Adoptions and Orphans

Posts Tagged ‘Australia’

 A 1984 modification to the ODA defined an Orphan Disease as any condition affecting fewer than 200,000 people in the US. The US was followed by both Japan and the EU which have also introduced their own orphan drug legislation in 1993 and 2000 respectively.

Industry has responded to this call for action and the FDA has approved over 270 orphan drugs in the US whilst there are another 583 listed in the Medtrack database as being in ongoing development. This has been motivated by the special incentives provided by the FDA and the EU including enhanced market exclusivity, research grants and tax incentives.

Orphan drugs present opportunities to pharma companies looking to move into more niche indications and away from mass market indications that have very low levels of clinical unmet need. Although there are many successful orphan drugs that have made large profits for their developers (Gleevec, NovoSeven etc) the development of these products must be approached carefully. This report uses a mixture of case studies and data from secondary sources to elucidate the key issues in the development of orphan drugs and ways to navigate them.

Key features of this report

• Overview of orphan drugs legislation in the US, EU, Japan and Australia including details of key incentives for developers and history of orphan designations and approvals.

• Evaluation of clinical trial and approval strategies including data on current clinical trial designs and strategies to maximize the chances of clinical trial success through the use of adaptive clinical trials.

• Overview of orphan drug reimbursement issues in both the US and the EU including details of the importance of the UK’s NICE and the development of novel risk sharing agreements.

• Examination of the role being played in orphan drug development by leading players in the pharmaceutical and biotech industry and the motivations behind this.

Scope of this report

• Gain an understanding of key orphan drugs legislation and the incentives available to the developers of drugs which successfully gain orphan designation.

• Get a quick overview of the overall orphan drug development pipeline in the EU and US.

• Gain strategic insight into the key differences between the development of orphan drugs and non-orphan drugs including the importance of patient advocacy groups.

• Discover key strategies to maximize revenues from orphan drugs through gaining market access and broadening the product label.

Key Market Issues

• Gaining orphan designation for a product confers access to various incentives. Once a product is granted orphan designation its developers gain a variety of incentives including extended market exclusivity, tax credits and research grants. These incentives have motivated the development of hundreds of products for orphan indications in the US, EU and Japan.

• The move away from reliance on blockbuster products towards niche indications is driving heightened interest in orphan drugs from companies of all sizes Orphan drugs lie at the opposite spectrum to blockbuster drugs and present Big Pharma with opportunities to enter specialist niche markets. Although on first analysis orphan drugs should not be particularly interesting to the pharma industry on account of their limited patient potential, by 2006 there were 50 orphan products with sales in excess of $200m of which 19 were blockbuster products and it is this which sustains the pharma industry’s interest in developing orphan drugs.

• Cancer continues to have the largest number of pipeline orphan drugs. This is largely motivated by the nature of cancer which allows it to be stratified into niche sub-diseases which qualify as orphan diseases. Developing orphan drugs for cancer indications makes additional sense as it presents opportunities to gain access to further orphan indications which have a similar disease mechanism.

Key findings from this report

• The developers of orphan drugs receive several incentives from the governments of countries that grant orphan status including extended market exclusivity, fast track assessment, research grants and tax incentives.
• There are 583 orphan drugs in active development with Cancer being the leading indication for which they are being developed with over 100 products in active development in the US alone.
• The development of an orphan drug is often a strategy pursued by small biotech and pharma companies with a view to licensing the product out in the later stages of development as part of an exit strategy. One example of this is PharmaMar’s development of Yondelis which has involved a complex network of partnerships as a source of both funding and expertise.

Key questions answered

• What is the key legislation concerning orphan drugs in the US, Japan and the EU?
• What incentives are available to the developers of orphan drugs in the US, Japan and the EU?
• What is the involvement of leading pharmaceutical companies in the development of orphan drugs?
• Why are orphan drugs an attractive proposition for pharmaceutical companies looking to expand their product portfolios?
 
 
  Table of Contents : 
Table of Contents
Opportunities in orphan drugs
Executive summary 8
Orphan drug legislation 8
The orphan drug pipeline 9
Orphan drug development trends and strategies 10
Orphan drug strategies 11
Chapter 1 Orphan drug legislation 14
Summary 14
Introduction 15
Orphan drug legislation in the US 15
Orphan drugs act 1983 15
Orphan drug designation 16
Orphan grant program 18
Orphan drug legislation in the EU 19
The Committee for Orphan Medicinal Products 19
Orphan designation process 20
EU incentives for orphan medicinal product developers 20
Orphan drug legislation in Japan 21
Incentives for orphan drug developers in Japan 22
Orphan drug legislation in Australia 23
Incentives for orphan drug developers in Australia 23
Chapter 2 The orphan drug pipeline 26
Summary 26
Introduction 27
Oncology 29
Istodax – romidepsin 29
Yondelis – trabectedin 30
Omapro – omacetaxine 33
Clolar – clofarabine 34
Onrigin – laromustine 35
TM601 36
EGEN – 001 37
Central nervous system 38
Zenas – amifampridine 38
H P Acthar Gel 39
ITI111 – midazolam 42
Respiratory and pulmonary systems 43
Surfaxin 43
Anti-infectives 44
Cayston – aztreonam lysine 44
ABthrax – raxibacumab 46
Autoimmune and inflammation 48
EN 101 48
Genetic diseases and dysmorphic syndromes 49
Uplyso – taliglucerase alfa 49
Chapter 3 Orphan drug development trends and issues 54
Summary 54
Introduction 55
Clinical trial and approval strategies for orphan drugs 55
Adaptive clinical trials in orphan drug development 58
The role of patient advocacy groups 62
Patient group/industry R&D partnerships 63
Orphan drug reimbursement issues 64
Orphan drug access in the US 65
Orphan drug access in the EU 65
Pharmaceutical risk-sharing agreements 70
Chapter 4 Orphan drug strategies 74
Summary 74
Introduction 75
The move towards niche indications 75
Opportunities in orphan drugs 76
Small pharma and biotech 77
Big pharma and established biotech 78
Pfizer 78
GlaxoSmithKline 82
Novartis 83
Sanofi-Aventis 85
Roche 86
Johnson & Johnson 88
Merck & Co 89
Eli Lilly 90
Bayer 91
Orphan drug specialists 92
Genzyme 92
Actelion 94
Strategies for sales growth for orphan drugs 96
Expansion into non-orphan indications 97
Expansion into further orphan indications 98
Using biomarkers to develop orphan drugs 101
Conclusion 104
Glossary 105
Index 106

List of Figures
Figure 1.1: Number of products receiving orphan designation in the US, 1984-December 2009 16
Figure 1.2: Number of Orphan drug approvals in the US, 1990-2008 17
Figure 1.3: Average NDA approval time, (months), 1995-2008 18
Figure 2.4: Therapeutic focus of products given orphan designation in the EU, 2009 27
Figure 2.5: Therapeutic focus of products given orphan designation in the US, 2009 28
Figure 2.6: Yondelis deals and alliances structure 32
Figure 2.7: EGEN-001 mode of action 38
Figure 2.8: Licensing deals for H P Acthar Gel 41
Figure 3.9: Blinding in US orphan drug trials, 2009 57
Figure 3.10: Randomization in US orphan drug trials, 2009 57
Figure 3.11: Comparators in US orphan drug trials, 2009 58
Figure 3.12: Reasons for adopting adaptive clinical trials 60
Figure 3.13: Overview of strategic considerations when designing adaptive clinical trials 61
Figure 3.14: Overview of operational considerations when designing adaptive trials 62
Figure 3.15: Relationship between annual cost of treatment (€) per patient and prevalence 69
Figure 3.16: The relationship between cost-effectiveness and societal value 70
Figure 4.17: Blockbuster to orphan drugs 75
Figure 4.18: Required conditions for stratified medicine 102

List of Tables
Table 1.1: Comparison of orphan drug systems 24
Table 2.2: Yondelis orphan designations 30
Table 2.3: Surfaxin’s US orphan designations by date 43
Table 3.4: Recent deals involving CFF and pharma companies 64
Table 4.5: Pfizer’s orphan designations since 2003 79
Table 4.6: Pfizer’s active orphan programs 79
Table 4.7: GlaxoSmithKline’s marketed US orphan drug portfolio 82
Table 4.8: GlaxoSmithKline’s active orphan programs 82
Table 4.9: Novartis’ marketed US orphan drug portfolio 84
Table 4.10: Novartis’s active orphan programs 84
Table 4.11: Sanofi-Aventis’s active orphan programs 86
Table 4.12: Roche’s marketed US orphan drug portfolio 86
Table 4.13: Roche’s active orphan programs 87
Table 4.14: J&J’s marketed US orphan drug portfolio 88
Table 4.15: J&J’s active orphan programs 89
Table 4.16: Merck & Co’s active orphan programs 90
Table 4.17: Eli Lilly’s marketed US orphan drug portfolio 90
Table 4.18: Eli Lilly’s active orphan programs 90
Table 4.19: Bayer’s marketed US orphan drug portfolio 91
Table 4.20: Genzyme’s global marketed orphan product portfolio ($m), 2008 92
Table 4.21: Genzyme’s lysosomal storage disorder product history 94
Table 4.22: Actelion’s global marketed portfolio ($m), 2008 95
Table 4.23: Actelion’s product pipeline 96
Table 4.24: Gleevec’s US orphan indications 99
Table 4.25: Nutropin’s US orphan indications 100
Table 4.26: Orphan designated products with at least one associated clinically validated biomarker 103 
 

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hi guys,

my name is denny and i live in Australia. One day i was surfing on net and all of sudden i faced the power cut. Actualy it was not only me who is facing that problem but the whole street batteling with it. But in that time i saw a house 5 blocks ahead, is still lighting up.

I went to that house and i was amazed to see that they were have no idea of power cut. Then i asked them what is the secret of their electricity. They show me their roof next day in morning. I saw a solar panel there. I asked them how it works? And where they find it??? They told me that they bought a guide about how to make your energy system? And they made it in their house.

I thought they were kidding with me!! But they show me the guide and even they also show me how they making money with it. For making money they are just assembling solar panal (costs about $200) and they salling it around $500 dollars.

I thought what an amezing idea!!!

Lets try it once. I went to that site which they told me and bought that guide. Now i am happy from my 2 way income

1st way :- from saving electricity bill upto $50/month.

2nd way :- i sold out 10 solar panal on price of $400 in just a month.

Now today i feels, that power cutt change my life and i got the new magic source of income on very less price. Then i thought about that old saying “ no one can steel your knowledge, it can make you the king or  bagger”

The best part of all this that so many sites are salling this but i bought it from orphanconsul.blogspot.com this site is dedicated to help orphans and if you want to earn some blessings from poors and orphans with good money then please buy it only from this site. Thank you

A tropical jewel in the Indian Ocean; a land of lush wetlands and thriving lagoon life; a country so filled with joy it will inspire you. This is Sri Lanka. Holidays to this small yet multi-faceted island country are a treasure trove of unexpected delights.

The stunning natural beauty of the island’s tropical landscape and the idyllic beaches of its coast make holidays in Sri Lanka extremely inviting. The temperate climate and renowned hospitality of the warm and engaging locals, has seen Sri Lanka holidays become increasingly popular.

From the rich cultural heritage of the island has emerged a diverse and eclectic ethnic community, yet the result is distinctly Sri Lanka. Holidays to the island are not just about relaxing on the beach – although the beaches are outstanding. And they aren’t just about the culture – although the history is fascinating. Holidays in Sri Lanka are truly experiential – with the sum of everything you see and do; eat and drink; hear and feel; combining to form the perfect portrait of a memory of Sri Lanka.

Pinnawela Elephant Orphanage

You will see elephants everywhere on your Sri Lanka holidays. They are depicted in carvings, artwork and coats of arms. They are almost a national emblem and one of the most rewarding things you can do in Sri Lanka is to visit the Pinnawela Elephant Orphanage. Halfway between the towns of Colombo and Kandy in the central hills, the orphanage was established as a sanctuary in 1975 with four motherless elephant calves. Many offspring from those original four now live happily in the orphanage but it has also more recently become an important breeding centre. Now housing over 70 elephants – the largest group of captive elephants in the world – the centre is a beacon for conservation as well as being a popular tourist site.

Polonnaruwa

The ancient city of Polonnaruwa is wonderfully well-preserved and a must see on your holidays in Sri Lanka. It was the island’s capital between the 11th and 13th Century and is filled with ancient architecture such as the intricate Dagobas, strikingly beautiful statues, and monuments. It is an easy area to explore as it is so compact, and it is lovely to wander around the ancient statues and feel the history emanating from this ancient site. Today, excavations and conservation are underway in order to further preserve these fascinating ruins.

Bentota Beaches

Reputedly the best beaches to visit on your Sri Lanka holidays are around the Bentota and Aluthgama areas. The wide sandy beaches are lapped by the warm waters of the Indian Ocean and are a popular location for diving, fishing and of course swimming. Although there are a lot of resorts in the area and it can get quite busy, it is still possible to find some quieter beaches on which to take a long, relaxing solitary stroll. You can also take a river cruise up the Bentota River to enjoy the wildlife and experience the unique geography of the river which, in some places is separated from the sea by only 100 metres of shoreline.

Statistics say that the chances of having a close encounter with an elephant are higher in Sri Lanka than anywhere else in the world. So if you’ve always dreamed of getting up close and personal with Dumbo’s distant cousins, a Sri Lanka holiday is your opportunity to make those wishes come true.

There are around about 2,500 to 3,000 Sri Lankan elephants living on the island and with a land mass of just over 25,000 square miles, it doesn’t take a mathematician to figure out that your odds of seeing an elephant are pretty good. Of course it doesn’t mean you’ll walk out of your hotel and find one waiting at the bus stop, but with a little planning you can turn your Sri Lanka holiday into a totally “elephanticious” experience.

National Parks

Sri Lanka abounds with national parks. It is a credit to the culture and also the past and present governments of the country, that wildlife conservation is an issue high on the agenda of national importance. One of the best opportunities to spend time really getting to know the wild elephants is to observe them going about their daily business in their natural habitat. You will have the best opportunity for elephant-spotting on your Sri Lanka holiday if you head to Yala National Park, Minneriya National Park, Udawalawe National Park and Wilpattu National Park. The elephant population roams freely and in most of the parks you can take a specialised jeep tour with trained rangers. They will give you a fascinating insight into the behaviour and breeding patterns of these wonderful creatures. The advantage of visiting elephants in a national park is that, even though they are the most popular inhabitants, there are many other intriguingly elusive species that manage to keep a lower profile but which you may encounter as well.

Elephant Orphanage

One of the most popular attractions of a holiday in Sri Lanka is a visit to the Pinnawala Elephant Orphanage. Originally set up to care for five abandoned elephant calves over thirty years ago, it has evolved into one of Sri Lanka’s premier tourist attractions, in a self-propagating project; as the animals matured and bred so too the number of visitors swelled.

The sensitive approach the orphanage takes to the nurturing of its residents makes Pinnawala a very special place. The elephants themselves are extremely gentle and you can walk amongst them freely without fear. You can wander down to the river and watch them wallowing in the shallows, and they are so used to human contact that often a cheeky trunk will curl out to nudge you in a shy, curious greeting. A visit to the orphanage is a magical day out and will be a highlight of any Sri Lanka holiday.

Elephant Back Safaris

But surely the platinum experience for any elephant-lover would have to be a safari through the exquisite wilderness of Sri Lanka on the back of your favourite new friend. These safaris are a wonderful way to really get to know these beautiful gentle giants. Not only will you spend the day on their backs slowly ambling through the terrain, but you can also swim in the river with them and have the opportunity to groom them. It is a truly relaxing day and an incredibly bonding experience. By the end of the safari you will have completely fallen under the spell of your companion and it will be almost impossible to say goodbye.

• ISBN13: 9780970183279
• Condition: NEW
• Notes: Brand New from Publisher. No Remainder Mark.

Product Description
From Alaska to Australia the word is spreading. Adoptive parents are discovering the enormous value of adoption lifebooks. But then the questions begin. Where do I start? What information should be included? Do I let my child bring it to school?

Beth O’Malley M.Ed. provides the answers to these and more. In her best selling book, LifeBooks: Creating a Treasure for the Adopted Child Beth guides you though the process, step-by-step and page by page as if she were right there with you.

Learn about the difference between a scrapbook or baby book and a lifebook. Or explaining tough truths, dealing with secrets and which pages are essential.

Newly revised 2002

Dozens of real life stories

Lists of hard-to-find lifebook resource websites

Sample pages for international and domestic

Special waiting parent section.

If you get really stuck, there are three full-length examples in the back section, including one for China adoptions.

Her life experiences as an adoptee combined with doing lifebook seminars with adoptive parents all over the country, gives Beth a special perspective on lifebooks. Most importantly, Beth has made countless lifebooks with children in her role as an adoption specialist in Massachusetts.

Beth O’Malley has helped thousands of adoptive families give their children the answers and security they crave.

This book is an indispensable guide to making your child’s lifebook. You will refer to it for years to come!

LifeBooks : Creating a Treasure for the Adopted Child

I have a girlfriend from a poor family in the Philippines who I will marry soon and bring to Australia with her 6 year old daughter. But she has a 7 year old niece living with her who she has cared for since birth (abandoned by her own parents). My girlfriend has been her only mother figure and provider all her life, and the two little girls are virtually sisters for that reason. She will have to be left behind to live a sad and lonely life with her grandmother unless my girlfriend can adopt her and bring both girls to Australia. My girlfriend can contact the girl’s birth parents for permission. Unknown yet if they will agree. Does anyone know the costs and complications involved if my girlfriend was to adopt her niece? At this point we need to know what adoption might cost.

Adopting a child, by all means, is a big step forward in life. It must not be an impulsive act, but a well thought out course of action.

Through adoption, you add someone to your family. There may be different reasons for a couple to choose in favour of adoption. For example, a celebrity like Angelina Jolie has been adopting to suit her fancy for a big and global family. However, the majority of couples adopt because they are not able to have their own biological child.

Whatever may be your reasons for adopting a child, go for it only after asking the following questions:

1. Do you find yourself willing to understand and grasp the day to day life in orphanage? This knowledge will help you extend a sensitive transition from the orphanage to the environment of your home.

2. Are you aware that children belonging to orphanages are at higher risk of suffering from developmental issues? These may be emotional issues or developmental delays.

3. Do you understand that an adopted child will face a major change from an orphanage to a home environment? It may be difficult for the child. The child will naturally take time to adjust to a home environment after living for a long time in an orphanage. He or she will take time to learn to trust. It would be difficult for him or her to accept your family and your affection.

If you feel you are confident about your decision to adopt, visit www.cyf.vic.gov.au This is a website of the State Government of Victoria, Australia. You will find all the help and information about adoption here on this site.

I keep hearing how Australia and the UK have different, and some say better, adoption practices.

How is it different?
What makes it better?

I’d happily look it up myself, but I keep getting sites on adopting Australian Shepard dogs! LOL!!

I’m really curious.
I don’t mean to sound like a arrogant American, or that I’m looking down on anything that isn’t “Truth, Justice and The American Way”. I just can’t find any info on the matter.

Thanks!

Opportunities in Orphan Drugs: Strategies for developing maximum returns from niche indications
 
 The US government introduced the Orphan Drugs Act (ODA) in 1983 as a means to provide incentives to drug developers so that they would develop treatments for diseases for which incidence is so low that that normal market forces are unable to deliver effective medication for these conditions. A 1984 modification to the ODA defined an Orphan Disease as any condition affecting fewer than 200,000 people in the US. The US was followed by both Japan and the EU which have also introduced their own orphan drug legislation in 1993 and 2000 respectively.  ( http://www.bharatbook.com/detail.asp?id=132643&rt=Opportunities-in-Orphan-Drugs-Strategies-for-developing-maximum-returns-from-niche-indications.html )
 
 Industry has responded to this call for action and the FDA has approved over 270 orphan drugs in the US whilst there are another 583 listed in the Medtrack database as being in ongoing development. This has been motivated by the special incentives provided by the FDA and the EU including enhanced market exclusivity, research grants and tax incentives.
 
 Orphan drugs present opportunities to pharma companies looking to move into more niche indications and away from mass market indications that have very low levels of clinical unmet need. Although there are many successful orphan drugs that have made large profits for their developers (Gleevec, NovoSeven etc) the development of these products must be approached carefully. This report uses a mixture of case studies and data from secondary sources to elucidate the key issues in the development of orphan drugs and ways to navigate them.
 
 Key features of this report
 
 • Overview of orphan drugs legislation in the US, EU, Japan and Australia including details of key incentives for developers and history of orphan designations and approvals.
 • Evaluation of clinical trial and approval strategies including data on current clinical trial designs and strategies to maximize the chances of clinical trial success through the use of adaptive clinical trials.
 • Overview of orphan drug reimbursement issues in both the US and the EU including details of the importance of the UK’s NICE and the development of novel risk sharing agreements.
 • Examination of the role being played in orphan drug development by leading players in the pharmaceutical and biotech industry and the motivations behind this.
 
 Scope of this report
 
 • Gain an understanding of key orphan drugs legislation and the incentives available to the developers of drugs which successfully gain orphan designation.
 • Get a quick overview of the overall orphan drug development pipeline in the EU and US.
 • Gain strategic insight into the key differences between the development of orphan drugs and non-orphan drugs including the importance of patient advocacy groups.
 • Discover key strategies to maximize revenues from orphan drugs through gaining market access and broadening the product label.
 
 To know more and to buy a copy of your report feel free to visit : http://www.bharatbook.com/detail.asp?id=132643&rt=Opportunities-in-Orphan-Drugs-Strategies-for-developing-maximum-returns-from-niche-indications.html
 
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What was the event that made preserving the natural family so important in these countries and what can Americans do to create an adoption reform that is similar to England’s and Australia’s adoption laws?