‘Orphan drugs’ are intended to treat rare/ultra rare diseases. They are developed to treat patients suffering from very serious diseases for which no treatment, or adequate one, is available.
Rare disease patient populations in key launch markets are defined in law as:
Orphan assets are often priced at a premium compared to non-orphan indications, with a high cost-per-patient driven by the manufacturer need to recoup the costs of R&D and incentivise development3, from small patient populations
Policy makers and regulators recognise the high unmet need and challenges to recoup investment from small patient numbers, resulting in a range of policies to incentivise orphan/rare disease therapeutic development across markets, including expedited approval and tax breaks
High prices of orphan assets, expedited regulatory approval processes, low patient numbers in clinical trials result significant payer pressure, driven by lack of long term data especially for curative treatments, limited understanding of long term costs, high up-front costs and high budget impact
This results in lack of or delays to reimbursement leading to manufacturer challenges.
Rare disease patient populations in key launch markets are defined in law as:
- US: < 200,000 people
- EU: 1 person in 2,000
- JPN: < 50,000 people (< 4 in 10,000)
Orphan assets are often priced at a premium compared to non-orphan indications, with a high cost-per-patient driven by the manufacturer need to recoup the costs of R&D and incentivise development3, from small patient populations
Policy makers and regulators recognise the high unmet need and challenges to recoup investment from small patient numbers, resulting in a range of policies to incentivise orphan/rare disease therapeutic development across markets, including expedited approval and tax breaks
High prices of orphan assets, expedited regulatory approval processes, low patient numbers in clinical trials result significant payer pressure, driven by lack of long term data especially for curative treatments, limited understanding of long term costs, high up-front costs and high budget impact
This results in lack of or delays to reimbursement leading to manufacturer challenges.
Table of Contents
1. Asset regulatory approval matrix2. Asset reimbursement overview
3. Detailed asset backgrounds
4. Price & Reimbursement overview
5. Health Technology Assessments (HTAs)
