The Orphan Drug Development Guidebook (ODDG)
The Orphan Drug Development Guidebook is a patient focused guidebook that describes the available tools, incentives, resources and practices specific for developing traditional and innovative drugs/therapies for rare disease indications and how to best use them. It can be used by academic, non-profit organizations, small and larger (innovative) biotechs and patient-driven drug developers. The Guidebook currently has two chapters: the chapter dedicated to orphan drug development (ODDG) focused on small molecules or innovative therapies, and a chapter dedicated to drug repurposing (DRG).
More information can also be found in the papers published on the Orphan Drug Development Guidebook, the Drug Repurposing Guidebook and START checklist.
Key Drug Development Milestones
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Orphan Drug Development Cases
TRADITIONAL TECHNOLOGY TARGETING A 'SUFFICIENTLY WELL UNDERSTOOD' RARE DISEASE
Basic case of development of a traditional, well-understood pharmaceutical (such as a small molecule or a protein) targeting a 'sufficiently well understood' rare disease to be registered in EU, US and Japan
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Assumptions
- The drug is a well-understood pharmaceutical product
- There is already a non-negligible body of knowledge around the disease in the regulatory and medical community
- Patient population is sufficiently understood and non-clinical development is possible (relevant animal model exists)
- Disease is present in children and adults
- Funding the development is not a problem, but return of investment might be
HIGHLY INNOVATIVE TECHNOLOGY TARGETING A 'SUFFICIENTLY WELL UNDERSTOOD' RARE DISEASE
Case of development of a highly innovative technology (i.e. advanced therapy medicinal products) targeting a 'sufficiently well understood' rare disease to be registered in EU, US and Japan
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Assumptions
- The drug is a highly innovative technology like the advanced therapy medicinal products
- There is already a non-negligible body of knowledge around the disease in the regulatory and medical community
- Patient population is sufficiently understood and non-clinical development is possible (relevant animal model exists)
- Disease is present in children and adults
- Funding the development is not a problem, but return of investment might be
TRADITIONAL TECHNOLOGY TARGETING A 'LESS UNDERSTOOD' RARE DISEASE
Case of development of a traditional, well-understood pharmaceutical (such as a small molecule or a protein) targeting a 'less understood' rare disease [i.e., extremely rare, extremely low prevalence, neglected (i.e., complete unmet medical need), scattered diseases knowledge, and no recognized endpoints or biomarkers, pediatric only indication] to be registered in EU, US and Japan
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Assumptions
- The drug is a well-understood pharmaceutical product
- Prevalence of the disease is < 1 per-million inhabitants in all geographies
- Medical body of knowledge around the disease is negligible
- Natural history of the disease is little known; no severity or prognostic phenotypes have been identified so far
- The disease exists only in pediatric patients
- No biomarkers or clinically-relevant endpoints are available
HIGHLY INNOVATIVE TECHNOLOGY TARGETING A 'LESS UNDERSTOOD' RARE DISEASE
Case of development of a highly innovative technology (i.e. advanced therapy medicinal products) targeting a 'less understood' rare disease (i.e., extremely low prevalence, scattered diseases knowledge, and no recognized endpoints or biomarkers)
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Assumptions
- The drug is an highly innovative technology like the advanced therapy medicinal products
- Prevalence of the disease is < 1 per-million inhabitants in all geographies
- Medical body of knowledge around the disease is negligible
- Natural history of the disease is little known; no severity or prognostic phenotypes have been identified so far
- The disease exists only in pediatric patients
- No biomarkers or clinically-relevant endpoints are available
DRUG REPURPOSING CASE FOR A SUFFICIENTLY WELL UNDERSTOOD DISEASE
Repurposing of an active substance that is well-understood (a small molecule) targeting a 'sufficiently well understood' rare disease to be registered for the new indication in EU, US and Japan
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Assumptions
- The active substance is well-understood, and has already been registered for a previous indication, as part of a drug product with specific dose strength
- The active substance that will be repurposed no longer is on patent
- The developer doing the repurposing is not the marketing authorization holder
- The active substance to be repurposed will be used as a monotherapy
- The repurposed pharmaceutical product makes use of the same mechanism of action
- There is already a non-negligible body of knowledge around the disease in the regulatory and medical community
- Patient population is sufficiently understood
- The repurposed pharmaceutical product is given in the same pharmaceutical form as the drug for the original indication
- Funding the development is not a problem, but return on investment might be
Building Blocks
Based on a systematic review of websites, literature search and the expertise of the Taskforce members, we have created fact sheets describing each BB by including key information on its use, duration, pros and cons, and among other aspects the TF’s advice on the best time to use the BB. All fact sheets can be found below. You can access to the BB forms by type or by geographical scope, or by type of development (i.e. orphan drug development or drug repurposing).