Rare Disease Areas of Interest (Unsolicited Research Proposals)

Rare Disease Areas of Interest (Unsolicited Research Proposals) is sponsored by Pfizer Inc.. This opportunity supports mission-aligned projects and measurable outcomes.

Rare Disease Areas of Interest | Pfizer Funding Global Health Through Grants Investigator Sponsored Research Rare Disease Areas of Interest Rare Disease Areas of Interest IMPORTANT: Pfizer has implemented application windows for unsolicited requests. Please click here to view the Application and Batched Review Cycles. 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] .

Endocrine: long-acting growth hormone Comparison of the clinical effects of daily vs long-acting growth hormone Evaluation of adherence of daily and long-acting growth hormone treatment in real-world setting Comparative pathophysiology of daily vs long-acting growth hormone Novel concepts in monitoring and follow-up with 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 At this time Pfizer will not supply drug for independent research studies.

Hemophilia and Other Bleeding Disorders Research areas to be considered for funding include: Basic Science of Tissue Factor Pathway Inhibitor (TFPI), anti-TFPI Monoclonal Antibodies, and marstacimab Basic biology of TFPI including interactions among key regulatory proteins in the intrinsic and/or extrinsic clotting cascades (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: Hemophilia A or B with or without inhibitors Evaluation of the contemporary treatment burden with factor and non-factor prophylactic treatments such clinical outcomes, healthcare resource utilization/cost, adherence, quality of life, work productivity, patient reported outcomes, and safety Novel clinical or digital biomarkers to evaluate near-term and/or long-term effectiveness of prophylactic therapies Arthropathy: presence, development, clinical burden & joint damage in Hemophilia Identify and quantify barriers that patients and clinicians may face when considering switching to a new treatment Innovative strategies to enable long-term data collection from patients and/or clinicians Real world data assessing marstacimab’s use in clinical practice Marstacimab outcomes in the real-world setting: effectiveness, safety, quality of life, healthcare resource utilization, activity, work productivity, patient reported outcomes, and adherence Real world data to inform safe and effective use of marstacimab in clinical practice including but not limited to: Switching prophylaxis treatments Co-administration with factor products or bypassing agents Pre/peri/post-operative management Pre-clinical assessment of marstacimab’s hemostatic potential for other inherited/general bleeding disorders Head-to-head/comparative studies Protocols which request placebo formations Studies that overlap with ongoing clinical research activities or other ISRs Research areas to be considered for Pfizer support include but are not limited to: Characterize the impact of improved anemia/hemolysis on quality of life (QoL) (e.g. fatigue, pain, icterus) outcomes in people living with SCD utilizing validated, high-quality methods and instruments Biomarker data to understand the impact of viscosity and abnormal red blood cell rheology and function on vaso-occlusive pain episodes Impact of improved anemia/hemolysis on inflammation/vasculopathy and/or end organ damage/progression (including clinical and pre-clinical) TTR Amyloidosis: tafamidis Research areas to be considered for Pfizer support from Eligible Countries* include: Approaches for the early identification and follow up of ATTR Cardiac Amyloidosis patients.

Including or focusing on: Genetic testing and family counseling Screening algorithms (including specific populations) Imaging and diagnostic techniques Gender and race as factors in diagnosing ATTR-CM Real world efficacy and safety of tafamidis in the clinical setting for the management of ATTR Cardiac Amyloidosis.

Including or focusing on: CV-related events (including CV-related hospitalizations and urgent HF visits) CV-related hospitalization is defined as non-elective admission to an acute care setting for medical therapy that results in at least 24 hours stay (or a date change if the time of admission/discharge is not available), or a hospital stay of less than 24 hours if the discharge diagnosis and interventions indicate that the purpose of the hospital stay was for intravenous diuretic therapy for management of decompensated heart failure (Hicks et al.

Circulation. 2018 Feb 27;137(9):961-972.

; 2017 Cardiovascular and Stroke Endpoint Definitions for Clinical Trials Approaches for a better HF management of ATTR patients (treatment, monitoring, HF progression and outcome improvement) in accordance with 2025 recommendations for heart failure management ( 2025 update) and monitoring of disease progression (REF) ATTR patient comorbidities management New biomarkers for assessing disease progression Evaluation of patients who have switched to or from tafamidis Use of imaging (e.g. MRI) to assess tafamidis effectiveness and evaluate disease progression *Eligible countries: USA, Australia, Austria, Belgium, Brazil, Canada, France, Germany, Italy, Japan, Spain, Switzerland, United Kingdom Greece, the Netherlands, and Sweden.

Applications should meet the following additional requirements : Submit a full study protocol that includes a project timeline detailing key milestones and feasibility for completion. A presentation/publication plan shall be included with the study protocol. The preferred project duration should be 12 months (start to finish, including submission of an interim/final report).

The 12-month project clock will start after contracting is complete and ethics committee approval obtained. However: Investigators should aim to complete contracting within 60 days of approval where possible. If allowed in the country, investigators should submit for ethics committee approval while contracting is underway (if permitted by the country EC), to facilitate project start at the earliest date.

We are not accepting proposals focusing on: Preclinical/animal studies (including requests for pure substance) Proof of concept investigations ATTR polyneuropathy exclusively Head-to-head studies (i.e., interventional and non-interventional) including indirect treatment comparisons Any indications outside of ATTR amyloidosis Artificial Intelligence for patient screening