JSS Medical Research India Private Limited is a leading CRO specializing in hemophilia and bleeding disorder studies. They have expertise conducting hemophilia trials through involvement with academia and industry. JSS has successfully completed 13 US FDA GCP audits without any findings. Hemophilia is a genetic bleeding disorder where individuals are deficient in clotting factor VIII or IX. Hemophilia A is more common occurring in 1 in 5,000 births while Hemophilia B is less common at 1 in 20,000 births. In India, it is estimated there are around 20,000 people living with hemophilia. JSS has experience conducting various phase hematology studies including hemophilia A, B, von W

1. JSS Medical Research India Private Limited
Presentation on
Hemophilia

2. § Company Profile
§ Hemophilia in India
§ Therapeutic Expertise
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Agenda

3. Company Profile
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4. Organizational Overview (1/2)
JSS medical Research India Private Limited is a leading full-service Contract Research Organization Head
quartered in New Delhi, India with global offices present in Canada, Bogota and Warsaw. Some of major
organizational highlights are:
§ Leading CRO in India that has a focus on hemophilia and bleeding disorder studies, in addition to other
indications.
§ Established a team of experts specialized in carrying out hemophilia trials in response to sponsor demand.
§ The team has the required expertise via involvement of academia with industry, hemophilia trial
experience and extensive database of best Investigators for indication to develop innovative approaches
to optimize complex study design
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5. Organizational Overview (2/2)
§ 3 of the Hemophilia trials conducted by JSS has successfully completed USFDA inspection for highest
global enrollment without any findings
§ Experience of total 13 US FDA GCP Audits without any findings
§ Full service CRO with an employee strength of more than 300 globally and 200+ in India.
§ Managed over 370+ clinical trials across different therapeutic areas with clients encompassing Pharma,
Biotech, Medical Devices and Generic companies.
§ ISO 9001:2008, ISO 27001:2013 and ISO 14155:2011 Certified organization for all services.
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6. Conception Gap analysis, study design, protocol development, CRF design
Study setup Regulatory, ethics, site qualification and initiation, document
management, database, IWRS and study software development
Study execution
Project management, data management, safety review, monitoring,
site management
Data analysis
SAP Development, biostatistical analysis and consulting, sample size
calculation, Randomization and CDISC (SDTM and ADaM)
QA Program SOPs,training, Vendor Qualification, Internal & Site audit
Services
Medical Writing
Clinical study reports, integrated safety reports, abstracts, posters
and manuscripts, review articles, case reports
As a fully integrated CRO, we offer a complete range of services
from study design to results dissemination.
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7. Hemophilia in India
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8. Hemophilia
§ Hemophilia is one of several disorders of the blood clotting process that greatly prolongs
coagulation (clotting) time. Hemophilia also called as "The Royal Disease", is a genetic, life
threatening bleeding disorder.
§ The two most common forms of hemophilia are hemophilia A and hemophilia B.
Ø Hemophilia A is also called as Classical Hemophilia. It is characterized by clotting factor VIII
deficiency.
Ø Hemophilia B or Christmas disease is characterized by Clotting Factor IX deficiency.
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9. Hemophilia in India (1/2)
§ As per National Health Portal of India:
Ø Hemophilia A (clotting factor VIII deficiency), which is more common and occurs in
about 1 in 5,000 births
Ø Hemophilia B (factor IX deficiency) is less common and occurs in around 1 in about
20,000 births
§ According to Hemophilia Federation India: There are about 19858 people with Hemophilia in India
(data till 31st March 2017) of which:
Ø 80% is Hemophilia A
Ø 12% is Hemophilia B and about
Ø 3% is attributable to Von Willebrand disease (VWD)
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10. Hemophilia in India (2/2)
§ At present Indian hemophilia (Bleeding disorder) registry has close to 20,000 hemophilia patients
all over the country
§ The annual global survey of World Federation of Hemophilia had in 2016 reported that almost 50
percent of the world’s hemophilia population lives in India
§ India contributes to the maximum proportion (10%) of the global population of people with
hemophilia (PwH)
Hemophilia Federation India (HFI):
§ Hemophilia Federation India (HFI) is the only national umbrella organization in India working for
the welfare of the PwH through a network of 92 chapters spread over four regions
§ They represent India as National Member Organization at the World Federation of Hemophilia
based in Canada.
§ They also work in close collaboration with World Health Organization (WHO) and National Aids
Control Organization (NACO).
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11. 11
Patient Load: Hemophilia
Site Type of Hospital
Hemophilia A Patient
Load / year
Hemophilia B Patient
Load / year
1 Public 80 – 100 10 – 15
2 Public 70 – 80 10 – 20
3 Private 35 – 50 7 – 10
4 Private 30 – 40 5 – 10
Total 215-270 32-55

12. 12
Hemophilia Enrollment Experience

13. Standard of Care For Hemophilia
§ 2 types of Treatment Schedules:
§ On Demand Treatment – More commonly followed
§ Prophylaxis Treatment
§ Infusions of the deficient clotting factor (Human Plasma Derived & Recombinant)
§ Factor VIII in hemophilia A
§ Factor IX in hemophilia B
§ Cryoprecipitate
§ Fresh Frozen Plasma
§ Physiotherapy Exercises - to strengthen the joints, particularly elbows, knees, and ankles
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14. Hospital for Hematology Trials (1/3)
Christian Medical College, Vellore:
§ Over 8,800 staff, including over 1,528 doctors and 2,400 nurses
§ One of the largest hospitals in India.
§ Every day over 8,000 patients choose to come here for their
treatment.
§ The Department of Haematology deals with:
– benign and malignant disorders of the haematopoietic
system (blood)
– runs a busy clinical service with over 80 in-patients, including 10-15 Stem Cell Transplant patients at any
given time
– Treatment of all paediatric and adult haematological malignancies – leukemia, lymphoma, myelodysplastic
syndromes.
– Stem cell transplantation for thalassemia, lymphoid and myeloid malignancies, bone marrow failure
syndromes, certain immune deficiency disorders and metabolic disorders.
– Laboratory services related to leukemia- Flow Cytometry, Cytogenetics and Molecular Diagnosis.
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15. Hospital for Hematology Trials (2/3)
St. John's Medical College Hospital, Bengaluru:
§ It is a tertiary medical service centre with 1200 beds.
§ It offers specialty and super specialty services, including
state-of-the-art diagnostic facilities to ensure the delivery
of holistic patient care.
§ St. John's Medical College Hospital has 24 full-fledged departments
to provide specialty and super specialty services.
§ Diagnostic facilities like:
§ C.T. Scan,
§ Radio-Therapy
§ Fully-equipped laboratory facilities with Pathology, Biochemistry, Microbiology,
§ Histopathology labs and a Blood Bank are now made available in the campus.
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16. Hospital for Hematology Trials (3/3)
Sahyadri Hospital, Pune:
§ The Sahyadri group has 8 Hospitals with more than 900 beds
and 200 ICU beds.
§ Currently they have more than 2000 clinicians along with
2600 supporting staff.
§ Sahyadri Hospitals has touched the lives of more than
5 million people by providing quality care.
§ India’s biggest and most successful bone marrow transplant program – conducted in more than 520 transplants
till date.
§ One of the Tertiary Haematology referral unit in India
§ Very busy Leukemia, Hemophilia/Lymphoma, Myloma programme
§ Busy Clinical Trial Unit which is FDA audited
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17. Therapeutic Expertise
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18. Over the last 5
years,
our projects have
encompassed both
pre- and post-
approval phases.
0%
5%
10%
15%
20%
25%
30%
35%
40%
45%
50%
II III IV PMOS Device
Phase
19%
48%
11% 12%
10%
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Comprehensive Clinical Research Expertise

19. Number of Projects by Therapeutic Areas
Oncology
59
Gastroenterology
3247 37
Respiratory
14
Ophthalmology
24
Dermatology
26
Neurology
16
Hematology
21
Psychiatry
16
Nutrition
9
Infectiousdiseases
5
Vaccines
20
Endocrinology/Metabolic
Disorders
Cardiology
MedicalDevices
33
Rheumatology/
Orthopedics
16
JSS -Therapeutic Expertise
Sponsors can rely on our expertise in a wide range of therapeutic areas
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20. Sr. No. Indication Phase Sites Total Target
Hematology
1 Hemophilia A II 3 10
2 Hemophilia A III 5 25
3 Hemophilia A II 3 10
4 Hemophilia A III 08 80
5 Hemophilia A IV 6 20
6 Hemophilia A III 3 8
7 Hemophilia A I / IIA 3 9
8 Hemophilia A IV 10 70
9 Hemophilia A 3 15
10 Hemophilia A III 3 3
Sample List of Hematology Studies (1/2)
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21. Sr. No. Indication Phase Sites
Total
Target
Hematology
11 Hemophilia B III 3 10
12 Hemophilia B III 5 10
13 Hemophilia B I/II/III 2 5
14 Hemophilia B III/IV 3 10
15 VON Willbrand Disease III 12 20
16 Thrombocytopenia III 5 26
17 Congenital Afibrinogenemia III 5 8
18 Congenital Fibrinogen Deficiency II 5 5
19 Primary Immune Thrombocytopenia (ITP) III 4 20
20 Iron Deficiency Anaemia BE 15 300
Sample List of Hematology Studies (2/2)
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22. Thank You!
JSS Medical Research India Limited
Plot No. 12/2, 6th Floor,
Vatika Mindscapes Tower – B
Sarai Khwaja Metro Station,
Mathura Road, Faridabad-121003
www.jssresearch.com
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