Rare Disease Areas of Interest

• Comparison of clinical effects, especially body composition, cardio-metabolic, IGF-1 and dose adjustments, of daily vs long-acting growth hormone (LAGH)
• Novel approaches including Quality of Life and Patient Reported Outcomes to evaluate and treat growth disorders
• Innovative methods to evaluate and compare adherence and treatment burden of daily and LAGH
• Novel strategies to address current unmet medical needs in short stature management with the use of LAGH
• Comparative pathophysiology of daily vs long-acting growth hormone
   

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:
    1. Switching prophylaxis treatments
    2. Co-administration with factor products or bypassing agents
    3. Pre/peri/post-operative management
    4. Clinical monitoring
• Pre-clinical assessment of marstacimab’s hemostatic potential for other inherited/general bleeding disorders

Out of Scope

• Head-to-head/comparative studies
• Protocols which request placebo formations
• Pediatric investigations
• 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)

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
    • Early signs and symptoms
    • 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)
    • ATTR patient comorbidities management
    • Frailty and physical performance and relationship to treatment outcomes
• Evaluation of patients with ATTR Cardiac Amyloidosis presenting with a mixed phenotype (e.g., cardiomyopathy and polyneuropathy).
  • Including or focusing on:
    • Causal relation of signs and symptoms to ATTR
    • Evolution of mixed phenotype and its manifestations throughout the ATTR disease progression journey
    • Mixed phenotype ATTR patient comorbidities management

*Eligible countries: USA, Australia, Austria, Argentina, Belgium, Brazil, Canada, Columbia, France, Germany, Italy, Japan, Saudi Arabia, South Korea, Spain, Switzerland, Taiwan, Turkey, United Arab Emirates, United Kingdom.

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
• Pediatric investigations
• End stage disease
• Dose response studies
• Artificial Intelligence solution at the initial testing/training/validation phases/unapproved Software Medical Device