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
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