Preface: In this interview, we speak with Prof. Dieter Haffner, Find For Rare Steering Committee member and Professor of Paediatrics and Head of the Department of Paediatric Kidney, Liver, Metabolic and Neurological Diseases at Hannover Medical School (MHH), Hannover, Germany, and Alessio Amadasi, Vice President of Medical Affairs EU and International at Chiesi Global Rare Diseases. Here, they share insights into the purpose of the Find For Rare Research Grant Initiative and the application and evaluation process for its second edition.
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Rare diseases often come with significant unmet needs and unanswered questions. Why does supporting independent research matter for Chiesi Global Rare Diseases, and how does the Find For Rare Research Grant Initiative reflect your long-term commitment to improving care for current and future generations?
Alessio Amadasi: At Chiesi Global Rare Diseases, we care deeply about the people and families living with these complex, lifelong conditions, which is why advancing research in lysosomal storage disorders remains one of our core commitments. While important progress has been made, many questions remain around earlier diagnosis, disease progression, optimal long-term management, and future therapeutic approaches. Independent scientific research is of paramount importance to address these challenges, as it brings together diverse perspectives, generates new evidence, and ultimately helps improve care for people living with rare conditions.
Find For Rare reflects our long-term commitment to supporting independent, investigator-led research. Progress in rare diseases is built over time through the collective efforts of patients, caregivers, healthcare professionals, researchers, and innovators, each contributing to a growing body of knowledge. By helping the scientific community in advancing our understanding of these rare conditions, we hope to help improve care for people living with lysosomal storage disorders today while laying the foundations for those who come next.
Looking back at the first edition of the Find For Rare research grant initiative, what were the key lessons learned, and how have they shaped this year’s edition?
Alessio Amadasi: The first edition confirmed that there is a strong interest from the scientific community for research grant initiatives that support research in lysosomal storage disorders. We received more high-quality applications than anticipated, which was very encouraging.
One key lesson was the need to adapt our evaluation process to reflect this level of interest. That’s why, for the second edition, we have expanded the independent Scientific Steering Committee with an additional expert, helping us manage the increased number of proposals while maintaining a rigorous and transparent review process.
What types of research is Find For Rare designed to support, and are there specific areas of research that are prioritised?
Prof. Dieter Haffner: Find For Rare supports independent, investigator-led research that aims to advance our understanding and improve the care and management of people living with the following rare lysosomal storage disorders:
· Fabry disease.
· Alpha-mannosidosis.
· Cystinosis.
In particular, we are looking for projects that improve our understanding of these diseases, from their underlying mechanisms through to disease progression, as well as studies that explore more patient-tailored approaches to clinical management. We also encourage collaborative research that brings together expertise across institutions and countries to address important unmet needs. Ultimately, we are looking for scientifically robust projects that address meaningful questions and have the potential to make a real difference for patients.
Who is eligible to apply for the Find For Rare research grant, and what are the key requirements?
Alessio Amadasi: Find For Rare is open to principal investigators, research scientists and physician scientists working at eligible academic or research institutions. Applicants should hold a relevant professional qualification, such as an MD, PhD or PharmD, be responsible for leading the proposed research, and have the support of their institution to apply.
As in the previous edition, Find For Rare welcomes applications from eligible researchers across Europe, Asia-Pacific, Africa and the Middle East. Applications from North, Central and Latin America are outside the scope of this programme. We encourage eligible investigators with innovative ideas in Fabry disease, alpha-mannosidosis and cystinosis to apply, and full eligibility criteria are available on the Find For Rare website.
Based on your experience from the first edition, what advice would you give researchers preparing an application this year?
Prof. Dieter Haffner: Based on what we learned from the first edition, my advice would be to invest your time in the application itself. A well-structured proposal that clearly explains the research question, the evidence underpinning it, the methodology and its potential impact will help the Steering Committee fully appreciate the scientific merit of your work.
The application process is designed to be straightforward. Applicants complete the application form, which guides them through all the key elements of the proposal, including the scientific rationale, study aims and methodology, the expected results and impact, and plans for future research. Supporting documents, including a CV and cover letter, are then submitted alongside the application form.
Why is an independent Steering Committee so important to Find For Rare, and what role does it play in supporting research excellence?
Prof. Dieter Haffner: An independent Steering Committee is essential because it gives researchers confidence that every application will be evaluated objectively and on its scientific merits. Our responsibility is to apply the same predefined assessment criteria to every proposal, ensuring a fair, transparent and rigorous review process.
As an independent panel of experts in Fabry disease, alpha-mannosidosis and cystinosis, we bring together complementary scientific and clinical expertise to identify the research with the greatest potential to advance knowledge and improve patient care. This independent peer review process helps ensure that the projects selected represent the highest standards of scientific excellence.
How does the Steering Committee evaluate applications, and what distinguishes an outstanding proposal from a good one?
Prof. Dieter Haffner: Every eligible application is assessed independently by the Steering Committee against predefined evaluation criteria to ensure a fair, consistent and objective review process. We evaluate scientific rigour, innovation, relevance to the disease area, the potential impact for patients, and the overall feasibility of the proposed research.
What distinguishes an outstanding proposal is the combination of originality and scientific credibility. We look for genuinely novel ideas, but they should be supported by a strong scientific rationale and evidence base, a robust methodology and a clear vision for how the findings could advance our understanding of Fabry disease, alpha-mannosidosis or cystinosis. We also particularly value innovative projects that have not previously received funding, as well as collaborative proposals that bring together complementary expertise.
Rare disease research cannot be advanced by a single stakeholder alone. How important is collaboration between researchers, clinicians, patient communities and industry in generating meaningful progress for people living with rare diseases?
Alessio Amadasi: Progress in rare diseases depends on collaboration. No single stakeholder can fully address the complex challenges faced by people living with rare diseases. Researchers, clinicians, patients, caregivers, advocacy groups, and industry each bring unique perspectives, experiences and expertise, and meaningful progress happens when these perspectives come together around a shared goal: improving outcomes for patients.
Initiatives such as Find For Rare demonstrate the importance of this collaborative approach. By supporting independent researchers, the initiative helps advance knowledge, strengthen scientific exchange, and foster innovation in areas where important questions remain.
This is particularly important in lysosomal storage disorders, where progress often depends on collaboration across disciplines, institutions, and countries. Every contribution, whether scientific, clinical, or rooted in lived experience, helps build a stronger foundation for future advances. Through initiatives such as Find For Rare, we hope to contribute to this collective effort and support progress that can ultimately benefit people living with these rare conditions.
Acknowledgments
We sincerely thank Prof. Dieter Haffner and Alessio Amadasi for taking the time to share their insights into the Find For Rare Research Grant Initiative. We deeply appreciate the Initiative’s contribution to supporting researchers working to make progress in rare disease research, specifically in lysosomal storage disorders.