Specifically, our Rare Disease business unit will consider applications for research, education and service quality improvement in the following areas:

Growth hormone disorders and acromegaly

• Projects to improve patient care or drive enhancements in the healthcare system
• Tools to improve access to care for affected patients
• Raising awareness of under-diagnosed endocrine disorders
• Increasing communication between treatment centres and referral centres
• Implementation of referral pathways
• Real world evidence data generation

Transthyretin amyloid cardiomyopathy (ATTR-CM)

• Raise awareness of amyloidosis in heart failure
• Increasing communication between treatment centres and referral centres
• Implementation of referral pathways
• Real world evidence data generation
• Increasing testing for and diagnosis of ATTR-CM

Sickle cell disease (SCD)

• Projects to increase HCP awareness and understanding of SCD and treatment options
• Quality improvement activities to standardise accident and emergency (A&E) protocols and hospital treatment pathways
• SCD epidemiological studies to better understand prevalence and clinical unmet need
• Projects aimed at understanding and improving treatment compliance in patients with SCD
• Measurement of quality of life in patients with SCD
• Projects with a focus on understanding health inequalities in SCD

Haemophilia and Duchenne’s Muscular Dystrophy (DMD)

• Medical education on disease area and genomics
• Projects with a focus on driving gene therapy service improvements
• Evaluation of prevalence and impact of AAV neutralising antibodies
• Strategies to circumvent pre-existing antibodies, with the goal of re-dosing and expanding potential eligibility for gene therapy
• Studies of the mechanisms underpinning hepatic and/or immunologic adverse effects of gene therapy and management strategies
• Evaluation of the impact of gene therapy on patient’s lifestyle and practical considerations
• Real world evidence data generation and innovative approaches to provide long-term follow-up in patients receiving gene therapy

For all independent medical grants, the grant recipient is responsible for the design, implementation, sponsorship, and conduct of the independent initiative supported by the grant, including compliance with any regulatory requirements.

Pfizer must not be involved in any aspect of project development, nor its conduct or monitoring of the program.

To discuss your potential independent medical grant application for any of the above areas, please phone 01304 616161 and ask to speak to a member of the rare disease medical affairs team.