pharmaceutical drug development

1. ORPHAN DRUG DESIGNATION AND APPROVALS
PQA MQA 106S SEMINAR-1
Department of Pharmaceutical Quality Assurance
SUPRAJA KOTAM
ROLL NO-230609019
M PHARM 1ST SEM
DEPARTMENT OF PHARMACEUTICAL
QUALITY ASSURANCE
GUIDED BY:
Dr. SUDHEER MOORKOTH
PROFESSOR & HEAD OF THE DEPARTMENT
DEPARTMENT OF PHARMACEUTICAL
QUALITY ASSURANCE

2. CONTENTS
Introduction
Orphan drug Act
Incentives
Designation
Regulations
Indian scenario
Approvals
Orphan drug market

3. Rare diseases

4. Haemophilia
A
Cll /sll
RARE
DISEASES
Cystic
fibrosis
Multiple
myeloma
REVLIMID
PNH
IMBRUVIKA
ECULIZUMAB
KOGANATE
HEMLIBRA
KALYDECO
TRIKAFTA
MAJOR DRUGS
USED IN
TREATMENT
OF RARE
DISEASES

5. ORPHAN DRUGS
Orphan drugs are medicinal products intended for diagnosis, prevention or
treatment of life-threatening or very serious diseases or disorders that are rare.
INNOVATIVE
NO SIGNIFICANT
MARKET
SUBSTANTIAL
GAINS

6. ORPHAN DRUG
ACT 1983
several countries have
through legislation like
Orphan Drug Act (ODA),
provided incentives to drug
manufactures to encourage
them to manufacture drugs
for rare diseases and
motivate drug companies to
develop orphan products.

7. IMPLEMENTATION & IMPACT

8. INCENTIVES
Federal funding allocation
Tax credit
 Exclusive marketing rights
 Fee Exemption
 Other Profits
 Synchronization

9. orphan incentive
programs
administered by the
US (FDA)
• The Orphan Drug Designation
Program
• The Humanitarian Use Device
(HUD) Designation Program
• The Orphan Products Clinical
Trials Grants Program
• The Pediatric Device Consortia
(PDC) Grant Program,
• The Orphan Products Natural
History Grants Program

10. Designation
Form 4032
Sponser
Orphan Drug
Designation Application
2 copies
Content and format for
O.D
Demonstration of
requirements
Request and
refusal
Considerations and
Requirements
The declination and
dismiss

12. ACCORDING TO FDA LAW BLOG

13. ORPHAN DRUG REGULATON
Examples : Antiviral drug ‘Remdesivir’
Inconsistent quality
Limited safety and
efficacy data
High costs
Limited access
Cases of accused of profiting from sales by
taking advantage of the law
Imatinib mesylate
Generic companies issue
Regulation of orphan drugs is crucial to ensure
that patients with rare diseases have access to
safe and effective treatments.

14. comparison of the various policies on orphan drugs worldwide
USA Japan Australia EU
Legal framework Orphan Drug Act
(1983)
Orphan Drug Regulation (1993) Orphan Drug Policy
(1998)
Regulation (CE)
N°141/2000 (2000)
Administrative authorities involved FDA / OOPD (*) MHLW/OPSR (*) (Orphan Drug
Division)
TGA (*) EMEA / COMP (*)
Estimation of the population
affected, prevalence rate (per 10,000
individuals)
20 millions
7,3
No information No information 25-30 millions
6, 6-8
Marketing exclusivity 7 years 10 years 5 years (similar to
other drugs)
10 years
Tax credit yes : 50% for
clinical studies
yes : 6% for any type of study +
limited to 10% of the company's
corporation tax
no managed by the member
states
Grants for research programs
of NIH and others
governmental funds no 'FP6' + national measures
Reconsideration of applications for
orphan designation
No yes yes
(every 12 months)
yes (every 6 years)
Technical assistance for elaboration
of the application file
yes yes no yes
Accelerated marketing procedure yes yes yes yes (via the centralised
procedure)

15. ORPHAN DRUGS IN INDIA
• INSA “To Develop a Scientific Program for Research on Rare Diseases” in 2016.
• National Policy for Rare Diseases (NPRD) ‘’National Policy for Treatment of Rare
Diseases in 2017’’.
• Review committee was constituted in 2018 - cost-sharing, disease coverage, and
patient eligibility.
• Central Drugs Standard Control Organization (CDSCO) -implemented its new Drugs
and Clinical Trials Rules 2019
• Comprehensive (NPRD) 2021- (In which included – Research & development to
manufacture orphan drugs, treatment of rare diseases)
• The new rules define orphan drugs – for the first time – as drugs to treat conditions
affecting less than 500,000 people in India.

16. CDSCO
• CDSCO issued a notice regarding waiver of clinical trial for approval of new drug in
the Indian population for rare disease and drugs.
ICMR
• ICMR are inviting projects for orphan disease research and initiation of registry for
rare disease and sponsoring/organizing workshops/conferences/training programs
on rare disease.
NGO organization
initiative
• IGIB, has conducted project funded by CSIR, named as “Genomics for Understanding
Rare Diseases India Alliance Network (Guardian),” for the purpose to bring together
and understand novel genetic variations t

17. IRD
registry
• launched on April 27, 2017. This registry covered all rare and ultra rare
diseases prevalent in India.
PEP
council
initiative
• IGIB, has conducted project funded by CSIR, named as “Genomics for
Understanding Rare Diseases India Alliance Network (Guardian),” for the
purpose to bring together and understand novel genetic variations to
achieve transitional applications.
CSIR & IGIB
initiative
• regular seminars, awareness campaigns regarding quality compliance and
orphan drugs, quality culture in good manufacturing practice (GMP)
compliance overseas marketing strategies, opportunities for orphan drugs

18.  Provide better treatment options
 Considerably cuts R&D costs
 Reduces drug development
timeline
 Lower risk of failure
Finding the new uses outside the
scope of the original medical
indications for existing drugs or
compounds.
APPROACHES FOR ADVANCING
TREATMENT

19. RECENT APPROVALS
PDUFA
Date
Orphan Drug Indication Company Status
1.20.2023 Zanubritinib CLL/SLL Beigene APPROVED
1.27.2023 Jaypirca Mantle cell lymphoma Lilly APPROVED
2.4.2023
TAKHZYRO
(lanadelumab-flyo)
Prevent attacks of
hereditary angioedema
(HAE)
Takeda APPROVED
2.11.2023 Aflibercept
Retinopathy of
Prematurity (ROP) in
preterm infants
Regeneron APPROVED
2.16.2023 Lamzede Alpha-mannosidosis Chiesi APPROVED

20. ORPHAN DRUG MARKET
The orphan drugs market data has been bifurcated into revlimid, rituxan, opdivo, keytruda,
imbruvica, soliris, jakaf, pomalyst, darzalex, and spinraza.
Orphan drugs market size was valued at USD 160.78 billion in 2021, USD 175.57 Billion in
2022 and is projected to grow from to USD 355.00 billion by 2030.
Source: Secondary Research, Primary Research, MRFR Database
and Analyst Review (Market research future)
Key Companies in the Orphan Drugs Market include :
F. Hoffmann-La Roche AG (Switzerland)
Mylan (US)
Celgene Corporation (US)
Novartis AG (Switzerland)
Biogen (US)
Takeda Pharmaceutical Company Limited (Japan)
Merck KGaA (Germany)
Eli Lilly And Company (US)
Sanofi (France)

21. CONCLUSION
 There is often a lack of clear and consistent guidance and incentives from regulatory
authorities on how to conduct orphan drug development, especially in regions or countries that
do not have specific orphan drug legislation or policies.
 This creates challenges in designing and conducting clinical trials, obtaining marketing
authorization, and ensuring access and affordability of orphan drugs.
 Developing treatments for rare diseases has many challenges. These challenges are different
and in many ways more complex than what is experienced during development of a drug
intended for a larger patient population.
 Complexity range from Scientific, operational, Financial, Ethical
 Pharmaceutical companies should not driven by commercial interests or the size of a
market
 Develop ability to strive and apply patient-centric approach
 Realising that Patients with rare disease are at the core of healthcare progress

22. REFERENCE
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innovation: new drug therapy approvals”. 2020. Accessed 8 12 2021. [Avaliable
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