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Orphan drugs presentation

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Presentation on Orphan drugs- focused on introduction and basics with current status

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Orphan drugs presentation

  1. 1. Orphan Drugs Presenter:- Dr Atul Rajpara Post Graduate Resident, Dept. of Pharmacology, Baroda Medical College. 26-Aug-15Venue: Department of Clinical Pharmacology, KEM Hospial, Mumbai.
  2. 2. Outline  Definition  Rare Diseases  Essential Vs Orphan  Orphan Drug Act’1983  Examples of drugs and Manufacturers  Orphan drug designation process  Indian Perspective  References 26-Aug-15 2 Venue: Department of Clinical Pharmacology, KEM Hospial, Mumbai.
  3. 3. Orphan Drug Definition  “The Orphan Drug Designation program provides orphan status to drugs and biologics which are defined as those intended for the safe and effective treatment, diagnosis or prevention of rare diseases/disorders.”  The so-called 'orphan drugs' are intended to treat diseases so rare that sponsors are reluctant to develop them under usual marketing condition. 26-Aug-15 3 Venue: Department of Clinical Pharmacology, KEM Hospial, Mumbai.
  4. 4. Rare Diseases  A rare disease occurs infrequently in a population, but there is no universal definition.  3 elements to the definition as used in various countries are as follows: 1. The total number of people having the disease 2. Its prevalence 3. Non-availability of treatment for the disorder 26-Aug-15 4 Venue: Department of Clinical Pharmacology, KEM Hospial, Mumbai.
  5. 5. Country Total population affected, less than 1 per Prevalence per 10000 of population USA 200,000 7.5 JAPAN 50,000 4 SOUTH KOREA 20,000 4 AUSTRALIA 2,000 1.1 TAIWAN 10,000 1 EUROPE - 5 China 500,000 - India   26-Aug-15 5 Venue: Department of Clinical Pharmacology, KEM Hospial, Mumbai.
  6. 6.  India, like many developing countries, currently has no standard definition. Considering the large population of India, ORDI (Organisation for Rare Diseases in India) suggests a disease to be defined as rare if it affects 1 in 5,000 people or less.  The World Health Organization (W.H.O.) has suggested that a rare disease should be defined as one with frequency less than 6.5 – 10 per 10,000 people. 26-Aug-15 6 Venue: Department of Clinical Pharmacology, KEM Hospial, Mumbai.
  7. 7. Rare Disease Facts  There are more than 7000+ Rare Diseases known/reported world-wide.  Over 350 Million people world-wide are affected ~30 Million in USA, ~30 Million in EU, ~70 Million in India.  About 80% of RDs are genetic in origin many of them being monogenic.  50% of RDs are onset at birth and the rest are late onset. 26-Aug-15 7 Venue: Department of Clinical Pharmacology, KEM Hospial, Mumbai.
  8. 8.  Only ~500 approved orphan drugs exist in the market and over one hundred in clinical trials.  Majority of the diseases have no treatment and when they exist, are mostly unaffordable  Early diagnosis is a critical challenge in RD management (Avg. time: 5-7 years)  Affordability 26-Aug-15 8 Venue: Department of Clinical Pharmacology, KEM Hospial, Mumbai.
  9. 9. Rare Diseases are...  Inherited cancers,  Autoimmune disorders,  Congenital malformations,  Infectious diseases and  Others like, Haemangiomas, Hirsch sprung Disease, Gaucher disease, Cystic Fibrosis, Muscular Dystrophies and Lysosomal Storage Disorders. 26-Aug-15 9 Venue: Department of Clinical Pharmacology, KEM Hospial, Mumbai.
  10. 10. Systemic Prevalence Represents orphan drug designations through 2006 26-Aug-15 10 Venue: Department of Clinical Pharmacology, KEM Hospial, Mumbai.
  11. 11. Essential Vs Orphan drugs 26-Aug-15 11 Venue: Department of Clinical Pharmacology, KEM Hospial, Mumbai.
  12. 12. Orphan Drug Act’1983  USA became the first country to enact Orphan Drug Act on January 4th,1983.  The law is designed to facilitate the development and commercialization of drugs to treat rare diseases, The salient features of the act are as follows: i. Incentives to Pharma companies investing in Orphan Drugs R&D ii. Faster/Priority clearance of Orphan Drugs Application through FDA iii. Market exclusivity for 7 years for Pharma companies to recover costs 26-Aug-15 12 Venue: Department of Clinical Pharmacology, KEM Hospial, Mumbai.
  13. 13. Impact Of The Acts  Marketed Orphan Designated Drugs: 281  Orphan Designated Drugs in Clinical Phase: 400  Highest Number of Drugs in Phase-2 Trial: 231  US Dominates Clinical Trial Process: 350 in Pipeline (Research till Registration)  Key Market: US (Sales > US$ 40 Billion) 26-Aug-15 13 Venue: Department of Clinical Pharmacology, KEM Hospial, Mumbai.
  14. 14. Companies involved in the manufacture of orphan drugs  Pfizer  GlaxoSmithKline  Novartis  Sanofi Aventis  Johnson and Johnson  Bayer  Orphan drug specialists - Genzyme - Actelion 26-Aug-15 15 Venue: Department of Clinical Pharmacology, KEM Hospial, Mumbai.
  15. 15. Few examples of Orphan drugs and it’s market players 26-Aug-15 16 Venue: Department of Clinical Pharmacology, KEM Hospial, Mumbai.
  16. 16. Orphan Drug Designation Process  Sponsors have to first send request to the Office of Orphan Products Development (OOPD) to grant orphan designation to their drug or biological product to take advantage of financial incentives available for further product development.  And then send NDA (New Drug Application) to the Centre for Drug Evaluation and Research (CDER) or the Centre for Biologics Evaluation and Research (CBER) to market their orphan drug or biological product. 26-Aug-15 17 Venue: Department of Clinical Pharmacology, KEM Hospial, Mumbai.
  17. 17.  No IND is required SUBMISSION OF NDA Pre-Clinical Development Clinical Development CAN SUBMIT DESIGNATION REQUEST 18 26-Aug-15Venue: Department of Clinical Pharmacology, KEM Hospial, Mumbai.
  18. 18. After Designation Request Is Submitted…  Typical review cycle ~ 90 days (often less)  Will either receive: -Designation Letter OR -Deficiency Letter  Once designated, sponsor is required to submit annual reports until drug is approved 26-Aug-15 19 Venue: Department of Clinical Pharmacology, KEM Hospial, Mumbai.
  19. 19. Review of a Designation Request involves… 1. What is the disease/condition? 2. Is the disease rare (prevalence)? 3. Is there sufficient scientific rationale that demonstrates “promise” that the drug/biologic will treat, diagnose or prevent the disease/condition at issue? 26-Aug-15 20 Venue: Department of Clinical Pharmacology, KEM Hospial, Mumbai.
  20. 20. Indian Perspective 26-Aug-15 21 Venue: Department of Clinical Pharmacology, KEM Hospial, Mumbai.
  21. 21. Rare disease treatments currently available in India  In India, about 6000-8000 rare diseases, mostly genetic in nature.  In theory, close to 400 US FDA approved orphan drugs and about 80 EMA approved orphan drugs are available in India and world-wide.  However, in practice, most of them are either not accessible to most patients in India or are unaffordable. 26-Aug-15 22 Venue: Department of Clinical Pharmacology, KEM Hospial, Mumbai.
  22. 22. Financial incentives available to orphan drug developers in India  Currently, drug developers in India are receiving no formal incentives from the Government and hence they are more focused on o developing affordable drugs for more common diseases such as oral insulin, o statins for preventing or slowing the progression of cardiovascular disease, o vaccines and antibiotics for a number of preventable infectious diseases, etc.  Hence, patients with rare diseases in India have to rely on imported drugs from western countries which makes these treatments (even when available) unaffordable. 26-Aug-15 23 Venue: Department of Clinical Pharmacology, KEM Hospital, Mumbai.
  23. 23. Financial assistance to rare disease patients in India  The healthcare system in India is mostly self-funded by the patients.  This restricts the affordability threshold for drugs and diagnostic tests to around 25 thousand rupees (~400- 500 USD) per instance or about a lac rupee (~1600- 2000 USD) per year on recurring expenses for most patients.  The costs of most available orphan drugs are significantly higher than this affordability range requiring the need for financial assistance. 26-Aug-15 24 Venue: Department of Clinical Pharmacology, KEM Hospial, Mumbai.
  24. 24.  Patients look for patient foundations, other non- governmental organizations, and charitable access programs offered by certain Pharma companies to cover these costs.  Examples of such initiations are: o Birth Defects Registry of India(BDRI) o Lysosomal Storage Disorders Support Society o Metabolic Errors and Rare Diseases organization o Association for Social and Health Advancement o Haemophilia Foundation 26-Aug-15 25 Venue: Department of Clinical Pharmacology, KEM Hospial, Mumbai.
  25. 25. o Sjogren’s India o Pompe Foundation o Muscular Dystrophy Foundation o Alzheimer and Related Disorders Society Of India o Rett Syndrome Foundation o Association of Persons with Rare Eye Diseases o Amrithavarshini o ABLE (Association of Biotechnology Led Enterprises) o Mumbai Marathon 26-Aug-15 26 Venue: Department of Clinical Pharmacology, KEM Hospial, Mumbai.
  26. 26. Biggest hurdles to improving patient access to orphan drugs in India  Awareness among doctors, patients and other stakeholders of rare diseases, relevant clinical trials and orphan drugs.  Affordability and Accessibility (easy import and distribution) of orphan drugs in India.  Enactment of the Orphan Drug Act by the Government of India. Without assurance of marketing exclusivity, IP rights and other financial incentives, the orphan drugs industry is unlikely to succeed in India. 26-Aug-15 27 Venue: Department of Clinical Pharmacology, KEM Hospial, Mumbai.
  27. 27. Conclusion  The new approved orphan drugs should be easily accessible and affordable to patients with rare diseases in India.  Government of India and the other Pharma/Biotech/Diagnostic industry should work together to enact an Orphan Drugs Act (ODA) and that will create incentives for orphan drug developers.  Government needs to create a framework that is conducive to enabling the manufacturing of orphan drugs in India 26-Aug-15Venue: Department of Clinical Pharmacology, KEM Hospial, Mumbai. 28
  28. 28. References: 26-Aug-15 29 Venue: Department of Clinical Pharmacology, KEM Hospial, Mumbai.
  29. 29.  Launched on Feb. 18, 2014.  Vision: ORDI aim to empower rare disease patients and their families in India with access to national and international resources to improve their quality of life.  Mission: ORDI strive to catalyse the rapid development and delivery of affordable diagnostics and treatments for rare diseases through innovative collaborations and partnerships among stakeholders to benefit rare disease patients in India. 30
  30. 30.  Orphanet is the reference portal for information on rare diseases and orphan drugs, for all audiences.  Orphanet’s aim is to help improve the diagnosis, care and treatment of patients with rare diseases. Rare diseases are rare, but rare disease patients are numerous 31
  31. 31. Orphanet services  An inventory of rare diseases and a classification of diseases elaborated using existing published expert classifications.  An encyclopaedia of rare diseases in English and French, progressively translated into the other languages of the website.  An inventory of orphan drugs at all stages of development.  A directory of expert resources, providing information on expert clinics, medical laboratories, ongoing research projects, clinical trials, registries, networks, technological platforms and patient organisations, in the field of rare diseases, in each of the countries in Orphanet’s consortium. 32
  32. 32.  An assistance-to-diagnosis tool allowing users to search by signs and symptoms.  An encyclopaedia of recommendations and guidelines for emergency medical care and anaesthesia.  A fortnightly newsletter, OrphaNews, which gives an overview of scientific and political current affairs in the field of rare diseases and orphan drugs, in English and French.  A collection of thematic reports, the Orphanet Reports Series, focusing on overarching themes, directly downloadable from the website. 33
  33. 33. Thank You… 26-Aug-15Venue: Department of Clinical Pharmacology, KEM Hospial, Mumbai. 34

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