Sorry, you need to enable JavaScript to visit this website.
Home About Programs & Policies Grants Investigator Sponsored Research Rare Disease Areas of Interest

Rare Disease Areas of Interest

Qualified researchers are invited to submit research proposals, according to the guidance and instructions found on www.pfizer.com/ISR. A proposal requesting Pfizer support (e.g., funding and/or drug supply) is not a guarantee of acceptance or approval of that proposal. Decisions on support for submissions are made by the applicable Pfizer Global Reviewers. A formal notification regarding the status of your application will be sent once a decision is reached. Pfizer support will only be extended upon the execution of a research agreement. For any questions, please send an email to [email protected].

  • Research areas to be considered for funding include:

    • Morbidity and mortality in Growth Hormone Excess
    • Impact and/or needs of patients with Growth Hormone Excess during the pediatric, transitional years and young adult life
    • Novel strategies including Quality of Life and Patient Reported Outcomes to evaluate and treat Growth Hormone Excess
    • Early diagnosis and treatment of Growth Hormone Excess
    • Pathophysiology of Growth Hormone Excess and/or deficiency and/or action
    • Morbidities in growth disorders
    • Novel strategies including Quality of Life and Patient Reported Outcomes to evaluate and treat growth disorders
    • Comparison of clinical effects of daily vs long-acting growth hormone
    • Comparative pathophysiology of daily vs long-acting growth hormone
    • Innovative methods to evaluate levels of adherence of daily and long-acting growth hormone treatment
    • Novel strategies to address current unmet medical needs in short stature management which could be addressed with long-acting growth hormone treatment
  • Research areas to be considered for funding include:

    • Basic and Clinical Science of Gene Therapy for Hemophilia and other Bleeding Disorders
      • Basic science, tropism, transduction efficiency & tolerability of Adeno-associated virus
      • AAV antibody seroprevalence, titer-assessment, reduction and tolerance
      • Role of immunosuppression in managing immune response and potential retreatment
      • Effect of liver-targeted rAAV vector Gene Therapy on the liver
      • Role of ultrasonography in the long-term monitoring of gene therapy
      • Impact on patient’s lifestyle (e.g. reproduction, alcohol consumption, level of physical activity)
    • Basic Science of Tissue Factor Pathway Inhibitor (TFPI) & anti-TFPI Monoclonal Antibodies
      • Basic biology of TFPI interactions
      • Cross talk among regulators (e.g. Protein S being a co-factor for both Protein C and TFPI)
      • Role of different TFPI pools in regulation of coagulation
      • Pharmacology resulting from concomitant therapies added to anti-TFPI
    • Burden of Disease: Clinical Hemophilia A or B
      • Natural history of Hemophilia and adherence to current standard of care
      • Arthropathy: presence, development, clinical burden & joint damage in Hemophilia
      • Patient experiences with hemophilia, treatment preferences and quality of care
      • Quality of Life/Work analysis and cost of care in Hemophilia
      • Novel strategies to promote long-term follow-up of patients
  • Research areas to be considered for Pfizer support include:

    • Early identification, evaluation, diagnosis, prognosis & treatment
    • Natural history
    • Epidemiology
      • Prevalence of TTR amyloidosis among at-risk populations (e.g. carpal tunnel syndrome, aortic stenosis, hypertrophic cardiomyopathy, lumbar spinal stenosis, hip & knee arthroplasty, atrial fibrillation)
      • Changing epidemiology of cardiac amyloid subtypes (hereditary vs wild-type)
    • Scintigraphy
      • Use of scintigraphy for diagnosis of early disease and/or monitoring disease progression
      • Phenotype and management of patients with Perugini Grade 1 uptake
    • Post organ transplant
      • Use of tafamidis
      • Natural course of disease
    • Study of hereditary ATTR genotypes and phenotypes
      • Non-Val30Met genotypes
      • Val122Ile, Thr60Ala, Val30Met, and others
    • Late-onset disease (onset after 50 years of age)
    • Mixed phenotypic manifestations (e.g. polyneuropathy and cardiomyopathy)
    • Development of new quality of life measures or patient report outcomes measures in ATTR amyloidosis
    • New methodology to accelerate appropriate ATTR CM patient identification including Machine learning/ Artificial Intelligence and new biomarkers
    • Use of tafamidis in the clinical setting (i.e. real world evidence)
    • Functional role of TTR in humans or non-human primates

    We are not currently accepting proposals focusing on:

    • Head to head studies
    • Any indications outside of TTR amyloidosis
    • Pediatric investigations
    • End stage disease
    • Dose response studies
    • Tafamidis Animal studies (except requests for pure substance only)
  • Research areas to be considered for funding include:

    • Preservation of renal function in kidney transplant patients
    • Reduction of post-transplant malignancy
    • Reduction of post-transplant viral infections
    • Improvement in understanding the management of side effects
    • Exploration of the use of sirolimus beyond kidney transplantation

    Drug-Supply Only on a case-by-case basis

    We are not currenlty accepting proposals focusing on:

    • Protocols which would specify drug supply for topical use
    • Active Drug substance for clinical use
    • Protocols which include product reformation or compounding
    • Protocols which request placebo formations