Innovation of orphan drugs to treat rare diseases is essential as the available treatment:rare disease ratio remains against the patients in need. I blogged recently about common mistakes made during late stage development of orphan drugs, so we decided to accumulate our research and provide some potential ideas to avoiding late stage failure and reduce pipeline attrition:
- Capital availability and patient enrolment
- Strategic alignment with patient advocacy groups
- Understanding biology of disease and biology of the therapeutic
- Having molecular tools or biomarkers to evaluate disease and impact of therapeutic early in the clinic
- Setting the appropriate regulatory strategy and vetting early and often with regulatory authorities.
- Adaptive clinical development plans
- Use of clinical markers
- Rigorous Phase 2a with solid PK/PD and biomarker data.
- You must asses safety first but the benefits must outweigh the risks but people should be able to have options
- Working with key experts in the chosen rare disease field
- Optimizing dosing regimens and route of administration
- Strategic partnerships
- Drug device combinations
Comments are welcome below if you want to add to these findings.
Click here for David Meeker, CEO, Genzyme's presentation on how Genzyme created the critical elements of orphan drug success.
The World Orphan Drug Congress 2012 will now be split into 3 tracks to focus on 3 distinct areas of orphan drug development – Clinical Development; Corporate & Strategic Development; Market Access, Pricing & Reimbursement