Preface: At ScientifyResearch, we are deeply committed to supporting a wide range of research endeavors, with a particular focus on those that have the potential to make a significant impact on the United Nation’s sustainable development goals (UN-SDGs). One area that is especially close to our heart, contributing to SDG3 “Good health and wellbeing”, is research aimed at advancing solutions for rare diseases. In this interview, we are delighted to speak with Professor Christoph Wanner and Alessio Amadasi to talk about the “Find For Rare” program from Chiesi Global Rare Diseases.
What was the impetus between starting the new Find for Rare Grant Program?
Alessio Amadasi: The inspiration for the Find for Rare Grant Program comes from our deep commitment to the rare disease community.
We invest in research with the ultimate goal of translating these efforts into tangible benefits for people living with rare disease. We strive to ensure that every advancement serves to improve lives and bring hope, particularly for those with rare and ultra-rare lysosomal storage disorders (LSDs) such as Fabry disease, alpha-mannosidosis, and cystinosis, which contribute to severe, progressive, life-long medical challenges
These hereditary genetic conditions are multifaceted, exhibiting initial symptoms that gradually evolve from childhood into adulthood, which poses challenges in both diagnosis and management. Despite significant advancements, there’s still a pressing need for further scientific investigation to improve diagnostic accuracy and enhance overall patient outcomes.
Chiesi Global Rare Diseases, alongside leading academics, recognize this need for further scientific investigation and are committed to realizing this, for example, through the Find for Rare grant program.
In this evolving environment, the independent, expert-led Research Grant Initiative ‘Find for Rare’ aims to improve patient care and management by promoting and recognizing research in these three LSD areas. Our hope is that this program will enhance our understanding and foster innovative solutions for these complex rare conditions we face today by supporting and highlighting research in these vital areas.
Tell us more about the scope of the Find for Rare Grant Program. Exactly what type of research are you looking to fund?
Alessio Amadasi: The Find for Rare Research Grant Initiative is all about supporting passionate researchers and physician scientists from health or scientific research institutions around the world (excluding North and South America).
We’re looking to fund original research projects that can advance knowledge in Fabry disease, alpha-mannosidosis, and cystinosis. There are two categories of research projects eligible for submission:
- Projects aimed at improving understanding of the factors that affect disease progression
- Studies of patient tailored clinical management, helping to create more personalized and effective care plans for those living with these conditions
Researchers can apply for grants up to €50,000 per project. Although Chiesi Global Rare Diseases is organizing and funding the Research Grant Initiative, Chiesi will not in any way influence the application, evaluation, nor grant selection process.
Could you kindly provide insight into the objectives and mission of Chiesi Global Rare Diseases?
Alessio Amadasi: Chiesi Global Rare Diseases is a business unit of the Chiesi Group, established to deliver innovative therapies and solutions for people living with rare diseases.
As a family business, Chiesi Global Rare Disease strives to create a world where it is common to have a therapy for all diseases and acts as a force for good, for society and the planet.
The unit collaborates with the rare disease community around the globe to bring a voice to underserved people in the healthcare system
Professor Wanner, please tell us more about your role with the Find for Rare Grant Program?
Professor Christoph Wanner: I’m one of the three Chairs of the Find for Rare Steering Committee, an independent scientific board, comprising of 10 leading experts in the field lysosomal storage disorders.
There are 3 Chairs – one Chair per therapeutic area (Fabry disease, alpha-mannosidosis, cystinosis) – I am the chair for Fabry disease. There are 7 Steering Committee members: 3 experts in Fabry diseases, 2 experts in alpha-mannosidosis, and 2 experts in cystinosis.
The roles and responsibilities of the Steering Committee include
- Approving the concept and project plan of the Research Grant Initiative
- Reviewing and evaluating project proposals according to the defined assessment criteria
- Discuss and identify the final recipients of the research grants
As a Steering Committee Chair, I also
- Serve as a primary point of contact between the Steering Committee, the sponsor (Chiesi), and the supporting medical communications agency
- Provide leadership and guidance to the Steering Committee members
- Collaborate with the Steering Committee board to make final decisions on project proposals to which research grants are awarded
How are the applications evaluated? Tell us more about the evaluation process that is planned.
Alessio Amadasi: Applications will be evaluated by the independent Steering Committee according to defined assessment criteria: strength of the proposed research project, innovation and novelty, relevance and impact, and potential for success.
Depending on the number and quality of applications, based on the review by the Steering Committee, the total number of grants and total amount per project awarded may vary, Researchers can apply for grants up to €50,000 per project.
Although Chiesi Global Rare Diseases are organizing and funding the Research Grant Initiative, Chiesi will not in any way influence the application, evaluation, nor grant selection process
Which elements of the projects are considered for the evaluation process?
Professor Christoph Wanner: The defined assessment criteria developed to evaluate the applications consider multiple elements of the projects, including:
- Strength of the proposed research project
- Scientific rigor of the study design
- Comprehensiveness of the proposal
- Appropriateness of the technology utilized to attain the goals
- Innovation and novelty
- Originality of the research topic
- Innovative design of the study methodology
- Potential for impactful future research
- Relevance and impact
- Relevance to the therapy area
- Clinical and research significance of potential findings
- Potential to improve patient care
- Potential for success
- Prospect of achieving proposed goals
- Timeliness for achieving proposed goals
- Relevance of the potential findings if goals are not fully achieved
- Clear explanation of budget allocation demonstrating effective resource deployment
What is the expected value that the selected projects will generate?
Professor Christoph Wanner: Our aim is to reach a better understanding of the factors affecting the rare lysosomal storage disorders and contribute to advanced patient-tailored clinical management.
We’re excited by the applications received so far and we look forward to seeing how the chosen projects could improve patient care and management.
What advice do you have for researchers interested in applying for this funding?
Professor Christoph Wanner: To download and complete the application form available on the Find For Rare website findforrare.com, keeping in mind the defined assessment criteria.
Submit your application to the Grant Office via email – FindForRare@ashfieldmedcomms.com – along with your CV and cover letter by the application deadline, 31 December 2024.
To be eligible for funding, the applicant must be a Principal Investigator (PI), research scientist or physician scientist at an accredited worldwide university, academic medical center, or a research institution.
The applicants must hold a professional degree (e.g., PhD, MD, PharmD), be responsible for the conduct of the study, and have permission from their institution to apply for a grant.
Is there an intention to replicate the program next year?
Alessio Amadasi: Early next year, we will be announcing the awardees and hosting an awards ceremony to honor the chosen projects
Further to this, our ambition is to continue this as a long-term program
Thank you Alessio and Professor Wanner for your insights, we are delighted to share these with our community of researchers focusing on rare disease research.
About our guests
- Christoph Wanner is Professor of Medicine and Head of the Division of Nephrology, University of Würzburg, Germany.
- Alessio Amadasi is Vice President, Global Medical Affairs Strategy – GRD Global Medical Affairs – Chiesi Global Rare Diseases