Therapy Development for Rare Diseases

Get hands-on support and guidance​

We provide hands-on support and/or advice to specific drug development projects. Each year, via a call, we invite proposals for in-depth (one-year) support from our multidisciplinary team. Furthermore, we have an advice desk that is always open for questions. Please see below for more details and contact us with your drug development related questions.

• Call for proposals

  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.

• Advice desk

  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.

• Product development plan checklist

  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?