Max Neeman International shares expertise with orphan drug development

1. Hemophilia Case Studies

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Hemophilia Overview
Recessive X –linked bleeding disorder
Clotting factor deficiency
Affects males; females are carriers
Hemophilia A (Classical Hemophilia)
Clotting factor VIII deficiency
1 in 5,000–10,000 male births (80%)
Hemophilia B (Christmas Disease)
Clotting Factor IX deficiency
1 in about 20,000 - 34,000 male births (20%)
 1330 hemophilic children are born every year in India*
*Indian Council of Medical Research
Contact Ann Vawter to learn about our Therapeutic Specific Report - P: 919.424.3332

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Study Title: Prospective, open-label, uncontrolled, phase III study to assess the
efficacy and safety of Octafibrin for on-demand treatment of acute bleeding and to
prevent bleeding during and after surgery in subjects with congenital fibrinogen deficiency Study Design: Multinational, multi-centre, prospective, open-label, uncontrolled / Phase: Phase III Regulatory Submissions: India regulatory approved the study in 4 months. No. of Subjects: 11 from India / No. of Sites: 2 Challenges Faced: Standardizing pre-analytical sample handling for surgical studies was very critical and required repeated training and site visit on the day of surgery. Approaching all 72 Hemophilia chapters across the country for short listing potential subjects for the study required additional resources and lot of repeat follow-ups. Max Neeman Strategy: Training the clinical pathology team on sample handling (which is giving end point data of the study) was planned. Teleconferences between Central lab experts, site pathologists and Investigators were very successful in overcoming sample handling risk. Investigator involvement was also sought for approaching Hemophilia Chapters. Public assurance was created by providing necessary literature, reference to public registration of study. Client feedback/Results: Client was very delighted and happy with the India contribution to such a challenging study. Client is in the process of registering the product in India for marketing. Sponsor has initiated three more studies in same therapeutic area in India.
Hemophilia Case Study

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Study Title: Efficacy And Safety Of B-Domain Deleted Recombinant Porcine Factor VIII (OBI-1) In The Treatment Of Acquired Hemophilia A Due To Factor Viii Inhibitory Auto-Antibodies
Study Design: Multinational, multi-centre, prospective, open-label / Phase: Phase III Regulatory Submissions: India regulatory approved the study in 6 months from submission.. No. of Subjects: 5 from India / No. of Sites: 3 Challenges Faced: As the clinical exposure of Physicians on this therapeutic indication is less, training the Investigator through experienced physicians from US and Canada was organized. Being a life threatening indication with no standard treatment patient mobilization was very critical for enrollment and air ambulance facility was kept ready. Due to enormous requirement of study product as dose varies between patients and arrangement for 24X7 IP supply through central depot had to be organized in collaboration with courier agency.
. Max Neeman Strategy: Selection of highly enthusiastic Investigators was considered a key factor in success of the study. Investigators with subject matter interest and relevant publications were approached. Air ambulance facility and IP delivery arrangements within 24 Hrs. was successfully supported through experienced pharmacy depot team. Need of networking with potential physicians and clinical pathologists where disease possibility is to be first suspected was the key engagement of study team. 24X7 support of Medical Monitor and study team was provided to Investigator and study team as this is being new class of treatment and no Investigator in India has experience in using this therapy and being life-threatening indication ready inputs from medical monitor to Investigator was vital in patient management and dosing compliance. Client feedback/Results: Max Neeman’s capability to engage Investigator in this demanding study of rare and emergency indication was acknowledged and appreciated by Sponsor. While Sponsor agreed to initiate the study purely on feasibility conducted by Max Neeman as published data on reported cases for this rare indication was not available in India. Clinical expertise of Indian Investigators and professional approach to research was in line with US and Europe and evident during group interactions.
Hemophilia Case Study

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To Learn More
Contact Information: Ann Vawter
Director, Business & Marketing
Max Neeman International
117 Edinburgh South Dr., Ste 105
Cary, NC 27511
P: 919.424.3332 / F: 919.852.5574
E: ann.vawter@neeman-medical.com
www.neeman-medical.com