Summary
Orphan Drugs to 2008- Understanding regulation & market opportunity in Europe is designed for executives to understand the market environment for orphan drugs. The content will help all global companies to better understand the opportunities presented by recent developments in Europe orphan drug legislation. Over the last four years the legislation has provided opportunities for big and small companies, employing deliberate or add-on orphan drug development programmes. As has been shown through the success of the more mature market environment for US orphan medicines, the impact of European measures is likely to be significant and is only just beginning to materialise.
The report uses detailed regulatory, company and primary source information in order to provide a detailed guide to employing orphan drug development and marketing strategies in Europe. The report looks first at the global opportunity for orphan medicines, before outlining the European regulations and procedures in full detail. In order to assess the impact of the legislation both designated and subsequently launched orphan drugs are analysed and profiled. Detailed case studies lead to a set of key conclusions and recommendations for all pharmaceutical and biotechnology companies.
The report contains detailed analysis of major orphan drugs:
Aldurazyme, Busilvex, Carbaglu,Fabrazyme, Glivec, Litak, PhotoBarr, Onsenal, Replagal, Somavert, Tracleer, Trisenox,Ventavis and Zavesca with details of:
- Disease description
- Prevalence and incidence of disease
- Development trials
- Pivotal clinical trials
- Development and regulatory timeline
- CPMP conclusions
- CPMP decision
- Post-marketing programme
- Steps taken after granting the Marketing Authorization
- Commercial impact
TABLE OF CONTENTS
Executive Summary
Chapter 1: An Introduction to Orphan Drugs
Report purpose
Approach and methodology
Report outline
Chapter 2: Orphan Drug Activity Outside Europe
The impact of more than 20 years of US orphan drug legislation Regulations and procedures
The US
- The Orphan Drug Act
- Orphan drug designation
- Orphan drug incentives
Japan
- Definition of an orphan product
- The role of the OPSR
- The role of the MHLW
Australia
- Key incentives
- Characteristics of the orphan drug programme
Singapore
- Definition
- Legal framework for imports
- Impact and uptake
- Orphan drug designations
Orphan drug approvals
- Leading orphan drug companies
Leading orphan drugs
Chapter 3: Case Studies of Leading Orphan Drugs
Epogen (Amgen)
- Anaemia associated with end-stage renal disease
- Anaemia in Zidovudine-treated HIV-infected patients
- Sales trends
IntronA (Schering-Plough)
- AIDS-related Kaposis sarcoma
- Chronic hepatitis C in paediatric patients
- Sales trends
Remicade (Johnson & Johnson)
- Moderately to severely active Crohns disease
- Sales trends
Rituxan (Genentech)
- Non-Hodgkins B-cell lymphoma
- Sales trends
Enbrel (Amgen)
- Moderately to severe active juvenile rheumatoid arthritis
- Sales trends
Neupogen (Amgen)
- Neutropenia in cancer patients receiving bone marrow transplant
- Severe chronic neutropenia
- Peripheral blood progenitor cell collection and therapy
- Acute myeloid leukaemia receiving induction or consolidation chemotherapy
- Sales trends
Avonex (Biogen IDEC)
- Multiple sclerosis
- Sales trends
Gleevec/Glivec (Novartis)
- Chronic myeloid leukaemia
- Gastrointestinal stromal tumours
- Sales trends
Topamax (Johnson & Johnson)
- Lennox-Gastaut syndrome
- Sales trends
Taxol (BMS)
- AIDS-related Kaposis sarcoma
- Sales trends
Chapter 4: Orphan Drug Regulations in Europe
Summary
Current legislation and regulations
- History of European orphan drug legislation
- Key purposes of European orphan drug legislation
- Defining an orphan drug
- The Committee for Orphan Medicinal Products
- The designation procedure
- Key incentives
- Protocol assistance
- Community marketing authorisation
- Market exclusivity
- Other incentives
- Subsequent regulations
- Orphan drug designation applications
- Orphan drug designation procedures
Orphan drug designation fee reductions
Orphan drug designation protocol assistance
Orphan drug designation inventory of incentives
Key protagonists
- The Committee for Orphan Medicinal Products
- The European Agency for the Evaluation of Medicinal Products
- The European Commission/European Parliament
- Patient groups
- European Organisation for Rare Diseases (Eurordis)
- European Alliance of Patient and Parent Organisations for Innovation in Genetic Services (EAGS)
- Industry representatives
- Learned societies
Key benefits and limitations
- Key benefits
- Key limitations
- Cross-regional comparison
Key ambiguities
- Significant benefit
- Similar product
- Medical plausibility
- Prevalence
- Trade name
Future legislation and regulations
Chapter 5: European Orphan Drug Activity
Orphan drug designations
Key orphan drug indications
Key orphan drug companies
Key orphan drug timelines
Key orphan drug strategies
- Extended indications/formulations of non-orphan drugs
- Global orphan drug designations
- Size of target indication prevalence
- Stage of development at designation
- Special licences for pre-approval use
Chapter 6: Fabrazyme - Genzyme Europe
Overview
Clinical pharmacology
Development trials
Development and regulatory timelines
CPMP conclusions
CPMP Decision
Post-marketing programme
- Steps taken after granting the marketing authorisation
Commercial impact
Chapter 7: Replagal - Transkaryotic Therapies
Overview
Fabry disease
Incidence/prevalence of Fabry disease
Development trials
- Pivotal clinical trial
- Other clinical trials
Fabry Outcome Survey
Development and regulatory timelines
CPMP conclusions
CPMP Decision
Post-marketing programme
- Steps taken after granting the marketing authorisation
Commercial impact
Chapter 8: Trisenox - Cell Therapeutics (UK)
Overview
- Acute promyelocytic leukaemia (APL)
- Myelodysplastic syndromes
Development trials
- Pivotal clinical trials
- Other clinical trials
Development and regulatory timelines
CPMP conclusions
CPMP Decision
Post-marketing programme
- Steps taken after granting the marketing authorisation
Commercial impact
Chapter 9: Zavesca - Actelion Registration
Overview
Gaucher disease description
- Affected individuals
- Prevalence and incidence of Gaucher disease
Development trials
Development and regulatory timelines
CPMP conclusions
CPMP Decision
Post-marketing programme
- Steps taken after granting the marketing authorisation
Commercial impact
Chapter 10: Carbaglu - Orphan Europe
Overview
N-Acetylglutamate synthase (NAGS) deficiency
Development trials
Development and regulatory timelines
CPMP conclusions
CPMP Decision
- Steps taken after granting the marketing authorisation
Commercial impact
Chapter 11: Busilvex - Pierre Fabre Mi??dicament
Overview
Haematopoietic progenitor cell transplantation
Conditioning regimen
Rationale for intravenous high-dose busulfan
Development trials
- Pivotal clinical trials
- Other clinical trials
Development and regulatory timelines
CPMP conclusions
CPMP Decision
Post-marketing programme
- Steps taken after granting the marketing authorisation
Commercial impact
Chapter 12: Aldurazyme - Genzyme Europe
Overview
Mucopolysaccharidosis, type I
- Signs and symptoms
- Disease progression
Development trials
Pivotal clinical trials
Other clinical trials
Development and regulatory timelines
CPMP conclusions
CPMP Decision
Post-marketing programme
- Steps taken after granting the marketing authorisation
Commercial impact
Chapter 13: Glivec - Novartis Europharm
Overview
Indications and usage
Drivers
Barriers
Chronic myeloid leukaemia
Development trials
- Pivotal clinical trials
- Other clinical trials
Development and regulatory timelines
CPMP conclusions
CPMP Decision
Post-marketing programme
- Steps taken after granting the marketing authorisation
Commercial impact
Chapter 14: Somavert - Pharmacia Enterprises
Overview
Acromegaly description
Prevalence and incidence of acromegaly
Development trials
- Pivotal clinical trials
- Adverse events
Other clinical trials
Development and regulatory timelines
CPMP conclusions
CPMP Decision
Post-marketing programme
- Steps taken after granting the marketing authorisation
Commercial impact
Chapter 15: Tracleer - Actelion Registration
Overview
Pulmonary arterial hypertension description
Development trials
- Pivotal clinical trials
- Other clinical trials
Development and regulatory timelines
CPMP conclusions
CPMP Decision
Post-marketing programme
- Steps taken after granting the marketing authorisation
Commercial impact
Chapter 16: Four Other Key Orphan Drugs
Ventavis
Litak
Onsenal
PhotoBarr
Chapter 17: Key Conclusions and Recommendations
Key orphan drug strategies
- Elements in the design of an orphan drug strategy
Trial design and initial indication approved
Speed to market
Pricing
Profile raising
Licensing and marketing agreements
Key success stories and bottlenecks
- Glivec: a success story
- Somavert: an example of bottlenecks
- Key recommendations
