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IHS Global Insight’s special multi-client study provides a comprehensive overview of the orphan drugs
market and associated legislation. It will enable pharmaceutical and biotech companies to define the
market opportunities presented by orphan drugs. Should pharmaceutical and biotech companies
include orphan drugs in their research and development strategies? What are the benefits?
DELIVERABLESAn electronic report (approximately 150 pages) delivered in Microsoft Word® format with eight detailed country profiles that include:
• Market trends for orphan drugs
• Executive summary, interview analysis, and interview extracts
Comparative cross-country analysis presented in Microsoft PowerPoint® (approximately 50 slides)
QUALITY CERTIFICATIONIHS GLOBAL INSIGHT FRANCE was acquired by IHS in late 2008.IHS GLOBAL INSIGHT FRANCE commits itself to due care and to the ethical standards (confidentiality, exclusivity)of the industry. As a member of « Syntec études marketing et opinion », IHS GLOBAL INSIGHT FRANCE appliesand requires application of:
The international code CCI/ ESOMAR
The French legislation n° 78-17 of 6 January 1978
EU directives
DAFSA products of IHS GLOBAL INSIGHT FRANCE are certified ISO 20252 (certificateN°QUAL/2008/33149, 15 December 2008, delivered by AFNOR CERTIFICATION). DAFSA was previously certified NF « Qualité des études de marché et d’opinion ».
IHS GLOBAL INSIGHT HEALTHCARE & PHARMACEUTICAL SERVICESIHS Global Insight’s Healthcare & Pharmaceutical practice provides a portfolio of intelligence solutions tooptimize the performance of companies and organizations across the pharmaceutical, biotech, and genericssectors. Our key focus is to provide actionable insights to support strategic decision making — particularly in the fields of market access, pricing and reimbursement (P&R), emerging markets,generics strategies, therapeutic development pathways, and general competitiveintelligence.
ORPHAN AND ULTRA-ORPHAN DRUGS:ATTACHING VALUE TO TREATMENTS FOR RARE DISEASES
THE POWER OF PERSPECTIVE
CONTACT US Web: www.ihsglobalinsight.com E-mail: [email protected]
NORTH AMERICA EUROPE AFRICA ASIAU.S./Canada 1 800 933 3374 Frankfurt +49 (0)69 20973 0 Pretoria +27 (0)12 665 5420 Beijing +86 10 6505 2966
London +44 (0)20 3159 3300 Gurgaon +91 124 454 2600Milan +39 02 86481 1 MIDDLE EAST Shanghai +86 21 5228 1470Paris +33 (0)1 55 45 2600 London +44 (0)20 3159 3300 Singapore +65 6576 5300
Tokyo +81 (0)3 5791 7291
ABOUT IHS GLOBAL INSIGHT: IHS Global Insight (www.ihsglobalinsight.com) provides the most comprehensive economic and financial information on countries, regions, and industries available from any source. IHS Global Insight’s immediate and insightful analysis of market conditions and key events enables innovative and successful decision making for clients around the world. IHS Global Insight employs approximately 700 employees in 13 countries.
10/09
ORPHAN AND ULTRA-ORPHAN DRUGS: ATTACHING VALUE TO TREATMENTS FOR RARE DISEASES
The risks of developing new medicines are increasing, as pharmaceutical and biotech companies face stringentpricing and reimbursement conditions that do not always cover their research and development investments. It is also becoming more difficult for the pharmaceutical industry to develop medicines that improve on existingtreatments for major diseases that have already seen breakthroughs in recent years.
Pharmaceutical and biotech companies are increasingly focusing on rarer “orphan” diseases, where there are fewercompetitors and significant regulatory investment incentives.
In our new multi-client study, Orphan and Ultra-Orphan Drugs: Attaching Value to Treatments for Rare Diseases, we explore the opportunities that orphan drugs present to pharmaceutical and biotech companies. We examineregulatory incentives, emerging competition, and the value of orphan drugs for Australia, France, Germany, Italy,Japan, Spain, the United Kingdom, and the United States.
The report answers key questions:What are the specific regulatory incentives in terms of clinical guidelines, patent registration, marketing approval,compassionate use, and specific conditions of prescription?
How does orphan status impact pricing, reimbursement conditions, and the timeline to market per country?
Who are the main stakeholders in the orphan drugs market? How are they involved?
Which orphan indications are most attractive?
What are the main drivers within the orphan drugs market for each country? What are the barriers? Is there a backlash emerging against orphan drugs?
What are the emerging regulatory and market trends?
How can patient advocacy groups influence regulation and corporate strategy?
METHODOLOGYUsing a combination of primary and secondary research, thereport provides in-depth insight into the orphan drugs market. It offers unique analysis based on detailed interviews withregulatory authorities, investigating their knowledge andperspectives on the market.
We encourage clients to review and provide input into theinterview guidelines and the scope of the analysis, to ensure that we accommodate specific business needs. The marketresearch is carried out by our extensive network of localconsultants, who work closely with our team of pharmaceutical experts and analysts.
Interview guidelines and other documents are drafted in English and then translated into the local language and reviewed by a professional translator. CVs and the translated interview guides are made available to clients upon request.
Primary Research: A Qualitative Approach UsingIn-depth Interviews
Telephone interviews of approximately 45 minutes
Email exchanges following the interview for furtherinformation
The telephone interviews are conducted by a qualifiedconsultant, and then reviewed by the IHS Global Insightproject manager to avoid inconsistencies and maintainaccurate results.
This report includes 15 – 20 interviews with regulatoryauthorities, investigating:
Conditions for market approval of orphan drugs compared with non-orphan drugs
Conditions of market access and an approximatetime-to-market assessment for new orphan drugs
Conditions of pricing and reimbursement compared withnon-orphan drugs
The processes of negotiations with the pharmaceuticaland biotech industry for marketing approval, pricing andreimbursement, and orphan drugs
Specific advantages granted to orphan drugs, such asincentive packages or higher prices
Expected trends in the orphan drugs market
Secondary ResearchExtensive research based on secondary sources anddata, analyzing market trends for orphan drugs
Sources include research materials from the EuropeanMedicine Agency, Federal Drug Agency, IHS GlobalInsight’s Pharmaceutical and Healthcare Group, and national pharmaceutical papers such asPharmaceutiques (France) and Farmacista (Italy)
Extensive analysis of the legal and regulatory framework
TABLE OF CONTENTS
I Marketing approval
Regulatory framework
• Drugs in general
• Orphan drugs
Ultra-orphan drugs
Timing
• Drugs in general
• Orphan drugs
• Ultra-orphan drugs
II Market trends of orphan drugs
Main stakeholders on the market
Main products on the market and therapeutic areas involved
Market data
III Pricing and reimbursement
Regulatory framework
• Drugs in general
• Orphan drugs
• Ultra-orphan drugs
Case studies
• Orphan drugs
• Ultra-orphan drugs
IV Executive summary
Opportunities and barriers to thedevelopment of orphan drugs
Expected trends
The report includes:Details of the legal and regulatory framework for orphan drugs in each country, with a specific focus on European legislation
An overview of current orphan drugs on the market
ORPHAN AND ULTRA-ORPHAN DRUGS: ATTACHING VALUE TO TREATMENTS FOR RARE DISEASES
The risks of developing new medicines are increasing, as pharmaceutical and biotech companies face stringentpricing and reimbursement conditions that do not always cover their research and development investments. It is also becoming more difficult for the pharmaceutical industry to develop medicines that improve on existingtreatments for major diseases that have already seen breakthroughs in recent years.
Pharmaceutical and biotech companies are increasingly focusing on rarer “orphan” diseases, where there are fewercompetitors and significant regulatory investment incentives.
In our new multi-client study, Orphan and Ultra-Orphan Drugs: Attaching Value to Treatments for Rare Diseases, we explore the opportunities that orphan drugs present to pharmaceutical and biotech companies. We examineregulatory incentives, emerging competition, and the value of orphan drugs for Australia, France, Germany, Italy,Japan, Spain, the United Kingdom, and the United States.
The report answers key questions:What are the specific regulatory incentives in terms of clinical guidelines, patent registration, marketing approval,compassionate use, and specific conditions of prescription?
How does orphan status impact pricing, reimbursement conditions, and the timeline to market per country?
Who are the main stakeholders in the orphan drugs market? How are they involved?
Which orphan indications are most attractive?
What are the main drivers within the orphan drugs market for each country? What are the barriers? Is there a backlash emerging against orphan drugs?
What are the emerging regulatory and market trends?
How can patient advocacy groups influence regulation and corporate strategy?
METHODOLOGYUsing a combination of primary and secondary research, thereport provides in-depth insight into the orphan drugs market. It offers unique analysis based on detailed interviews withregulatory authorities, investigating their knowledge andperspectives on the market.
We encourage clients to review and provide input into theinterview guidelines and the scope of the analysis, to ensure that we accommodate specific business needs. The marketresearch is carried out by our extensive network of localconsultants, who work closely with our team of pharmaceutical experts and analysts.
Interview guidelines and other documents are drafted in English and then translated into the local language and reviewed by a professional translator. CVs and the translated interview guides are made available to clients upon request.
Primary Research: A Qualitative Approach UsingIn-depth Interviews
Telephone interviews of approximately 45 minutes
Email exchanges following the interview for furtherinformation
The telephone interviews are conducted by a qualifiedconsultant, and then reviewed by the IHS Global Insightproject manager to avoid inconsistencies and maintainaccurate results.
This report includes 15 – 20 interviews with regulatoryauthorities, investigating:
Conditions for market approval of orphan drugs compared with non-orphan drugs
Conditions of market access and an approximatetime-to-market assessment for new orphan drugs
Conditions of pricing and reimbursement compared withnon-orphan drugs
The processes of negotiations with the pharmaceuticaland biotech industry for marketing approval, pricing andreimbursement, and orphan drugs
Specific advantages granted to orphan drugs, such asincentive packages or higher prices
Expected trends in the orphan drugs market
Secondary ResearchExtensive research based on secondary sources anddata, analyzing market trends for orphan drugs
Sources include research materials from the EuropeanMedicine Agency, Federal Drug Agency, IHS GlobalInsight’s Pharmaceutical and Healthcare Group, and national pharmaceutical papers such asPharmaceutiques (France) and Farmacista (Italy)
Extensive analysis of the legal and regulatory framework
TABLE OF CONTENTS
I Marketing approval
Regulatory framework
• Drugs in general
• Orphan drugs
Ultra-orphan drugs
Timing
• Drugs in general
• Orphan drugs
• Ultra-orphan drugs
II Market trends of orphan drugs
Main stakeholders on the market
Main products on the market and therapeutic areas involved
Market data
III Pricing and reimbursement
Regulatory framework
• Drugs in general
• Orphan drugs
• Ultra-orphan drugs
Case studies
• Orphan drugs
• Ultra-orphan drugs
IV Executive summary
Opportunities and barriers to thedevelopment of orphan drugs
Expected trends
The report includes:Details of the legal and regulatory framework for orphan drugs in each country, with a specific focus on European legislation
An overview of current orphan drugs on the market
IHS Global Insight’s special multi-client study provides a comprehensive overview of the orphan drugs
market and associated legislation. It will enable pharmaceutical and biotech companies to define the
market opportunities presented by orphan drugs. Should pharmaceutical and biotech companies
include orphan drugs in their research and development strategies? What are the benefits?
DELIVERABLESAn electronic report (approximately 150 pages) delivered in Microsoft Word® format with eight detailed country profiles that include:
• Market trends for orphan drugs
• Executive summary, interview analysis, and interview extracts
Comparative cross-country analysis presented in Microsoft PowerPoint® (approximately 50 slides)
QUALITY CERTIFICATIONIHS GLOBAL INSIGHT FRANCE was acquired by IHS in late 2008.IHS GLOBAL INSIGHT FRANCE commits itself to due care and to the ethical standards (confidentiality, exclusivity)of the industry. As a member of « Syntec études marketing et opinion », IHS GLOBAL INSIGHT FRANCE appliesand requires application of:
The international code CCI/ ESOMAR
The French legislation n° 78-17 of 6 January 1978
EU directives
DAFSA products of IHS GLOBAL INSIGHT FRANCE are certified ISO 20252 (certificateN°QUAL/2008/33149, 15 December 2008, delivered by AFNOR CERTIFICATION). DAFSA was previously certified NF « Qualité des études de marché et d’opinion ».
IHS GLOBAL INSIGHT HEALTHCARE & PHARMACEUTICAL SERVICESIHS Global Insight’s Healthcare & Pharmaceutical practice provides a portfolio of intelligence solutions tooptimize the performance of companies and organizations across the pharmaceutical, biotech, and genericssectors. Our key focus is to provide actionable insights to support strategic decision making — particularly in the fields of market access, pricing and reimbursement (P&R), emerging markets,generics strategies, therapeutic development pathways, and general competitiveintelligence.
ORPHAN AND ULTRA-ORPHAN DRUGS:ATTACHING VALUE TO TREATMENTS FOR RARE DISEASES
THE POWER OF PERSPECTIVE
CONTACT US Web: www.ihsglobalinsight.com E-mail: [email protected]
NORTH AMERICA EUROPE AFRICA ASIAU.S./Canada 1 800 933 3374 Frankfurt +49 (0)69 20973 0 Pretoria +27 (0)12 665 5420 Beijing +86 10 6505 2966
London +44 (0)20 3159 3300 Gurgaon +91 124 454 2600Milan +39 02 86481 1 MIDDLE EAST Shanghai +86 21 5228 1470Paris +33 (0)1 55 45 2600 London +44 (0)20 3159 3300 Singapore +65 6576 5300
Tokyo +81 (0)3 5791 7291
ABOUT IHS GLOBAL INSIGHT: IHS Global Insight (www.ihsglobalinsight.com) provides the most comprehensive economic and financial information on countries, regions, and industries available from any source. IHS Global Insight’s immediate and insightful analysis of market conditions and key events enables innovative and successful decision making for clients around the world. IHS Global Insight employs approximately 700 employees in 13 countries.
10/09
