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ORPHAN DRUGS 
9/29/2014 1
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 
conditions. 
• Because the manufacturer wont be able to recover the costs incurred, 
& it remains in the phase 3 clinical trial , even after the approval from 
the Ethics Committee. 
9/29/2014 2
WHAT IS ‘RARE’ ? 
9/29/2014 3
oDepending on the frequency of a disease in the 
general population: 
"Rare" means "less than 1 patient per xxx people." 
oDefinition is arbitrary. Authorities in different countries or regions use 
different cut-off values: 
• WHO defines an incidence of 0.65-1/1000 as ‘Rare’ 
• US defines <2,00,000 (1/1500) patients as ‘Rare’ 
• EU defines <5/10,000 as ‘Rare’ 
• Japan defines <50,000 as ‘Rare’ 
• Australia defines <2000 as ‘Rare’ 
9/29/2014 4
ACTS & REGULATIONS 
9/29/2014 6
• ORPHAN DRUG ACT-The Orphan Drug Act (ODA) of 
January 1983, passed in the United States, is meant to 
encourage pharmaceutical companies to develop drugs for 
diseases that have a small market 
• Rare Diseases Act, 2002- establish the Office of Rare Diseases. It also 
increased funding for the development of treatments for patients with rare 
diseases. 
• European Union(EU)- Committee on Orphan Medicinal Products- broader 
definition than that of the USA, in that it also covers some tropical diseases 
• The Humanitarian Use Device (HUD) program designates a device that is 
intended to benefit patients by treating or diagnosing a disease or condition 
that affects fewer than 4,000 individuals in the United States per year. 
9/29/2014 7
9/29/2014 8
IMPACT OF THE ACTS 
• Orphan Designated Drugs in Clinical Phase: 400 
• Marketed Orphan Designated Drugs: 281 
• Highest Number of Drugs in Phase-2 Trial: 231 
• US Dominates Clinical Trial Process: 350 in Pipeline (Research till 
Registration) 
• Indication for Clinical Trials for Orphan Drug: More than 30% for 
Cancer Treatment 
• Key Market: US (Sales > US$ 40 Billion) 
9/29/2014 9
INDIAN PERSPECTIVE 
• About 6000-8000 rare diseases, mostly genetic in nature. 
• Initial estimation- over 31 million Indians are suffering from rare 
diseases 
• Taking the lower limit of global prevalence estimate, populous nations 
like India and China should have more than 70 million rare disease 
cases each. 
• With a rare disease population estimated to be around 72,611,605 and 
with little in place so far, India represents a lucrative market to 
developers and pharma looking to expand their orphan operations. 
9/29/2014 10
9/29/2014 11
ORGANISATION FOR RARE 
DISEASES INDIA (ORDI) 
• Launched on Feb. 18, 2014 
• Theoretically, 400 US FDA & 80 EMA approved orphan drugs- Available 
• Practically, most are either not accessible to most patients in India or are 
unaffordable 
• It is ORDI’s mission and hope to make these and new approved orphan 
drugs easily accessible and affordable to patients with rare diseases in India. 
• Currently, drug developers in India are receiving no formal incentives from 
the Government and hence are more focused on developing affordable 
drugs 
9/29/2014 12
COMPARISON OF ORPHAN AND 
ESSENTIAL DRUGS 
Aspect Essential drugs Orphan drugs 
Concrete policies 
in place since 
1977 worldwide 1983:USA 
2000: EU 
Primary focus Public health Individual patient 
Initiated by WHO & member states USA, EU, Japan, 
Australia 
Criteria Drug driven Disease driven 
Policies aim to 
provide 
established medicines new medicines to yet 
untreated patients 
Economics Cost effectiveness, 
sustainable & affordable access 
High prices per patient 
9/29/2014 13
PRIORITIES IN BRINGING DRUGS TO 
PATIENTS 
9/29/2014 14
PREVALENCE OF RARE 
DISEASES 
9/29/2014 15
CLINICAL TRIALS OF ORPHAN DISEASES 
9/29/2014 16
Scientific Limitations 
• Due to the rarity of the disease, a statistically powerful study with a 
high sample size may not be feasible. "Statistical significance" may be 
limited. 
• Two studies (as otherwise typical) may not be feasible, because the 
existing patients may hardly be sufficient to fill one study. 
• Prognostic factors or disease course indicators might not be known. 
9/29/2014 17
Use of Placebo 
?! 
• When there is no treatment and the safe investigational drug is the 
only (even if only theoretical) hope 
– how can we justify not giving it? 
• When the disease will inevitably create damage 
– how can we justify withdrawing concurrent treatment in order to 
have a "clean" placebo-controlled study? 
9/29/2014 18
HOW TO APPLY FOR ORPHAN 
DRUG DESIGNATION 
A sponsor shall submit two copies of a completed, dated, and signed request 
for designation that contains the following: 
1) A statement that the sponsor requests orphan-drug designation for a rare 
disease or condition 
2) The name and address of the sponsor; the name of the sponsor's primary 
contact person 
3) A description of the rare disease or condition for which the drug is being 
or will be investigated, the proposed use of the drug, and the reasons why 
such therapy is needed. 
contd.. 
9/29/2014 19
HOW TO APPLY FOR ORPHAN 
DRUG DESIGNATION- Contd.. 
4. A description of the drug, to include the identity of the active moiety, all relevant 
data from in vitro laboratory studies, preclinical efficacy studies. 
5. Where the sponsor seeks approval of an already approved orphan-drug, stating an 
explanation as to why the proposed variation in his drug may be clinically superior 
to the first drug. 
6. A summary of the regulatory status and marketing history of the drug in the 
United States and in foreign countries 
7. Documentation, with appended authoritative references 
9/29/2014 20
CHALLENGES FACED BY ORPHAN 
DRUGS 
• Difficulties in attracting public and private funding for research and 
development 
• Insufficient numbers of research participants for clinical studies 
• Lack of knowledge and training for many rare diseases 
• Lack of adequate expertise and review by authorities 
• Deficient diagnostic systems 
• High price of "orphan drugs" 
9/29/2014 21
Development of Orphan drugs is costlier because: 
The limitations of medical and scientific knowledge related to 
the pathogenesis 
A limited market 
High research and manufacturing costs (50 to 80 per cent of 
rare diseases are from genetic origin) 
 Use of high tech products genetic therapies, 
cellular therapies, recombinant proteins 
Impossibility of patenting certain ingredients 
9/29/2014 22
INCENTIVES 
The incentives include: 
• Funding towards investigation 
• Tax credit for clinical research 
• Waiver of fees for new drug application 
• Market exclusivity of “Orphan drugs” 
• Accelerated approval or fast track or priority review, may also be 
available for sponsors of orphan drugs. 
• Enhanced patent protection. 
9/29/2014 23
EXCLUSIVITY PERIOD 
COUNTRY EXCLUSIVITY TME (YEARS) 
USA 7 
JAPAN 10 
AUSTRALIA 5 
EUROPE (EU) 10 
9/29/2014 24
COMPANIES INVOLVED IN THE 
MANUFACTURE OF ORPHAN DRUGS 
 Pfizer 
 GlaxoSmithKline 
 Novartis 
 Sanofi Aventis 
 Johnson and Johnson 
 Bayer 
 Orphan drug specialists 
- Genzyme 
- Actelion 
9/29/2014 25
ORPHAN DRUGS AND MARKET PLAYERS 
DRUG COMPANY THERAPEUTIC 
INDICATION 
Zavesca (miglustat) Actelion Pharmaceuticals US, Inc Type 1 Gaucher’s disease 
Trisenox (arsenic trioxide injection) Cephalon, Inc. Acute promyelocytic leukemia 
(APL) 
Aldurazyme (Laronidase) Genzyme Ltd. Mucopolysaccharidosis I 
Glivec (Imatinib mesylate) Novartis Philadelphia chromosome positive 
chronic myeloid leukemia 
Fabrazyme (Agalsidase beta) Genzyme Ltd. Fabry disease 
Ventavis (iloprost) Actelion Pharmaceuticals US, Inc Pulmonary arterial hypertension 
(WHO Group I) in patients with 
NYHA Class III or IV symptoms. 
Litak (cladribine) Lipomed Hairy cell leukemia 
9/29/2014 26
CLASSIFICATION OF ORPHAN DRUGS 
ACCORDING TO INDICATIONS AND COUNTRIES 
DRUG INDICATIONS COUNTRY 
Acetylsalicylic acid Polycythemia Vera Europe 
Tobramycin Inhalational Powder/ 
Solution 
Cystic Fibrosis 
Pneumonia due to Pseudomonas 
Aeruginosa 
US 
Europe 
Desipramine Chlorhydrate Rett Syndrome Europe 
Indomethacin Patent Ductus Arteriosus Japan 
Histamine Dihydrochloride Acute Myeloid Leukemia 
Acute Erythroid/Promyelocytic 
Leukemia 
Europe 
USA 
9/29/2014 
Contd..
CLASSIFICATION OF ORPHAN DRUGS ACCORDING TO 
INDICATIONS AND COUNTRIES- Contd.. 
DRUG INDICATIONS COUNTRY 
Adrenomedullin (AM/ADM) Acute Lung Injury Europe 
Acadesine B- Cell leukemia 
Multiple Myeloma 
Europe 
Afamelanotide Solar Urticaria 
Familial Benign Chronic Pemphigus 
Europe 
USA 
Aclotine (Human Antithrombin III) Congenital Antithrombin III 
Deficiency 
France 
9/29/2014 28
ORPHAN DRUGS IN INDIA 
• The Hyderabad based NATCO Pharma’s novel anti-cancer drug, 
NRC-AN-019 has received “ Orphan Drug Designation” from the 
US-FDA for 3 indications- 
1. Glioma 
2. Pancreatic Cancer 
3. Chronic Myeloid Leukemia 
contd… 
9/29/2014 29
ORPHAN DRUGS IN INDIA- Contd.. 
• Troikaa Pharmaceuticals, Ahmedabad manufactures following 
orphan preparations: 
1. Tachyban ( Adenosine injection) 
2. Hemaprot (Aprotinin injection) 
3. Neopam ( Pralidoxime injection) 
4. Narcotam ( Naloxone hydrochloride injection) 
5. Cyan SOS (Cyanide antidote kit) 
9/29/2014 30
THE ORPHAN DRUG PIPELINE 
ONCOLOGY- BRAND NAME GENERIC NAME 
Istodex Romidepsin 
Yondelis Trabectedin 
Onrigin Laromustine 
CENTRAL NERVOUS SYSTEM-BRAND 
NAME GENERIC NAME 
Zenas Amiframpidine 
ITI 111 Midazolam 
ANTI-INFECTIVES 
BRAND NAME GENERIC NAME 
Cayston Aztreonam Lysine 
ABthrax Raxibacumab 
9/29/2014 31
RECENT DEVELOPMENTS IN REGULATING ‘ORPHAN 
DRUG’ APPROVAL 
• The US FDA & EMA- announced a more streamlined process to help 
regulators identify and share information in a better way, throughout 
the development process of orphan drug and biologic products. 
• If an orphan product was granted designation on the exact same day in 
both the US and EU, the sponsors must submit separate reports to their 
respective regulatory agency. 
• Use of one annual report will also benefit sponsors by eliminating the 
duplication of efforts and by simplifying the process 
9/29/2014 32
CONCLUSION 
• Orphan drugs may help pharma companies to reduce the impact of 
revenue loss caused by patent expiries of blockbuster drugs. 
• The new business model of orphan drugs offer an integrated healthcare 
solution that enables pharma companies to develop newer areas of- 
Therapeutics 
Diagnosis 
Treatment 
Monitoring and 
Patient Support 
9/29/2014 33

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ORPHAN DRUGS- ACTS, PAST AND CURRENT SCENARIO, WITH UPCOMING DRUGS

  • 2. 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 conditions. • Because the manufacturer wont be able to recover the costs incurred, & it remains in the phase 3 clinical trial , even after the approval from the Ethics Committee. 9/29/2014 2
  • 3. WHAT IS ‘RARE’ ? 9/29/2014 3
  • 4. oDepending on the frequency of a disease in the general population: "Rare" means "less than 1 patient per xxx people." oDefinition is arbitrary. Authorities in different countries or regions use different cut-off values: • WHO defines an incidence of 0.65-1/1000 as ‘Rare’ • US defines <2,00,000 (1/1500) patients as ‘Rare’ • EU defines <5/10,000 as ‘Rare’ • Japan defines <50,000 as ‘Rare’ • Australia defines <2000 as ‘Rare’ 9/29/2014 4
  • 5. ACTS & REGULATIONS 9/29/2014 6
  • 6. • ORPHAN DRUG ACT-The Orphan Drug Act (ODA) of January 1983, passed in the United States, is meant to encourage pharmaceutical companies to develop drugs for diseases that have a small market • Rare Diseases Act, 2002- establish the Office of Rare Diseases. It also increased funding for the development of treatments for patients with rare diseases. • European Union(EU)- Committee on Orphan Medicinal Products- broader definition than that of the USA, in that it also covers some tropical diseases • The Humanitarian Use Device (HUD) program designates a device that is intended to benefit patients by treating or diagnosing a disease or condition that affects fewer than 4,000 individuals in the United States per year. 9/29/2014 7
  • 8. IMPACT OF THE ACTS • Orphan Designated Drugs in Clinical Phase: 400 • Marketed Orphan Designated Drugs: 281 • Highest Number of Drugs in Phase-2 Trial: 231 • US Dominates Clinical Trial Process: 350 in Pipeline (Research till Registration) • Indication for Clinical Trials for Orphan Drug: More than 30% for Cancer Treatment • Key Market: US (Sales > US$ 40 Billion) 9/29/2014 9
  • 9. INDIAN PERSPECTIVE • About 6000-8000 rare diseases, mostly genetic in nature. • Initial estimation- over 31 million Indians are suffering from rare diseases • Taking the lower limit of global prevalence estimate, populous nations like India and China should have more than 70 million rare disease cases each. • With a rare disease population estimated to be around 72,611,605 and with little in place so far, India represents a lucrative market to developers and pharma looking to expand their orphan operations. 9/29/2014 10
  • 11. ORGANISATION FOR RARE DISEASES INDIA (ORDI) • Launched on Feb. 18, 2014 • Theoretically, 400 US FDA & 80 EMA approved orphan drugs- Available • Practically, most are either not accessible to most patients in India or are unaffordable • It is ORDI’s mission and hope to make these and new approved orphan drugs easily accessible and affordable to patients with rare diseases in India. • Currently, drug developers in India are receiving no formal incentives from the Government and hence are more focused on developing affordable drugs 9/29/2014 12
  • 12. COMPARISON OF ORPHAN AND ESSENTIAL DRUGS Aspect Essential drugs Orphan drugs Concrete policies in place since 1977 worldwide 1983:USA 2000: EU Primary focus Public health Individual patient Initiated by WHO & member states USA, EU, Japan, Australia Criteria Drug driven Disease driven Policies aim to provide established medicines new medicines to yet untreated patients Economics Cost effectiveness, sustainable & affordable access High prices per patient 9/29/2014 13
  • 13. PRIORITIES IN BRINGING DRUGS TO PATIENTS 9/29/2014 14
  • 14. PREVALENCE OF RARE DISEASES 9/29/2014 15
  • 15. CLINICAL TRIALS OF ORPHAN DISEASES 9/29/2014 16
  • 16. Scientific Limitations • Due to the rarity of the disease, a statistically powerful study with a high sample size may not be feasible. "Statistical significance" may be limited. • Two studies (as otherwise typical) may not be feasible, because the existing patients may hardly be sufficient to fill one study. • Prognostic factors or disease course indicators might not be known. 9/29/2014 17
  • 17. Use of Placebo ?! • When there is no treatment and the safe investigational drug is the only (even if only theoretical) hope – how can we justify not giving it? • When the disease will inevitably create damage – how can we justify withdrawing concurrent treatment in order to have a "clean" placebo-controlled study? 9/29/2014 18
  • 18. HOW TO APPLY FOR ORPHAN DRUG DESIGNATION A sponsor shall submit two copies of a completed, dated, and signed request for designation that contains the following: 1) A statement that the sponsor requests orphan-drug designation for a rare disease or condition 2) The name and address of the sponsor; the name of the sponsor's primary contact person 3) A description of the rare disease or condition for which the drug is being or will be investigated, the proposed use of the drug, and the reasons why such therapy is needed. contd.. 9/29/2014 19
  • 19. HOW TO APPLY FOR ORPHAN DRUG DESIGNATION- Contd.. 4. A description of the drug, to include the identity of the active moiety, all relevant data from in vitro laboratory studies, preclinical efficacy studies. 5. Where the sponsor seeks approval of an already approved orphan-drug, stating an explanation as to why the proposed variation in his drug may be clinically superior to the first drug. 6. A summary of the regulatory status and marketing history of the drug in the United States and in foreign countries 7. Documentation, with appended authoritative references 9/29/2014 20
  • 20. CHALLENGES FACED BY ORPHAN DRUGS • Difficulties in attracting public and private funding for research and development • Insufficient numbers of research participants for clinical studies • Lack of knowledge and training for many rare diseases • Lack of adequate expertise and review by authorities • Deficient diagnostic systems • High price of "orphan drugs" 9/29/2014 21
  • 21. Development of Orphan drugs is costlier because: The limitations of medical and scientific knowledge related to the pathogenesis A limited market High research and manufacturing costs (50 to 80 per cent of rare diseases are from genetic origin)  Use of high tech products genetic therapies, cellular therapies, recombinant proteins Impossibility of patenting certain ingredients 9/29/2014 22
  • 22. INCENTIVES The incentives include: • Funding towards investigation • Tax credit for clinical research • Waiver of fees for new drug application • Market exclusivity of “Orphan drugs” • Accelerated approval or fast track or priority review, may also be available for sponsors of orphan drugs. • Enhanced patent protection. 9/29/2014 23
  • 23. EXCLUSIVITY PERIOD COUNTRY EXCLUSIVITY TME (YEARS) USA 7 JAPAN 10 AUSTRALIA 5 EUROPE (EU) 10 9/29/2014 24
  • 24. COMPANIES INVOLVED IN THE MANUFACTURE OF ORPHAN DRUGS  Pfizer  GlaxoSmithKline  Novartis  Sanofi Aventis  Johnson and Johnson  Bayer  Orphan drug specialists - Genzyme - Actelion 9/29/2014 25
  • 25. ORPHAN DRUGS AND MARKET PLAYERS DRUG COMPANY THERAPEUTIC INDICATION Zavesca (miglustat) Actelion Pharmaceuticals US, Inc Type 1 Gaucher’s disease Trisenox (arsenic trioxide injection) Cephalon, Inc. Acute promyelocytic leukemia (APL) Aldurazyme (Laronidase) Genzyme Ltd. Mucopolysaccharidosis I Glivec (Imatinib mesylate) Novartis Philadelphia chromosome positive chronic myeloid leukemia Fabrazyme (Agalsidase beta) Genzyme Ltd. Fabry disease Ventavis (iloprost) Actelion Pharmaceuticals US, Inc Pulmonary arterial hypertension (WHO Group I) in patients with NYHA Class III or IV symptoms. Litak (cladribine) Lipomed Hairy cell leukemia 9/29/2014 26
  • 26. CLASSIFICATION OF ORPHAN DRUGS ACCORDING TO INDICATIONS AND COUNTRIES DRUG INDICATIONS COUNTRY Acetylsalicylic acid Polycythemia Vera Europe Tobramycin Inhalational Powder/ Solution Cystic Fibrosis Pneumonia due to Pseudomonas Aeruginosa US Europe Desipramine Chlorhydrate Rett Syndrome Europe Indomethacin Patent Ductus Arteriosus Japan Histamine Dihydrochloride Acute Myeloid Leukemia Acute Erythroid/Promyelocytic Leukemia Europe USA 9/29/2014 Contd..
  • 27. CLASSIFICATION OF ORPHAN DRUGS ACCORDING TO INDICATIONS AND COUNTRIES- Contd.. DRUG INDICATIONS COUNTRY Adrenomedullin (AM/ADM) Acute Lung Injury Europe Acadesine B- Cell leukemia Multiple Myeloma Europe Afamelanotide Solar Urticaria Familial Benign Chronic Pemphigus Europe USA Aclotine (Human Antithrombin III) Congenital Antithrombin III Deficiency France 9/29/2014 28
  • 28. ORPHAN DRUGS IN INDIA • The Hyderabad based NATCO Pharma’s novel anti-cancer drug, NRC-AN-019 has received “ Orphan Drug Designation” from the US-FDA for 3 indications- 1. Glioma 2. Pancreatic Cancer 3. Chronic Myeloid Leukemia contd… 9/29/2014 29
  • 29. ORPHAN DRUGS IN INDIA- Contd.. • Troikaa Pharmaceuticals, Ahmedabad manufactures following orphan preparations: 1. Tachyban ( Adenosine injection) 2. Hemaprot (Aprotinin injection) 3. Neopam ( Pralidoxime injection) 4. Narcotam ( Naloxone hydrochloride injection) 5. Cyan SOS (Cyanide antidote kit) 9/29/2014 30
  • 30. THE ORPHAN DRUG PIPELINE ONCOLOGY- BRAND NAME GENERIC NAME Istodex Romidepsin Yondelis Trabectedin Onrigin Laromustine CENTRAL NERVOUS SYSTEM-BRAND NAME GENERIC NAME Zenas Amiframpidine ITI 111 Midazolam ANTI-INFECTIVES BRAND NAME GENERIC NAME Cayston Aztreonam Lysine ABthrax Raxibacumab 9/29/2014 31
  • 31. RECENT DEVELOPMENTS IN REGULATING ‘ORPHAN DRUG’ APPROVAL • The US FDA & EMA- announced a more streamlined process to help regulators identify and share information in a better way, throughout the development process of orphan drug and biologic products. • If an orphan product was granted designation on the exact same day in both the US and EU, the sponsors must submit separate reports to their respective regulatory agency. • Use of one annual report will also benefit sponsors by eliminating the duplication of efforts and by simplifying the process 9/29/2014 32
  • 32. CONCLUSION • Orphan drugs may help pharma companies to reduce the impact of revenue loss caused by patent expiries of blockbuster drugs. • The new business model of orphan drugs offer an integrated healthcare solution that enables pharma companies to develop newer areas of- Therapeutics Diagnosis Treatment Monitoring and Patient Support 9/29/2014 33

Notes de l'éditeur

  1. 6-8% of world’s population- Orphan Diseases
  2. Before Congress enacted the ODA in 1983 only 38 drugs were approved in the USA specifically to treat orphan diseases.[10] In the USA, from January 1983 to June 2004, a total of 1,129 different orphan drug designations have been granted by the Office of Orphan Products Development (OOPD). From the passage of the ODA in 1983 until May 2010, the FDA approved 353 orphan drugs and granted orphan designations to 2,116 compounds.
  3. EC- EUROPEAN COMMISSION OOPD- Office of Orphan Products Development MHLW (Ministry of Health, Labor and Welfare) TGA- Therapeutic Goods Authority (Regulations) EMEA-European Medicines (EMERGENCY) Agency COMP- Committee on Orphan Medicinal Products
  4. Majority of these drugs are being developed in US followed by Europe. US dominates the development of orphan drugs with more than 300 orphan designated drugs being under clinical trial process.
  5. Over 350 Million people world-wide are affected 1 in 20 Indians are affected About 80% of RDs are genetic in origin 50% of RDs have their onset at birth and remaining half are of late onset
  6. Rare Diseases India (RDI) is a volunteer-driven online entity, established in 2009, relying on contribution of knowledge and time by volunteers for the cause of rare diseases and disorders.
  7. patients with rare diseases in India have to rely on imported drugs from western countries which makes these treatments (even when available) unaffordable. ORDI aims to work between the Government of India and the Pharma/Biotech/Diagnostic industry to enact an Orphan Drugs Act (ODA) that will create incentives for orphan drug developers. We also need the Government to create a framework that is conducive to enabling the manufacturing of orphan drugs in India. Rare dis. HELPLINE NO.-+91 8892 555 000
  8. Rare Diseases India (RDI) is a volunteer-driven online entity, established in 2009, relying on contribution of knowledge and time by volunteers for the cause of rare diseases and disorders.
  9. Drug-driven refers to more emphasis on the drug compound for decision-making (e.g., cost-effectiveness, evidence base). "Disease-driven" refers to more emphasis on the characteristics of the disease-making process. The arrows indicate a future trend based on recent developments
  10. Which makes it all the more difficult to conduct a clinical trial for such an orphan disease
  11. Investigational product is just an add-on to baseline care Cross-over design where everyone gets the new drug at least once,should be followed
  12. Health professionals are often deficient in appropriate training and awareness to be able to diagnose and adequately treat these diseases. For many diseases, no diagnostic methods exist, or diagnostic facilities are unavailable. In these cases, diagnosis may be problematic-validity, coding, reproducibility. Prices of orphan drugs per treatment episode can be very high. For example, the cost of treatment with enzyme replacement therapies may reach more than US$150,000 per treatment year.
  13. Patents are granted by the patent and trademark office anywhere along the development lifeline of a drug and can encompass a wide range of claims. Exclusivity is exclusive marketing rights granted by the FDA upon approval of a drug and can run concurrently with a patent or not. Exclusivity is a statutory provision and is granted to an NDA applicant if statutory requirements are met.  Exclusivity was designed to promote a balance between new drug innovation and generic drug competition.
  14. Fabrazyme -Marketing authorization with orphan designation  - Japan , Australia. Not in Europe.
  15. Rett Syndrome-Cerebroatrophic hyperammonemia
  16. AM was initially identified as a vasodilator, some have cited this as the most potent endogenous vasodilatory peptide found in the body.
  17. NRC-AN-019= a tyrosine kinase inhibitor, in imatinib-resistant chronic myeloid leukemia
  18. APROTININ- small protein bovine pancreatic trypsin inhibitor (BPTI), an antifibrinolytic molecule that inhibits trypsin and related proteolytic enzymes, USED IN LUNG INJURIES.
  19. Following similar footsteps as USA & EU, India should also encourage its domestic pharmaceutical industry to get engaged in research to discover drugs for rare diseases by putting an "ODA" in place, extending financial support, tax exemptions and regulatory concessions like smaller and shorter clinical trials, without further delay
  20. Following similar footsteps as USA & EU, India should also encourage its domestic pharmaceutical industry to get engaged in research to discover drugs for rare diseases by putting an "ODA" in place, extending financial support, tax exemptions and regulatory concessions like smaller and shorter clinical trials, without further delay