Orphan Business in Taiwan
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Transcript of Orphan Business in Taiwan
Developing orphan Developing orphan drugs in Asia: drugs in Asia:
Successful Successful collaborative effort collaborative effort between between biotechsbiotechs & &
policy makerspolicy makersFred LinFred Lin
Sep. 15, 2011Sep. 15, 2011
Orphan drugs become a Orphan drugs become a focusfocus
• Replace the loss of revenues as their products lose patent protection
• Reduced competition• Effective drugs can be priced at a premium• Smaller patient populations allow for smaller
trials which can be completed more rapidly at a reduced cost
• Reduces the need for complex and expensive physician marketing campaigns
• Increasing of medical care on rare disease patient as economic growth of country
Legislation of Orphan Drug Legislation of Orphan Drug ActAct
US Orphan Drug Act 1983
Singapore The medicine (Orphan Drug) exemption Order 1991
Japan Orphan Drug Legislation 1993
Australia Orphan Drug Program 1998
EU Orphan Drug Legislation 2000
Taiwan Rare Disease and Orphan Drug Act 2000
Market Overview
Rare disease & Orphan DrugRare disease & Orphan Drug• Ideally:
– Rare Disease is Disease with Low Prevalence and – Most rare diseases are results of genetic defects, and
are often described as congenital diseases– Due to the high cost of developing medical treatment
and special nutrients and a small market with so few patients, pharmaceutical industry is not willing to develop, produce or import those products. Therefore, those medical treatment and nutrients are called “Orphan Drugs”
• Reality– Different country has different definition due to country’s
specific situation– Financial support of the medication is the big concern.
Definition of Rare DiseaseDefinition of Rare Disease
Prevalence criteria Per 100,000
US < 200,000 patients / year 7.5/10,000
Canada < 200,000 patients / year 7.5/10,000
Japan < 50,000 patients / year 4.0/10,000
Australia < 2,000 patients / year 1.1/10,000
EU < 185,000 patients / year 5.0/10,000
Taiwan < 2,000 patients / year 1.0/10,000
Rare Disease and Orphan Drug ActRare Disease and Orphan Drug Act
• Commenced since 2000• Stakeholders:
– Patients– TFRD (Taiwan Foundation of Rare Disorders)– Bureau of Pharmaceutical Affairs (TFDA now)– Orphan drug advisory committee – Legislation Yuan and legislators– Public media
Funding & SubsidizationFunding & Subsidization• Government shall place the budget for
RARE Disease Treatment which is separated from National Health Insurance Budget.
• Government shall finance those international medical cooperation project of which approved by central medical committee.
• All related diagnosis, medical treatment, nutrients can be subsidized.
Pay for Rare Disease Pay for Rare Disease
National Health Insurance Budget
Rare DiseaseBudget
Usage of Orphan DrugUsage of Orphan Drug• The major indications of the medicinal
products are for the prevention, diagnosis, and treatment of rare diseases.
• Medicinal products that have been approved by other countries.
• Documents required for registration and market approval, review procedures, and other relevant issues shall be regulated by the central competent authority
• Local clinical trial may require if necessary• Permit license valid for ten years with
exclusivity
Definition of Rare Disease in Definition of Rare Disease in TaiwanTaiwan
1. Rarity2. Refractory to conventional
therapy3. Genetic (mainly) and
Metabolic diseases
Process of Orphan BusinessProcess of Orphan Business
CompanyOrphan Drug
SpecialistMedical institute
Check if rare disease listed
Patient availableClinical support & SignificanceReference approval country
NO
YES
Listing of orphan drug
Reimbursement
Product registration
ImportationName patient baseRegular importation
Reference price, Importation price with expected profitReimbursement criteria
Patent productionDossier availabilityOthers
Industrial AspectIndustrial Aspect1. Definition of orphan drug is
different from US and EU2. The treatment and subsidization
of the therapy are written in law3. Patient service and source are
the key marketing focus4. Growing market with moderate
but stable profit margin.
Market Size of Orphan Drugs Taiwan
Current related information: No. of Rare disorders: 185 items announced No. of orphan drugs: 74 items listed No. of orphan drugs reimbursed: 55 products
Reimbursement market sizeNo. of Patient reimbursed: ~ 2,700Reimbursed orphan drug: ~ USD 50-55 million
Some ExamplesSome ExamplesIndication Product
Alpha-galactosidase
A deficiency (Fabry
disease) Agalsidase-alpha (Replagal), Agalsidase-beta
(Fabrizym)
Gaucher's
disease type I 第一型高雪氏症 Imiglucerase
(Cerezym), Miglustat
(Zevasca)
Primary pulmonary hypertension 原發性肺高血壓
Bosentan, Epoprostenol, Iloprost, Treprostinil
sodium, Ambrisentan, Sildenafil
citrate
Niemann-Pick Disease Miglustat
(Zevasca)
Pompe
disease 龐貝氏症 Alpha-glucosidase
(Calzyme)
Multiple Sclerosis 多發性硬化症 Glatiramer
acetate, Interferon-Beta-1a, Natalizumab
DiGeorge
Syndrome Thymosin
alfa
1
Huntington disease 亨汀頓氏舞蹈症 Tetrabenazine
Laron
Syndrome Recombinant human insulin-like growth factor 1, rhIGF-1
Mucopolysaccharidosis
VI 黏多醣症第6型 Galsulfase
Mucopolysaccharidosis
I 黏多醣儲積症第一型 Laronidase
Nephropathic
cystinosis Cysteamine
Bitartrate
Wilson Disease威爾森氏病 Trientine
HCl, Zinc Acetate
Hunter Syndrome Idursulfase
(Elaprase)
Expenditure of Rare DiseaseExpenditure of Rare Disease
25.3
36.1 37.1
44.950.5
57.9
0
10
20
30
40
50
60
70
2005 2006 2007 2008 2009 2010
USD (M)
Attractiveness of Orphan BusinessAttractiveness of Orphan Business• Treatment on patient with rare disease is
protected by law – government has obligation to guarantee availability
of the product supplier– guarantee market growth – global pricing
• Less administration hurdle– regulatory process and lead time– hospital listing– reimbursement application
• Less competition– first mover advantage: take all existing patient at
one time and control source of new patient– Replacement is not easy to happen
Characteristics of Orphan MarketCharacteristics of Orphan Market• Patient
– Small number of patient with insufficient medical knowledge
– Suffering in seeking optimal medical support– Family involved
• Physician– No so many doctors familiar with the disease and the
treatment– No so many doctors are interest on caring the disease
and the patient
• Finance and Incentive– Treatment cost is high and never the ending– Most patients can not afford to the treatment
ChallengesChallenges• Market size / Patient number
– Theoretical market size• Prevalence rate• Incidence rate
– Accessible market size• Awareness of the disease• Late, miss or neglected diagnosis• National Screen Project
– Actual market size• Affordability (Reimbursement, social support,
insurance, …)• Diagnosis availability
Key successful factorsKey successful factors• Become the first mover • Science leads to the business• Patient is the focus• Collaboration with professional society,
charity society and patient foundation• Financial supporting program• Professional & service oriented
representative• Good connection with media and law
makers
Case 1: ZXX in NPCCase 1: ZXX in NPC• Doctor• Patients• Public media• Local distributor• Orphan drug under development• Compassion use
– 50% free, 50% charged by supplier– TFRD, Social charity funding and TNHB
• Rare disease committee and Taiwan DOH (TFDA now)
• EMEA approval• Taiwan approval immediately
Case 2: Case 2: MultikineMultikine• Orphan drug in US but not in Taiwan• OEP joins in clinical development to
exchange distribution right in the territories.
• Global phase III study makes product approval at same time as US.
• May consider business of NPB before license approval.
SummarySummary• Definition of Orphan drug in Western
countries do not exactly the same as Asia countries
• Rare disease is not a matter of medical care but social care.
• Name patient basis is mostly used business model
• Dealing unexpected situation is a NORM for doing orphan business
Q&AQ&A