Module 6

First page Table of contents Previous page 16 Next page Last page

02/10/2024

Handout Decision Tree

Target Product Profile Other considerations: Orphan Drug

• An Orphan Drug is intended to treat patients with a rare (“orphan”) disease

• The orphan drug regulatory framework provides pharma companies with financial, regulatory and marketing incentives to encourage development in rare diseases, which affect smaller populations of patients and represent unmet medical need

• The statutory and regulatory framework for orphan drug designation varies considerably worldwide

• The official benefits differ across regions, but may include:

Tax incentives or Research grants

•

• Fee reductions for regulatory procedures (Free advice from EMA)

• Shorter review period for marketing application (accelerated assessment in EU or breakthrough designation / fast-track assessment US)

• Marketing exclusivity (EU & JP: 10 years; US: 7 years)

• Pricing and Reimbursement benefits (e.g. premium pricing; quicker market access via shorter HTA procedures; automatic entitlement to reimbursement for patients).

• Acknowledgement of rarity of disease which can frame HA interactions.

• Development teams to make fundamental decisions on value of orphan status

• The TPP should consider the timelines to obtain orphan drug requirements

The Organisation for Professionals in Regulatory Affairs

Lecture 1: Part 1 – Develop and Shape the Brand

Handout Decision Tree

EU Orphan Drug Designation (ODD)

Strategic Preparation •

Key elements •

Qualitative and quantitative report on the incidences and prevalence of the disease across the EU • Demonstrate there is n o ‘similarity’ to other approved products for same indication i.e. must NOT be identical active substance or active substance with same principle molecular structural features and act via same mechanism of action Demonstrate significant benefit over all existing authorised products or methods for the same indication. Definition of ‘significant benefit’: a clinically relevant advantage or a major contribution to patient care • Based on assumptions at the time of ODD • Examples: new mechanisms of action; improved efficacy; or more convenient administration route; or complimentary safety profile. • •

EU Regulation (EC) No.141/2000

Fulfil ODD criteria for the rare condition •

•

Medicine must treat, prevent, or diagnose a disease which is life-threatening or chronically debilitating, or it is unlikely that the medicine will generate sufficient returns to justify the investment needed for its development. The disease must not affect more than 5 in 10,000 people across EU. No satisfactory method of diagnosis, prevention or treatment exists, or if such a method already exists, the medicine must be of significant additional benefit to those affected by the condition

•

Examination by the EMA COMP • No fee •

•

90 days assessment from validation COMP opinion sent to EU Commission who grants ODD

•

Over authorised product (=satisfactory methods) Re-confirm prior to MAA to maintain ODD status - ‘Maintenance Report’. A much higher level of evidence required at time of MAA compared to time of ODD application, which is aligned to development stages.