About Therapy development for rare diseases
At Radboudumc, we are dedicated to developing innovative therapies for rare diseases. By combining cutting-edge fundamental research with clinical expertise, and working closely with patients and partners, we enable the rapid translation of promising discoveries into effective treatments.
read moreAbout Therapy development for rare diseases
At Radboudumc, we are dedicated to developing innovative therapies for rare diseases. By combining cutting-edge fundamental research with clinical expertise, and working closely with patients and partners, we enable the rapid translation of promising discoveries into effective treatments.
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Rare diseases are largely ignored by pharmaceutical companies with respect to therapy development yet frequently pose a huge burden on the quality of life of patients and their families. The Radboudumc is excellently positioned to take a leading role in the development and application of personalized therapies for rare diseases, because of:
- Excellent preclinical research on genetics and (cancer) immunology of rare diseases, generating promising leads for therapeutic intervention.
- Its organization of care for rare disease patients and connections to care networks, ensuring access to patient groups.
- Expertise on innovative clinical trial design for rare diseases
- Availability of infrastructure (i.e. GMP facility) for and knowledge on development and production of advanced therapy medicinal products (ATMPs), which include cell- and gene therapy products.
Meet our researchers and their research
More information coming soon.
Get hands-on support and guidance
In Radboudumc the Therapy Accelerator for Rare Disease can provide hands-on support and/or advice to specific drug development projects. Through an annual call for proposals, researchers can apply for intensive, in-kind support from the multidisciplinary team. Furthermore, an advice desk is always open for questions. Please see below for more details and contact information.
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Scope
Proposals should focus on a rare disease and may involve cell or gene therapy, antisense oligonucleotides as well as small molecules (often for repurposing). Applicants will be applying for 1 year of in-kind support from our multidisciplinary team (quality/manufacturing and control, preclinical, clinical, regulatory, project management, business development). This concerns actual support (advice, documents, review, interactions, coordination etc.) and will be tailored to the specific needs of your project.
Example questions
Some examples of the support we have already been or are giving to Radboudumc projects:
- Finding suitable models for candidate testing
- Determining nonclinical and quality requirements for a first-in-human study
- Contributing to clinical trial applications, clinical trial design and dosing support (e.g. PK/PD modelling)
- Preparing for interaction (orphan drug designation, scientific advice) with regulatory agencies
- Tackling of reimbursement challenges
- Liaising with external partners for commercialisation
Timeline
What When Call open July Call closed September Decision proposal granted yes/no November Start collaboration for 1 year January How do your apply?
Fill in this application form and submit to TherapyAcceleratorRareDiseases@radboudumc.nl.
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Scope
If you have a (relatively) short question, you can request advice from us. This can either be written advice or an advisory meeting (1 hour). These meetings are free of charge.
Example questions
- How can I protect my product from competition?
- What is a named patient program?
- What are the timelines for a scientific advice?
- I am using an off-label drug which will probably be retracted from the market, what are my options?
- How do I ensure reimbursed patient access?
- Is testing my drug in a cell model enough or do I need to perform also animal studies?
- How to build a business model to get enough funding?
How
Please contact us via e-mail.
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Your project will benefit from a Product development plan with the end in mind. The checklist we provide is not exhaustive, but can really help to focus on key developmental aspects as well as project feasibility.
You’re not expected to have all the answers. It is our pleasure to help obtaining them and to help completing a product development plan using our template - please reach out.
The checklist
Please find the checklist below or download the checklist here.
Product
- What type of product are you developing? (ATMP? small chemical? AON?)
- What is the mode of action of the drug?
- In what stage of development is the drug?
Rare disease
- What is the intended therapeutic indication?
- Does the disease condition meet orphan disease criteria (debilitating nature, prevalence <1 in 2000, no authorised other treatment)?
- Can medical plausibility (i.e. potential efficacy) be substantiated?
- Is any therapy available for treatment of the disease?
Business case
- Is a target product profile available?
- Have you made a business plan?
- Can/should your invention/idea/product be protected by a patent?
- Do you have freedom to operate (not infringing existing patent or originator market exclusivity)?
- Which are approved drugs and which drugs in development are competitors?
- Has early health technology assessment been performed? Has a pricing and reimbursement strategy been defined?
Quality
- Has a quality product profile been established?
- If repurposed, can the active substance be readily obtained?
- Is GMP manufacturing in place?
- Have critical quality attributes and process parameters been identified?
- How is the manufacturing process and drug quality controlled?
Preclinical
- What preclinical evidence of efficacy and save (clinical) use has been obtained?
- Are suitable in vivo animal models available? If not, which alternatives are available and how predictive are these?
- Which preclinical evidence is yet to be obtained?
- Which preclinical information (e.g. originator, literature) can be relied on?
- How are intended clinical dose, regimen and administration route justified?
Clinical
- Which data to support clinical efficacy and safety has been/will be obtained?
- Which clinical endpoints are relevant to the disease and target population?
- Has any pharmacokinetic (pharmacodynamic) modelling been performed?
- Do external data exist? (real world data, registries, natural history)
- Is an innovative trial design required?
- Are patient organizations involved?
Regulatory
- Is GLP/GMP/GCP complied with?
- Which are the applicable guidelines and regulations?
- Has a target product profile been established?
- Is orphan drug designation considered?
- Have regulatory goals been defined? (e.g. marketing authorization)?
- Has (early) interaction with a health authority been considered?
Project management
- Up to what stage do you intend to execute development yourself?
- How is your project financed?
- Do you have a project manager?
- Do you have an overview of potential investors?
- Which external partners/networks are relevant to/working on the project?
- What are the aimed/envisaged timelines for key development steps?
Access research infrastructure and partnerships
We collaborate national and international with several initiatives and can help you get in contact with these initiatives. More information is coming soon.