Developing orphan drugs in Asia: Successfulcollaborative effortbetween biotechs & policy makers Fred Lin Sep. 15, 2011
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
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
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 country’s specific situation – Financial support of the medication is the big concern.
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
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
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.
Pay for Rare Disease National Health Insurance Budget Rare Disease Budget
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
Definition of Rare Disease in Taiwan1. Rarity2. Refractory to conventional therapy3. Genetic (mainly) and Metabolic diseases
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
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.
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
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
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
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 • Affordability (Reimbursement, social support, insurance, …) • Diagnosis availability
Key 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 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: Multikine• 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.
Summary• 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