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Global Orphan Assets Market Trends & Outlook Report 2020 - Orphan Assets are Often Priced at a Premium Compared to Non-orphan Indications

Research and Markets
·2-min read

Dublin, March 18, 2021 (GLOBE NEWSWIRE) -- The "Market Trends for Orphan Assets: A Global Outlook" report has been added to ResearchAndMarkets.com's offering.

'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.

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)

Key Topics Covered:

1. Asset regulatory approval matrix

2. Asset reimbursement overview

3. Detailed asset backgrounds

4. Price & Reimbursement overview

5. Health Technology Assessments (HTAs)

For more information about this report visit https://www.researchandmarkets.com/r/dqjc5f

CONTACT: CONTACT: ResearchAndMarkets.com Laura Wood, Senior Press Manager press@researchandmarkets.com For E.S.T Office Hours Call 1-917-300-0470 For U.S./CAN Toll Free Call 1-800-526-8630 For GMT Office Hours Call +353-1-416-8900