Rare Disease Areas of Interest
Qualified researchers are invited to submit research proposals, according to the guidance and instructions found on www.kddsystems.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].
Endocrine: pegvisomant
Research areas to be considered for funding include:
- Morbidity and mortality in patients with Acromegaly/GH Excess
- Novel strategies including Quality of Life and Patient Reported Outcomes to evaluate and treat acromegaly/GH Excess
- Early diagnosis and treatment of acromegaly/GH Excess; consequences of late diagnosis/treatment initiation
- Pharmacoeconomics such as but not limited to resource utilization; patient-reported outcomes; treatment patterns
- Preclinical studies (Material Transfer Agreements with funding request) would be considered in the ISR program
Gaucher Disease: taliglucerase
Research areas to be considered for Pfizer support include:
Coming Soon!
Hemophilia
Research areas to be considered for Pfizer support include:
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Basic Science of Gene Therapy for Hemophilia
- Basic science, tropism, transduction efficiency & tolerability of Adeno-associated virus
- AAV antibody Titer assessment, reduction, tolerance
- Role of immunosuppression in managing transaminitis
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Patients with MILD Hemophilia A or B
- Natural history of mild Hemophilia
- Arthropathy: presence, development, clinical burden & Joint damage in mild Hemophilia
- Quality of Life/Work analysis and cost of care in mild Hemophilia
- Development and validation of PRO (Patient Reported Outcomes) instruments in Hemophilia
Sickle Cell Disease
Research areas to be considered for Pfizer support include:
Coming Soon!
TTR Amyloidosis: tafamidis
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
- 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)
Transplant: sirolimus
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
