1 2 1 3 1 9 Benign prostatic hyperplasia 1 1 Cancer 3 2 4 4 6 3 1 4 5 4 36 Cardiovascular (ex , BP) 1 1 1 3 Cystic fibrosis 1 1 Cholesterol 1 1 1 3 Chronic kidney disease 1 2 1 4 COPD 1 1 Diabetes 2 3 2 1 2 10 Dysmenorrhea 1 1 Endometriosis 1 1 Erectile dysfunction 1 1 2 Eye disorders 1 1 2 Gastrointestinal 1 1 1 1 1 1 6 Genetic disorders 1 1 3 5 Growth disorders 1 1 2 Hepatitis B 1 2 3 HIV 1 2 1 3 1 8 Imaging 1 2 1 2 2 8 Immunodeficiency 1 1 2 4 IVF 1 1 1 3 Major depressive disorder 1 1 1 3 Multiple sclerosis 1 1 2 Neurological 1 1 Oral contraceptives 1 1 Osteoporosis 1 1 2 4 Overactive bladder 2 1 3 Pain 1 1 1 3 Parkinson’s disease 1 1 1 3 Premature ejaculation 1 1 Prevention of blood loss 2 1 2 5

* An explanation for the coding can be found in Section 7 (Background and methods). MEDICINES MILESTONES 14 A decade of persistence, prevention and promise

Condition* 2002 2003 2004 2005 2006 2007 2008 2009 2010 2011 Total Prevention of blood clots 1 2 2 2 7 Psoriasis 2 1 3 Pulmonary hypertension 2 2 Respiratory distress syndrome 1 1 1 3 Schizophrenia 1 1 2 Seizures 1 1 1 3 Testosterone deficiency 1 1 Transplant 1 1 1 3 Vaccine 1 2 3 1 4 5 4 3 23 Total 224

* An explanation for the coding can be found in Section 7 (Background and methods). To present the data from Table 3 graphically, additional recoding was completed (Table 4). All conditions that had at least five new molecules approved over the period were left as separate codes and the remaining molecules were recoded, where possible, to attempt to consolidate the number of conditions.

Table 4 Recoding of conditions with new molecules approved from 2002-2011 for graphical presentation

Condition* Recoding Condition* Recoding Addictive disorder Mental health Genetic disorders Genetic disorders Allergies Allergies Growth disorders Growth disorders Age related macular Eye disorders Hepatitis B Antiviral degeneration Anaemia Blood disorders HIV HIV Anaesthetic Surgery Imaging Imaging Anti-bacterial Anti-bacterial/anti-fungal Immunodeficiency Immunodeficiency Anti-fungal Anti-bacterial/anti-fungal IVF Gynaecological Arthritis Arthritis Major depressive disorder Mental health Asthma Respiratory Multiple sclerosis Neurological ADHD Mental health Neurological Neurological Bleeding – surgery Surgery Oral contraceptives Gynaecological Blood disorders Blood disorders Osteoporosis Osteoporosis Blood pressure Blood pressure Overactive bladder Urology Benign prostatic hyperplasia Urology Pain Neurological Cancer Cancer Parkinson’s disease Neurological Cardiovascular Cardiovascular Premature ejaculation Urology Cystic fibrosis Respiratory Prevention of blood loss Prevention of blood loss Cholesterol Cardiovascular Prevention of blood clots Prevention of blood clots Chronic kidney disease Urology Psoriasis Arthritis COPD Respiratory Pulmonary hypertension Cardiovascular Diabetes Diabetes Respiratory distress syndrome Respiratory Dysmenorrhea Gynaecological Schizophrenia Mental health Endometriosis Gynaecological Seizures Neurological Erectile dysfunction Urology Testosterone deficiency Hormone treatment Eye disorders Eye disorder Transplant Surgery Gastrointestinal Gastrointestinal Vaccine Vaccine

* An explanation for the coding can be found in Section 7 (Background and methods). MEDICINES MILESTONES A decade of persistence, prevention and promise 15

Figure 8 presents the conditions for which new molecules were approved by the TGA between 2002 and 2011. Codes were based on Table 4. Any codes that have five or fewer new molecules were combined into an ‘other’ category (Figure 9).

40 36 35 30 25 23 20 15 12 12 10 9 10 11 10 8 66887 7 6 7 5 0 HIV ders ology accine essu re Cancer Arthritis ological Ur Surgery V Imagin g evention Diabetes ointestinal Pr diovascular Respiratory Neur Mental health of blood clot s Car Blood pr Gynaecological Gastr Blood disor

Figure 8 Number of new molecules (>5) for each condition between 2002 and 2011 (n=186)

6 5 5 55 5 44 4 4 3 3 2 2 1 1 0 osis ders ders ders owth Antiviral Gr Allergies Genetic disor disor Hormone blood loss anti-fungal evention of Osteopor Eye disor Pr Anti-bacterial/ Immunodeficiency

Figure 9 Number of new molecules (5 or fewer) for conditions from 2002 to 2011 (n=38) MEDICINES MILESTONES 16 A decade of persistence, prevention and promise

3.4 number of prevention medications (including vaccines)

Between 2002 and 2011, 134 medicines that were indicated for disease prevention or prophylaxis were registered by the TGA (Figure 10). This includes all vaccinations and immunisations. While there had been a steady decline in the number of regulatory registrations for preventative medicines between 2002 and 2007, there was a significant increase in 2009. This spike coincides with the Swine Flu pandemic of 2009.

PREVE NTION, PROPHYLAXIS, VACCINES — BY YEAR

20 19 18

S 18

AL 16 14 14 14

OV 14 12 12

PR 12 11 10 10

AP 10

8 OF 6 4 2

NUMB ER 0 2002 2003 2004 2005 2006 2007 2008 2009 2010 2011

YEAR OF TGA APPROVAL

Figure 10 Number of TGA registrations for preventative/prophylactic medicines, 2002 to 2011 (n= 134)

I have been a parent advocate for the use of vaccinations as a preventative strategy against vaccine preventable diseases for over 20 years. Bacterial meningitis left my six month old daughter Ashleigh with significant disabilities in 1989. My mission since then has been to promote and lobby for the inclusion of meningitis vaccines onto the National Immunisation Program. Over these years we have seen a dramatic reduction in bacterial meningitis in Australia, due directly to the inclusion of three very effective vaccines on the NIP. Every child has the right to protection and we have witnessed the positive effect vaccines are having as an extremely valuable line of defence.

Bruce Langoulant Chairman, The Meningitis Centre President, Confederation of Meningitis Organisations Inc. MEDICINES MILESTONES A decade of persistence, prevention and promise 17

4 national Health Priority Areas

4.1 number of TGA approvals

Between 2002 and 2011, there were 184 medicines approved by the TGA for conditions within the Federal Government’s National Health Priority Area (NHPA) framework focus areas recorded in Pretium’s Drug Tracker database (either from single or group applications). The National Health Priority Area with the most TGA registrations was cancer control2 with 56 registrations, followed by arthritis and musculoskeletal conditions with 53 registrations and cardiovascular health with 29 registrations then diabetes treatments (excluding blood glucose indicators) with 27 registrations (Table 5). The distribution of new medicines registered by the TGA across the National Health Priorities can be seen in Figure 11.

15% 29% 3% Arthritis and musculoskeletal conditions Asthma

16% Cancer control Cardiovascular health 7% Depression (mental health) Diabetes mellitus

30%

Figure 11 Number of TGA medicine approvals, stratified by NHPA, 2002-2011 (n = 184)

Table 5 Number of TGA medicine approvals, by National Health Priority Areas

National Health 2002 2003 2004 2005 2006 2007 2008 2009 2010 2011 Total Priority Area Depression (mental 0 1 2 0 0 0 1 1 1 0 6 health NHPA)

Asthma 3 1 1 2 2 2 1 0 0 1 13

Diabetes mellitus 1 1 7 0 3 4 3 4 2 2 27

Cardiovascular health 4 3 2 1 1 1 4 4 5 4 29

Arthritis and musculoskeletal 4 8 4 6 1 5 4 11 5 5 53 conditions

Cancer control 4 7 7 8 6 6 6 3 7 2 56

Obesity 0 0 0 0 0 0 0 0 0 0 0

Total 16 21 23 17 13 18 19 23 20 14 184

2 This includes only cancer medicines – does not include all medicines coded in ATC code L MEDICINES MILESTONES 18 A decade of persistence, prevention and promise

4.2 Cancer focus areas

4.2.1 Number of approvals each year over 10 years Cancer control was the most popular NHPA in terms of the number of TGA medicine approvals between 2002 and 2011. As shown in Figure 12, there has been a general decline in the number of TGA medicine approvals for the cancer focus areas since 2004, with the exception of 2010. The focus areas included in the cancer control National Health Priority Area were lung cancer, melanoma, non-melanoma skin cancers, colorectal cancer, prostate cancer, non-Hodgkin , cervical cancer and breast cancer.

9 S 8 8 AL 77 7 7 OV 666 6 PR 5 AP

4 4

OF 3 3 2 2 1

NUMB ER 0 2002 2003 2004 2005 2006 2007 2008 2009 2010 2011

YEAR OF TGA APPROVALS

Figure 12 TGA medicine approvals, NHPA: Cancer control (n=56)

As a committed organisation to support people affected by cancer, developments over the past five years through a transparent engagement process has seen Cancer Voices Australia at the forefront of issues surrounding Government policy and access to life saving cancer . The cost of healthcare in this country is under review. Access to new and important oncology drugs which play a vital role in the treatment of people affected by cancer will continue to be a major factor in our advocacy agenda. The importance of continued collaboration, transparency and engagement of Medicines Australia and consumer groups will have a positive impact on people in this country affected by cancer.

John Stubbs Chief Executive, Cancer Voices Australia

4.2.2 Split between focus areas The focus areas of lung, colorectal, prostate, cervical and breast cancers, melanoma and non-Hodgkin lymphoma resulted in 56 TGA medicine approvals. Figure 13 provides the trend for the number of medicines approved stratified by NHPA cancer control focus areas. MEDICINES MILESTONES A decade of persistence, prevention and promise 19

4% 5% Breast cancer 34% Colorectal cancer 18% Melanoma Multiple cancer indications Lung cancer Prostate cancer 16% Non-Hodgkin lymphoma 14% Non-melanoma skin cancer 7% 2% Figure 13 TGA medicine approvals, NHPA: Cancer control (focus conditions), n=56

4.2.3 Focus vs non-focus areas The number of medicines approved for focus cancers compared with those for non-focus cancers is shown in Figure 14. When comparing cancers categorised as focus to other cancers for which approvals were given, the number of medicines approved for the focus cancers was slightly less than those from the non-focus cancers (e.g. laryngeal cancer, renal cell carcinoma, ovarian cancer).

49% Other cancers 51% "Focus cancers"

Figure 14 TGA medicine approvals, NHPA: Cancer control (comparison of focus and non-focus areas), n=115

For cancers as a whole, five-year survival rates have improved from 41% for males diagnosed in 1982-86 to 58% for those diagnosed in 1998-2004: improvements in cancer treatments have contributed to these gains. Australia’s Health 2010, Australian Institute of Health and Welfare, (p.138)

The risk of being diagnosed with cancer before the age of 75 years is 1 in 3 for males and 1 in 4 for females. In 2007, 39,884 Australians died from cancer – the equivalent of 109 people every day. The most commonly diagnosed cancers in Australia are (in order): • Prostate • Bowel • Breast • Melanoma of skin • Lung. MEDICINES MILESTONES 20 A decade of persistence, prevention and promise

The most common causes of cancer death are (in order): • Lung • Bowel • Prostate • Breast • Lymphoid. Age standardised mortality rate for all cancers combined fell by 16 per cent from 209 deaths per 100,000 people in 1982 to 176 deaths per 100,000 people in 2007. Australian Cancer Incidence and Mortality (ACIM) books, Australian Institute of Health and Welfare

4.3 Arthritis and musculoskeletal conditions

4.3.1 Number of medicines approved each year over 10 years The category of arthritis and musculoskeletal conditions focuses on rheumatoid arthritis, osteoarthritis and osteoporosis. As Figure 15 indicates, there were at least four medicines for rheumatoid arthritis, osteoarthritis and osteoporosis approved by the TGA each year, with the exception of 2006, when there was only one medicine approved.

12 11 S 10 AL 8 OV 8 PR 6

AP 6

5 55 4 4 4 OF

4

2 1

NUMB ER 0 2002 2003 2004 2005 2006 2007 2008 2009 2010 2011

YEAR OF TGA APPROVAL

Figure 15 TGA medicines approved, NHPA: Arthritis and musculoskeletal conditions (n=53)

In the past decade, we have seen extraordinary improvements in arthritis treatment in Australia. Not only do we know more about early and proper diagnosis, but the development and access to new and often life-changing medicines have created unprecedented opportunities for people with arthritis. It is our hope that the enormous progress that has been made in the last ten years will encourage further research to benefit people living with arthritis and musculoskeletal conditions.

Ainslie Cahill CEO Arthritis Australia MEDICINES MILESTONES A decade of persistence, prevention and promise 21

4.3.2 Split between focus areas The split of medicine approvals between the focus areas in the arthritis and musculoskeletal National Health Priority Area is shown in Figure 16. The results indicate that the osteoporosis and rheumatoid arthritis indications are the most prevalent indications, with more than 91 per cent of the medicines split between these two areas. Medicines approved for osteoarthritis were also approved for rheumatoid arthritis but the reverse was not true.

9%

38%

Osteoporosis Rhematoid arthritis

Both osteoarthritis and rheumatoid arthritis

53%

Figure 16 TGA registrations, NHPA: Arthritis and musculoskeletal conditions, split of focus areas (n=53)

More than 6.3 million Australians (31 per cent) have arthritis or some other musculoskeletal condition. Australia’s Health 2010, Australian Institute of Health and Welfare, (p. 187)

4.4 Cardiovascular health

4.4.1 Number of medicines approved each year over 10 years The cardiovascular health National Health Priority Area focuses on both the maintenance of cardiovascular health and the prevention of cardiovascular disease. It therefore consists not only of coronary heart disease and heart failure but also of preventative medicines for stroke and peripheral vascular disease. There were 29 medicines approved by the TGA for conditions in the cardiovascular health National Health Priority Area between 2002 and 2011. Figure 17 shows the 10-year trend in the number of medicine approvals, with a period of decreasing registrations, until a spike in 2008 which has continued through to 2011.

S 6

AL 5 5 OV 4 44 4

PR 4

AP 3

3

OF 2 2 111 1

NUMB ER 0 2002 2003 2004 2005 2006 2007 2008 2009 2010 2011

YEAR OF TGA APPROVAL

Figure 17 TGA medicine approvals, NHPA: Cardiovascular health (n=29) MEDICINES MILESTONES 22 A decade of persistence, prevention and promise

Medicines have revolutionised treatment of ischaemic stroke in Australia. If given within 4.5 hours after the onset of a stroke, the right medicine can increase the number of stroke patients with good outcomes and limit their disability. It can mean the difference between life and death; between returning home and living with a dependent disability. Where it is administered in time and in appropriate cases, it is remarkably effective. Medicine has allowed many stroke survivors to recover limb function and a degree of independence they may otherwise not have had. Although its effects are not permanent, treatment can be highly effective in managing spasticity in hands, arms and legs.

Dr Erin Lalor CEO, National Stroke Foundation

4.4.2 Split between focus areas The different indications for medicines for cardiovascular health indicate that medicines to help prevent blood clotting or stroke prevention accounted for the majority of medicine approvals (Figure 18). Heart failure and heart disease have had a similar number of approvals but the combination of these two indications accounts for less than a quarter of all medicines approved in the cardiovascular health focus area.

14%

10% Stroke Heart failure Heart disease

76%

Figure 18 TGA medicine approvals, NHPA: Cardiovascular health, split of focus areas (n =29)

High blood pressure and high cholesterol, for example, are often controlled by prescription , typically in conjunction with diet and exercise. Australia’s Health 2010, Australian Institute of Health and Welfare, (p. 112)

With appropriate medication patients with cardiovascular disease, or those at risk of the disease, can improve their quality of life and increase their life expectancy. The use of cardiovascular medicines and the adoption of lifestyle measures, such as regular exercise and a healthy diet, can reduce important cardiovascular disease risk factors such as high blood cholesterol and high blood pressure. Cardiovascular Disease: Australian Facts 2011 – Australian Institute of Health and Wefare MEDICINES MILESTONES A decade of persistence, prevention and promise 23

4.5 diabetes focus areas

4.5.1 Number of medicines approved each year over 10 years Between 2002 and 2011, 27 medicines for diabetes were approved. As can be seen in Figure 19, this number was greatly boosted by the seven new medicines registered in 2004. Apart from this, the number of medicines approved has remained relatively steady over the past 10 years.

DIABETES

8 7 6 4 4 4 3 3 22 2 11 0 0 2002 2003 2004 2005 2006 2007 2008 2009 2010 2011

Figure 19 TGA medicine approvals, NHPA: Diabetes (n=27)

The deadly face of diabetes changed forever in 1921 when two Canadian researchers, Dr Frederick Banting and his assistant, medical student Charles Best discovered insulin, winning them a Nobel Prize in 1923. And to this day injectable insulin is a life-saving treatment for millions of people living with both Type 1 and late stage Type 2 diabetes. Over the past 10 years, the number and classes of diabetes medicines continues to increase and improve, and many are subsidised on the Pharmaceutical Benefits Scheme. The decade has also seen the ratio of those diagnosed with Type 2 diabetes relative to those undiagnosed drop from 1:1 down to 5:4, leaving an estimated 680,000 Australians living with undiagnosed diabetes. Today there is no reason why Australians cannot live a long and otherwise healthy life with a diagnosis of diabetes, if they choose to self-manage to the best of their ability. For most Australians there is good support available via GPs, diabetes educators, Diabetes Australia, the National Diabetes Services Scheme, and subsidised medications together with a growing societal awareness of diabetes and pre-diabetes (now affecting 3.7 million Australians) and a slow reduction in stigma and discrimination.

Lewis Kaplan CEO, Diabetes Australia MEDICINES MILESTONES 24 A decade of persistence, prevention and promise

Four per cent of Australians have diabetes – that’s around 898,000 people. The proportion of Australians with diabetes has risen from 1.5 per cent in 1989 to 4 per cent in 2012. The good news is that the death rate from diabetes is not rising. 222,544 people began using insulin to treat their diabetes between 2000 and 2009. Australian Institute of Health and Welfare: http://www.aihw.gov.au/diabetes/ (Accessed 1 July 2012)

An estimated 275 Australians develop Type 2 diabetes every day. By 2023, Type 2 diabetes is projected to become the leading specific cause of disease burden for men and the second leading cause for women. Type 2 Diabetes: The 21st Century Pandemic, Diabetes Australia

4.6 Depression*

4.6.1 Number of medicines approved each year over 10 years There were few medicines approved by the TGA for mental health focusing on depression between 2002 and 2011 (n=6). Figure 20 shows that in only five of the last 10 years were medicines for depression approved by the TGA. As can be seen from the below graph, the medicine approvals were tightly grouped in 2003-04 and 2008-10.

DEPRESSION 3 2 2 2 1 111 1 1 0 000 0 0 2002 2003 2004 2005 2006 2007 2008 2009 2010 2011

Figure 20 TGA medicine approvals, NHPA: Mental health (n=6)

Ten years ago people really struggled to live with mental illness. Now with comprehensive treatment packages which include medications, psychological interventions, community services, family support programs as well as campaigns to reduce stigma and discrimination, many more people are able to lead healthy and productive lives.

Barbara Hocking former executive Director, SANE Australia

* This is the only mental health National Health Priority Area. MEDICINES MILESTONES A decade of persistence, prevention and promise 25

4.6.2 Focus vs non-focus areas Figure 21 shows that the majority of new medicines approved by the TGA in the mental health area over the past decade are not the National Health Priority focus area (depression).

10%

"Focus" Other mental health conditions

90%

Figure 21 TGA medicine approvals, NHPA: Mental health, split of focus areas (n=59)

Mental health is one of the leading causes of non-fatal burden of disease and injury in Australia. Australian Institute of Health and Welfare, http://www.aihw.gov.au/mental-health-priority-area/ Accessed 1 July 2012

Depression was the most frequently managed mental health-related problem in 2008-09, accounting for 34.3 per cent of all mental health-related problems managed and 2.8 per cent of all health problems managed. Australian Institute of Health and Welfare, http://www.aihw.gov.au/mental-health-priority-area/ Accessed 1 July 2012

Suicide is one of the main causes of premature death among people with a mental illness. Sane Research Bulletin 11, http://www.sane.org/images/stories/information/research/rb11.pdf Accessed 1 July 2012

One in five Australians experience some form of mental illness every year. Sane Research Bulletin 14, http://www.sane.org/images/stories/information/research/1108_info_rb14work.pdf Accessed 1 July 2012

4.7 Asthma

4.7.1 Number of medicine approvals each year over 10 years There were only 13 TGA medicine approvals for asthma medicines between the years of 2002 and 2011. As can be seen in Figure 22, there was no real trend in the number of medicines approved. However, what was apparent was the reduced number of approvals from 2008 to present. MEDICINES MILESTONES 26 A decade of persistence, prevention and promise

3.5 3 S 3 AL

OV 2.5 222 PR 2 AP 1.5 OF

11 1 1 1 0.5 00 NUMB ER 0 2002 2003 2004 2005 2006 2007 2008 2009 2010 2011

Figure 22 TGA medicine approvals, NHPA: Asthma (n=13) YEAR OF TGA APPROVAL

Between 1997 and 2009, the mortality rate due to asthma decreased by 45 per cent. 1 in 10 Australians suffer from asthma. The rate of general practice encounters for asthma decreased among adults (by 33 per cent) and children (by 27 per cent) between 2000–01 and 2009–10. Drug therapy is the mainstay of asthma management. Asthma in Australia 2011, Australian Institute of Health and Welfare MEDICINES MILESTONES A decade of persistence, prevention and promise 27

5 Vaccines

There has been no clear trend in the number of vaccines approved by the TGA in Australia over the last decade. There are a number of peaks and troughs (Figure 23). Vaccines that became available in the past 10 years include vaccines on the National Immunisation Program such as measles, mumps, rubella, polio, diphtheria and pertussis. Other vaccinations include meningococcal and meningococcal meningitis, influenza, HPV, tetanus, smallpox and cholera.

NUMBER OF VACCINES A PPROVED BY THE TGA

12 11 S

AL 10

OV 8 8 8

PR 7 7 AP 6 5 5 OF

4 2 2 2 2

NUMB ER 0 2002 2003 2004 2005 2006 2007 2008 2009 2010 2011

YEAR OF TGA APPROVAL

Figure 23 Number of vaccines registered by the TGA, 2002-2011 (n=57)

Immunisation has had a dramatic influence on rates of illness and death from a wide variety of . Australia’s Health 2010, Australian Institute of Health and Welfare

The number of reported cases of meningococcal disease in 2003, the year before routine immunisation began, was 490. By 2009 that number was down to 259. Australia’s Health 2010, Australian Institute of Health and Welfare MEDICINES MILESTONES 28 A decade of persistence, prevention and promise

6 tHe way forward

The medicines industry has brought an extraordinary number and range of treatments to market over the past decade. But what will be the focus for pharmaceutical researchers and manufacturers over the next decade, and what will be the benefit for patients? In short, the immediate future is promising. The industry remains as committed to developing new cures and treatments to fight disease as it was a decade ago as companies continue to discover and develop new medicines and respond to changing clinical need. Today there are more than 3,000 medicines and vaccines in development by the global medicines industry to help people live longer and more productive lives. Among these are hundreds of medicines that meet Australia’s current national health priorities. For example, there are more than 800 medicines in development to treat various forms of cancer, around 300 to treat rare diseases such as Addison’s disease and cystic fibrosis, over 250 to treat cardiovascular disease and nearly as many to treat diabetes. Australia is playing its part in this global effort not only by hosting clinical trials for many of these new medicines but also by making significant contributions to an understanding of human disease through basic research. There are also more than 900 biological medicines and vaccines in development, targeting more than 100 diseases. One of the most important trends over the next decade is likely to be a steady rise in the number of new biological medicines in development. These are medicines developed using biological process, with living cells or organisms. Biological medicines marshal the body’s own defences to treat disease. With the mapping of the human genome and the development of genetic testing, companies are increasingly developing “targeted” therapies for people with specific gene sequences. So treatment can be targeted to the patients whose genetic make-up indicates that they are likely to have a better response. The burden of disease is likely to look very different in 2022 from what it is today. An ageing population will drive an increase in degenerative diseases such as osteoporosis and Alzheimer’s disease. It is estimated that the number of people with dementia in Australia will increase from 280,000 today to more than 1 million in 2050. Other diseases likely to become more prominent in Australia are obesity-related conditions such as Type 2 diabetes, high cholesterol and heart disease. The medicines industry is currently developing newer and improved treatments for each of these diseases. There is also likely to be an increased focus on preventative medicines. In addition, there are 145 vaccines in the pipeline, including a number of HIV/AIDS vaccines that may prevent the spread of the virus. Australia is playing an important role in this quest to develop new medicines and vaccines. The Australian medicines industry currently invests more than $1 billion a year in R&D and funds more than 700 clinical trials – 10 times more than the Government’s investment. This research is essential to ensure we continue to develop new medicines and vaccines into the future, and capitalise on the medical advances that have already been made. The industry is hard at work building on the progress of the last decade. The future is extremely promising. MEDICINES MILESTONES A decade of persistence, prevention and promise 29

Alzheimer’s and other - 98 Cancer - 878 Rare diseases - 303 - 119 Mental and Behavioral disorders - 252 Lung Cancer - 120 Parkinson’s Disease - 25 Colorectal Cancer - 82 Depression - 71 Breast Cancer - 125 Vaccines - 145 Skin Cancer - 86 Cardiovascular disorders - 299 /Antibacterials - 88 Diabetes Mellitus - 193 Antivirals - 96 HIV/AIDS - 81 Psoriasis - 41 Arthritis - 67 Respiratory disorders - 334

Figure 24 Medicines in development worldwide

Source: Medicines Australia Facts Book Updated, 2012

Innovative treatments are improving the quality of life of patients and enabling them to live longer, work, raise a family and pursue their goals – things they wouldn’t have been able to do as easily just a few decades ago. Collaboration is increasingly important, and I envision that there will be more meaningful dialogues and partnerships among stakeholders – governments, industry, patients, physicians to ensure patients have access to the best care possible.

Kirsten O’Doherty General Manager, Abbott Australasia

Personalised medicines are set to become a way of life for future Australian generations. Personalised or targeted medicines refer to the process of mapping out a person’s genetic predisposition to particular treatments; it allows accurate predictions to be made regarding the likely success of a particular medication for each individual patient. The concept of personalised medicines has become a reality in recent years, particularly in the area of cancer. These advances mean patients will be given the right medicine to suit their particular genetic makeup. This will result in better outcomes for patients, as well as cost savings in alleviating wastage of inappropriate medicines.

Mark Fladrich Managing Director, AstraZeneca MEDICINES MILESTONES 30 A decade of persistence, prevention and promise

Illnesses accelerated by poor lifestyle choices such as diabetes, COPD, heart disease and some cancers as well as those caused by resistant bacteria and new viral strains are going to be some of the biggest health issues faced by Australians in the next 20 years. Future generations have much to look forward to with not only new medicines but more targeted treatments specific to their genetic make-up. More medicines that stop or prevent reoccurrence of illnesses are all on the agenda and side effect profiles are getting better and better. Melanoma and other forms of cancer will see some of the greatest advancements in both treatments and preventions. I think there will also be better solutions for genetic based disorders and many of the hard to treat viral illnesses.

Geoff McDonald General Manager, GlaxoSmithKline Australia

Our industry has enormous potential to improve the lives of all Australians. Through our lifetime, we’ve seen advances in the treatment of a vast range of diseases, extending lives and keeping people out of hospital. Vaccines have also played an increasing role in preventing disease. There is still much to be done. As work in dementia, pain and inflammation, cancer and cardiovascular disease and diabetes continues, so must we accept the challenge of addressing nearly 7,000 rare diseases – fewer than 5% of these have approved treatments. In Australia we must continue to harness our advantages in education, research and our global reputation for quality in joining the global community addressing these challenges. The outcomes make an enormous positive difference to the everyday lives of Australians.

John Latham Managing Director, Pfizer Australia

Significant areas with the potential for major breakthroughs over the next 20 years include: oncology, diabetes, neuroscience, auto-immune and cardiovascular disease. The unmet medical need for patients living with these diseases is immense. For example, Australia has an ageing population, yet there are limited treatment options for age-related conditions such as Alzheimer’s and dementia, which are incredibly debilitating – both for patients and for their families. To be able to provide hope and relief to patients in these disease areas would be a significant milestone, not only for the industry but for society as a whole. The need for breakthrough medicines to meet the needs of patients has never been greater. As an industry, we must continue to work closely with key Government decision makers to provide access to important medicines for patients.

Chris Miskel Managing Director, Eli Lilly Australia MEDICINES MILESTONES A decade of persistence, prevention and promise 31

The medicines industry has discovered and developed truly innovative medicines which have raised the standard of care in many disease areas leading to improved quality of life and patient outcomes. I believe that the latest innovations in adult vaccinations have led and will continue to lead to a reduction in the incidence of infection and disease. This, coupled with increased access to these innovative medicines, has resulted in treatment paradigms and morbidity and mortality being significantly improved in areas like diabetes, hyperlipidaemia, HIV, hepatitis and infertility to name a few.

Dr. Gary Jankelowitz Medical Director, MSD

Australia has a very high standard of healthcare and the challenge over the next 20 years will be to maintain that with an ageing population and the introduction of more complex targeted therapies. Aging diseases such as dementia will also pose an increasing challenge that companies are already beginning to work cooperatively together to address. I believe the biggest shift over the next 10 to 20 years will see medicine change from treating illness to preventing illness or even curing some conditions. Diseases such as Hepatitis C and HIV will be controllable on one pill once a day and possibly even cured for some people. In addition, advances in medicine development will see immunological disorders and cancers, addressed on an individual basis through biomarkers and targeted therapies. This targeting will mean that patients will receive the right treatment for them leading to much better outcomes

Bruce Goodwin Managing Director, Janssen

The Australian medicines industry is still faced with meeting unmet patient need and producing better health outcomes for Australians. It starts with being committed to conducting patient trials in Australia and working to have those treatments registered and reimbursed to improve the lives of Australians in the years ahead. We are committed to doing the hard work so Australians who are confronted with cancer, macular degeneration, pulmonary hypertension, atrial fibrillation and deep vein thrombosis get the research-based innovative treatments they deserve. The medicines industry has done a lot to improve the lives of Australians over the years, but the challenge is to realise death and disability can be reduced through the quality use of innovative medicines. We are committed to playing our part to advance medicine in the decade ahead.

Rene Klemm Country Representative, Bayer HealthCare MEDICINES MILESTONES 32 A decade of persistence, prevention and promise

7 BAckground and Methods

7.1 project background

The Australian Medicines Industry commissioned Pretium to undertake a research project using Pretium’s Drug Tracker database to provide an overview of the last 10 years of TGA registrations in Australia, noting any relevant changes or trends. Particular focus was given to the primary national health priority areas (arthritis and musculoskeletal conditions, asthma, cancer control, cardiovascular health, diabetes, and mental health). The results of this research project are presented in this report and its appendices.

7.1.1 Pretium’s Drug Tracker database Pretium’s Drug Tracker database is a proprietary database owned and managed by Pretium Pty Ltd. The database contains information on regulatory approvals, PBAC outcomes and PBS listings. Pretium’s Drug Tracker delivers a comprehensive update on the progress of drugs through the approval and reimbursement systems and also provides regular in-depth trend analyses which can be used as a guide for forward planning.

7.1.2 National Health Priority Areas The National Health Priority Areas initiative was Australia’s response to the World Health Organisation’s global strategy “Health for All by the year 2000” and its subsequent revision. The initial 1996 set of NHPAs included cardiovascular health, cancer control, injury prevention and control and mental health. Diabetes mellitus was added in 1997, followed by asthma in 1999, arthritis and musculoskeletal conditions in 2002 and obesity in 2008. Currently, the eight NHPAs are listed below, along with each area’s particular focus (in brackets): • Arthritis and musculoskeletal conditions (osteoarthritis, osteoporosis, and rheumatoid arthritis); • Asthma; • Cancer control (lung cancer, melanoma, non-melanoma skin cancers, colorectal cancer, prostate cancer, non-Hodgkin lymphoma, cervical cancer, and breast cancer); • Cardiovascular health (coronary heart disease, stroke, heart failure, and peripheral vascular disease); • Diabetes mellitus (Type 1, Type 2, and gestational); • Injury prevention and control; • Mental health (depression); and • Obesity. The purpose of this report is to provide an overview of the TGA medications registered in Australia over the past ten years, as such the NHPAs of injury prevention and obesity have been excluded from the analysis as there were no medications registered for these indications. MEDICINES MILESTONES A decade of persistence, prevention and promise 33

7.2 Objectives

The primary objective of this project is to provide an overall picture of the regulatory profile in Australia over the past decade.

7.3 Methods

Pretium’s Drug Tracker is stored as a Microsoft Access® database which contains information relating to pharmaceuticals progressing through the reimbursement process in Australia. Basic information captured for all tables include: • generic drug name • indication • ATC code • brand name • sponsor name. In this project, regulatory approval information was captured from ADEC and ACPM recommendations from February 2002 to June 2010, at which point these became unavailable. For the period July 2010 to December 2011, relevant data was collected from the AusPAR documents and the date of recommendation used as a proxy for marketing approval dates. The information captured includes: • date of decision/recommendation • recommendation type (e.g. new chemical entity, new combination product, change to indications, etc) • recommendation notes (e.g. indication request) • drug type (e.g. tablets, injection, solution for injection, capsules and oral solution, etc). Using the Drug Tracker database, the above data are extracted through a variety of queries written in MS Access® and the use of Business Objects’ Crystal Reporting software and presented in the form of graphs and tables in the following sections.

7.3.1 Disease coding The allocated Anatomical Therapeutic Chemical (ATC) codes were used as the basis for coding. In this system, the active substances are divided into different groups according to the organ or system on which they act and their therapeutic, pharmacological and chemical properties. Drugs are classified in groups at five different levels. The drugs are divided into fourteen main groups (1st level), with pharmacological/therapeutic subgroups (2nd level). The 3rd and 4th levels are chemical/pharmacological/therapeutic subgroups and the 5th level is the chemical substance. The 2nd, 3rd and 4th levels are often used to identify pharmacological subgroups when that is considered more appropriate than therapeutic or chemical subgroups.3 (Table 6). Some of the categorisations are specific to the pharmacological and chemical properties of the drug and where this occurred and a specific condition for the category of the drug couldn’t be allocated, the drug was re-coded based on the condition the drug treats rather than the drug properties. For example, ATC code L contains both antineoplastic and anti-immunomodulating drugs. Under code L03AB are the drugs and in this category there are drugs indicated for multiple sclerosis, hepatitis C and hepatitis B. As the category could not be used as a whole, the drugs were recoded based on their registered indication/condition for their use. If there were difficulties in the coding of the drug to a particular condition, a medical opinion was sought and the coding was based on that recommendation. Where it was possible to keep all of the drugs together as a code (for example, J07: Vaccines) no re-coding was completed. A full list of the final coding for all drugs is available upon request. The coding was also based on the indication for which the drug was registered. A result of this is that some drugs which treat specific symptoms of a disease may be coded for the symptoms rather than the specific disease. For example, a drug that is indicated for seizures (and possibly used in epilepsy) would be coded as seizure. Similarly, the use of TGA indication information does not guarantee the final indication for which it may be available to the general public on the PBS as changes may have been made during the submission process.

3 http://www.whocc.no/atc/structure_and_principles/ MEDICINES MILESTONES 34 A decade of persistence, prevention and promise

Table 6 List of ATC codes

A Alimentary tract and D Dermatologicals A01 Stomatological preparations D01 for dermatological use A02 Drugs for acid related disorders D02 Emolients and protective A03 Drugs for functional gastrointestinal disorders D03 Preparations for wounds and ulcers A04 and antinauseants D04 incl antihistamines, aesthetics A05 Bile and liver therapy D05 Antipsoriatics A06 D06 Antibiotics and chemotherapeutics for dermatologic A07 Antidiarrheals, intestinal anti inflammatory D07 , dermatologicals preparations A08 Antiobesity preparations excl diet products D08 Antiseptics and disinfectants A09 Digestives incl. enzymes D09 Medicated dressings A10 Drugs in diabetes D10 Anti-acne preparations A11 D11 Other dermatological preparations A12 supplements

A13 Tonics G Genito urinary system and sex hormones A14 Anabolic agents for systemic use G01 Gynecological antiinfectives and antiseptics A15 Appetite G02 Other gynaecologicals A16 Other alimentary tract and metabolism products G03 Sex hormoines and modulators of the genital system G04 Urologicals B Blood and blood forming organs

B01 agents H Systemic hormonal preparations excl. sex hormones B02 H01 Pituitary and hypothalamic hormones and analogues B03 Antianemic preparations H02 Corticosteroids for systemic use B05 Blood substitutes and perfusions solutions H03 Thyroid therapy B06 Other haematological agents H04 Pancreatic hormones H05 Calcium homeostasis C Cardiovascular system

C01 Cardiac therapy J Antiinfectives for systemic use C02 Antihypertensives J01 Antibacterials for systemic use C03 J02 Antimycotics for systemic use C04 Peripheral vasodilators J04 C05 Vasoprotectives J05 Antivirals for systemic use C07 Beta blocking agents J06 Immune sera and Immunogloblins C08 Calcium channel blockers J07 Vaccines C09 Agents acting on the renin-angiotensin system C10 Lipid modifying agents MEDICINES MILESTONES A decade of persistence, prevention and promise 35

L Antineoplastic and immunomodulating agents R Respiratory system L01 Antineoplastic agents R01 Nasal preparations L02 Endocrine therapy R02 Throat preparations L03 R03 Drugs for obstructive airway diseases L04 Immunosuppressants R05 Cough and cold preparations R06 Antihistamines for systemic use M Musculo-skeletal system

M01 Antiinflammatory and antirheumtics products S Sensory organs M02 Topical products for and muscular pain S01 Ophthalmologicals Mo3 Muscle relaxants S02 Otologicals M04 Antigout preparations S03 Ophthalmological and ontological preparations M05 Drugs for treatment of bone disease Other drugs for disorders of the musculo-skeletal M09 V Various system V01 Allergens V03 All other therapeutic products N V04 Diagnostic agents N01 Anaesthetics V06 General nutrients N02 V07 All other non-therapeutic products N03 Antiepileptics V08 Contrast media N04 Anti-Parkinson drugs V09 Diagnostic radiopharmaceuticals N05 V10 Therapeutic radiopharmaceuticals N06 Psychoanaleptics

N07 Other nervous system drugs http://www.pbs.gov.au/browse/body-system?depth=1&codes=v

P products insecticides and repellent P01 P02 P03 Ectoparasiticeds incl scabicides, insecticides The Australian Medicines Industry makes the medicines and vaccines that save lives, reduce pain and prevent disease. We invest $1 billion a year in R&D and export $4 billion worth of medicines – more than the car and wine industries. The Australian Medicines Industry employs over 14,000 Australians, each contributing to the health and wealth of the nation. Find out more at www.ausmedindustry.com.au

The Australian Medicines Industry are the following companies: Abbott Australasia Pty Ltd / Actelion Pharmaceuticals Australia Pty Limited / Allergan Australia Pty Ltd / Amgen Australia Pty Ltd / Andrew’s Refrigerated Transport / Astellas Pharma Australia / AstraZeneca Pty Ltd / Baxter Healthcare Pty Limited / Bayer Australia Limited / Biogen Idec Australia Pty Ltd / Boehringer Ingelheim Pty Limited / Bristol-Myers Squibb Australia Pty Ltd / Celgene Pty Ltd / Commercial Eyes Pty Ltd / Covance Pty Ltd / CSL Ltd / Eli Lilly Australia Pty Ltd / FIT-BioCeuticals Limited / Fresenius-Kabi Australia Pty Ltd / Genzyme Australasia Pty Ltd / Gilead Sciences Pty Ltd / GlaxoSmithKline Australia Pty Ltd / IDT Australia Limited / IMS Health Australia Pty Ltd / iNova Pharmaceuticals Pty Ltd / Invida Australia Pty Ltd / Ipsen Pty Ltd / IQnovate Ltd / Iris Interactive Pty Ltd / Janssen / Kendle Pty Limited / KMC Health Care / Leo Pharma / Lundbeck Australia Pty Ltd / Merck Serono Australia / MSD / Mundipharma Pty Limited / Norgine Pty Limited / Novartis Pharmaceuticals Australia Pty Ltd / Novo Nordisk Pharmaceuticals Pty Ltd / Nycomed: a Takeda Company / Pfizer Australia Pty Limited / Pretium Pty Ltd / PricewaterhouseCoopers / Princeton Publishing Pty Ltd / Quintiles Australia Pty Ltd / Roche Products Pty Limited / Sanofi Pty Ltd / Servier Laboratories (Aust) Pty Ltd / Shire Australia Pty Ltd / Smith & Nephew Pty Ltd / UCB Australia Pty Ltd / Vifor Pharma Pty Ltd.