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Orphan & Rare Disease

A dedicated division for the advancement of rare disease research and specialized clinical trial design.

With approximately 7,000 rare diseases affecting 300 million people worldwide, clinical trial research in rare diseases continues to be a top priority for BioFicher and many of our clients. Currently only 5% of rare diseases have an approved treatment even though the number of FDA orphan drug and biologic approvals has grown exponentially since passage of the Orphan Drug Act.

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BioFicher has a long history supporting global research in cell and gene therapies as well as rare diseases across a wide array of therapeutic areas. Expertise in biostatistics, biomedical data science & bioinformatics and adaptive/alternative study designs has enabled BioFicher’s clients to conduct even the most challenging rare disease studies.

BioFicher has a long history supporting global research in cell and gene therapies as well as rare diseases across a wide array of therapeutic areas. Expertise in biostatistics, biomedical data science & bioinformatics and adaptive/alternative study designs has enabled BioFicher’s clients to conduct even the most challenging rare disease studies.

The challenges of delivering clinical trials within the rare disease community are unique. A CRO offering this service must have a strong track record, understanding of the environment that the trial is working within, and the skills to manage problems with efficiency, so that any unforeseen circumstances can be handled promptly when they arise. A successful trial is built upon the quality of the relationships, agile nature of delivery and knowledge of the ecosystem involved. Our global presence and dedicated staff allow for us to tailor protocols for even the rarest clinical trials. It is essential for each of our rare disease focused team members to have experience and understanding of delivering research in the rare disease community environment, so that issues can be predicted and proactively overcome.

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We strive to contribute and be at the forefront of groundbreaking research for these small, yet collectively large populations, to increase the treatments available within the rare disease space. Our dedicated division, Orphan Reach will enable us to achieve this, through several optimized approaches.

  • NORTH AMERICA
    Headquarter 4130 Parklake Ave, # 202, Raleigh, NC 27612 Tell: 984-884-6888 Email: info@bioficher.com
  • LATIN AMERICA
    Buenos Aires, Argentina Avda. Del Libertador 7270, 19th Floor C1429, Buenos Aires C1429 BMS Mexico City, Mexico Gustavo Baz #315 Edificio A-1 Piso 1 Fraccionamiento Industrial La Loma Tlalnepantla, EMEX México City, México C.P. 54060
  • EUROPE
    Amsterdam, The Netherlands Oval Tower De Entrée 99 197, 14th floor 1101 HE Amsterdam, The Netherlands+31 (0)20 3018 500 Barcelona, Spain L’Illa Diagonal Avenida Diagonal 571-M3-3rd Floor 08029 Barcelona, Spain+34 93 255 5600 Belgrade, Serbia Starine Novaka 23 11000 Belgrade, Serbia +381 11 715 8044 Bucharest, Romania Str. Tudor Arghezi No 8 UNIMED Center Building 6th floor, Room A2
  • ASIA PACIFIC
    Adelaide, Australia Level 24, 91 Westpac House King William St. Adelaide, 5000, Australia Bangkok, Thailand 23/F M Tower, All Seasons Pl 87 Wireless Road Lumpini, Phatumwan Bangkok 10330 Thailand Beijing, China Block C 5th Floor, Yintai Centre No. 2 Jiang Guo Men Wai Street Chaoyang District Beijing, China 100022 Hong Kong Level 23, One Island East 18 Westlands Road Hong Kong Tokyo, Japan Urbannet Nihonbashi 2-Chome Building 5F, 2-1-3 Nihonbashi, Chuo-ku Tokyo 103-0027 Seoul, South Korea 4F, Gangnam N Tower 129 Teheran-ro, Gangnam-gu Seoul, South Korea 06133 Singapore 21 Biopolis Road, #03-03 Nucleos Singapore 138567 Mandaluyong, Philippines BioFicher Sciences Philippines, Inc. 9th Floor, The Podium West Tower 12 ADB Avenue, Ortigas Center Mandaluyong 1555 Philippines
  • MIDDLE EAST
    Mar Roukoz, Lebanon Berytech Technological Pole 1107 2240 Mkalles, Lebanon
  • AFRICA
    Lagos State, Nigeria 183 Primerose Tower, Adeola Odeku Street 3rd Ground Floor Lagos State, Nigeria 23401 Bloemfontein, South Africa 183 Nelson Mandela Drive, Ground Floor College Court Bloemfontein, South Africa 9301 Cairo, Egypt Florida Tower El Sheikh Aly Gad El Haq St. 1229 Square, Sheraton Buildings, 8th Floor Heliopolis Cairo, Egypt
  • CARDIOVASCULAR & NEUROVASCULAR
    We work to deliver innovations that extend and improve the lives of people with cancer.
  • OPHTHAMOLOGY
    We work to deliver innovations that extend and improve the lives of people with cancer.
  • INFECTIOUS DISEASES AND VACCINES
    Infectious Disease research is a dynamic space requiring nimble and innovative solutions. Our team of infectious diseases and vaccines experts respond to the need for speed and flexibility through novel trial design and technology solutions that allow for real-time data delivery.
  • ONCOLOGY & HEMATOLOGY
    We work to deliver innovations that extend and improve the lives of people with cancer. Our work in oncology
  • PEDIATRICS
    We work to deliver innovations that extend and improve the lives of people with cancer.
  • CELL & GENE THERAPY
    Cell and gene therapies offer tremendous promise where traditional treatments fall short. These innovative therapies require charting new, riskier territory. Our multi-disciplinary cell and gene therapy teams are poised to help you bring life changing treatments to patients earlier.
  • CENTRAL NERVOUS SYSTEM
    Delivering novel CNS therapies is scientifically, medically and operationally complex. At BioFicher, we have more data and insights than ever before to drive innovation forward. With over three decades of experience across neurology, neurodegenerative diseases, psychiatry, sleep, CNS rare diseases and pain, our CNS team can help you design and deliver complex clinical trials.
  • ALLERGY & RESPIRATORY
    Your goal is to differentiate and deliver the best respiratory treatment in a competitive arena. Rely on BioFicher’s insightful and actionable analytics and technology platforms to successfully help you meet those challenges.
  • MEDTECH/MEDICAL DEVICE
    Encompasses our capabilities in digital therapeutics, medical device, and diagnostics — supporting your needs in every therapeutic indication. SCHEDULE A CONSULTATION WITH OUR MEDTECH EXPERT
  • WOMEN HEALTH
    Disease and treatment impact women differently than men. That’s why women’s health research deserves a specialized approach.

A Sample of BioFicher’s Experience in Rare Disease

Image by National Cancer Institute

Ophthalmologic Rare diseases including inherited retinal diseases

  • ABCA4 Retinopathy, Stargardt’s disease, Usher syndrome, Xlinked Juvenile Retinoschisis, Macular Telangiectasia Type 2, Retinitis Pigmentosa, Leber Congenital Amaurosis (LCA), Albinism, Best’s disease, Uveal Coloboma, Spinocerebellar Ataxia Type 7 (SCA7), Von Hippel Lindau, Behcet’s syndrome.

Blood Disorders including some blood cancers.

  • Sickle Cell disease, Aplastic Anemia, Acute Myeloid Leukemia, Multiple Myeloma, Myelodysplastic syndromes, Bone Marrow Failure syndromes

Infectious Diseases

  • Yellow Fever, Smallpox, Anthrax, Tularemia, Severe Acute Respiratory syndrome (SARS), Cytomegalovirus Infection, Neonatal Herpes (HSV)

Autoimmune

  • Ulcerative Colitis, Primary Sclerosing Cholangitis

Oncology

  • AIDS-related lymphoma (e.g., Hodgkin and non-Hodgkin, Plasmablastic lymphoma (PBL), Burkitt’s lymphoma, Primary effusion lymphoma), Kaposi sarcoma, Anal cancer, Diffuse large B-cell lymphoma (DLBCL), T-Cell Lymphoma, other various rare leukemias/lymphomas

Neurology

  • Status epilepticus, Spasticity associated w/ Cerebral Palsy

Lysosomal Storage Disorders

  • Fabry disease, Mucopolysaccharidosis II (MPS II or Hunter Syndrome), Niemann-Pick Type C, Cystinosis

Other Rare diseases/disorders

  • Bronchopulmonary Dysplasia (BPD), Limb-Girdle Muscular Dystrophies, Apnea of Prematurity

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