Orphan Business in Taiwan
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I had a presentation Biopharma Orphan Drug Congress during Sep. 14-15 in Seoul.
Transcript of Orphan Business in Taiwan
- 1. Developing orphan drugs in Asia: Successfulcollaborative effortbetween biotechs & policy makers Fred Lin Sep. 15, 2011
- 2. Orphan drugs become a focus 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
- 3. Legislation of Orphan Drug Market Overview ActUS Orphan Drug Act 1983Singapore The medicine (Orphan Drug) exemption Order 1991Japan Orphan Drug Legislation 1993Australia Orphan Drug Program 1998EU Orphan Drug Legislation 2000Taiwan Rare Disease and Orphan Drug Act 2000
- 4. Rare 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 countrys specific situation Financial support of the medication is the big concern.
- 5. Definition of Rare Disease Prevalence criteria Per 100,000US < 200,000 patients / year 7.5/10,000Canada < 200,000 patients / year 7.5/10,000Japan < 50,000 patients / year 4.0/10,000Australia < 2,000 patients / year 1.1/10,000EU < 185,000 patients / year 5.0/10,000Taiwan < 2,000 patients / year 1.0/10,000
- 6. Rare 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
- 7. Funding & 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.
- 8. Pay for Rare Disease National Health Insurance Budget Rare Disease Budget
- 9. Usage 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
- 10. Definition of Rare Disease in Taiwan1. Rarity2. Refractory to conventional therapy3. Genetic (mainly) and Metabolic diseases
- 11. Process of Orphan Business Name patient base Importation Regular importation Company Reference price, Importation price Orphan Drug Reimbursement with expected profit Reimbursement criteria Patent production Product Dossier availability registration Others Check if rare Listing of disease listed orphan drug Specialist Patient available YES Medical Clinical support & Significance institute Reference approval country NO
- 12. Industrial 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.
- 13. Market Size of Orphan Drugs TaiwanCurrent related information: No. of Rare disorders: 185 items announced No. of orphan drugs: 74 items listed No. of orphan drugs reimbursed: 55 productsReimbursement market size No. of Patient reimbursed: ~ 2,700 Reimbursed orphan drug: ~ USD 50-55 million
- 14. Some ExamplesIndication ProductAlpha-galactosidase A deficiency (Fabry disease) Agalsidase-alpha (Replagal), Agalsidase-beta (Fabrizym)Gauchers disease type I Imiglucerase (Cerezym), Miglustat (Zevasca) Bosentan, Epoprostenol, Iloprost, Treprostinil sodium,Primary pulmonary hypertension Ambrisentan, Sildenafil citrateNiemann-Pick Disease Miglustat (Zevasca)Pompe disease Alpha-glucosidase (Calzyme)Multiple Sclerosis Glatiramer acetate, Interferon-Beta-1a, NatalizumabDiGeorge Syndrome Thymosin alfa 1Huntington disease TetrabenazineLaron Syndrome Recombinant human insulin-like growth factor 1, rhIGF-1Mucopolysaccharidosis VI 6 GalsulfaseMucopolysaccharidosis I LaronidaseNephropathic cystinosis Cysteamine BitartrateWilson Disease Trientine HCl, Zinc AcetateHunter Syndrome Idursulfase (Elaprase)
- 15. Expenditure of Rare Disease70 USD (M) 57.960 50.550 44.9 36.1 37.14030 25.32010 0 2005 2006 2007 2008 2009 2010
- 16. Attractiveness 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
- 17. Characteristics 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
- 18. Challenges 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 Affordabilit