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Orphan drugs in emerging markets
1. PARIOFORMA
An international pharmaceutical company commissioned Parioforma to investigate
learnings from recent reimbursement applications for Orphan Drugs in selected
Emerging Markets.
Client objectives
◦ Gain a better understanding of the treatment environment for orphan diseases in selected
Emerging Markets:
Funding mechanisms
Unmet needs
Current clinical practice and access to care
◦ Gain a better understanding of the Evidence requirements to support the reimbursement of
Orphan Drugs in these countries:
Assessment of the importance of trial parameters on the decision to reimburse
Assessment of the importance of clinical parameters on the decision to reimburse
◦ Identify informal practices different to the formal policy described in Government documents
◦ Identify reasons for the non-reimbursement of certain Orphan therapies in recent years
Programme of work
◦ Desk research was used to gain an overview into formal Government published procedures
regarding patient access to and the funding of Orphan Drugs
◦ Interviews were conducted with payers and KOLs to confirm and validate official requirements
and procedures for the reimbursement of such drugs as well as to gain a more balanced view on
official procedures vs. informal practices and the reasons behind recent unsuccessful
reimbursement applications
◦ Available evidence was assessed and a report was prepared covering the detailed findings, with
key recommendations for the client
Value
◦ The results of the project allowed the client to gain a clear understanding of the parameters that
influence access to funding of Orphan Drugs in these markets, enabling them to adapt their global
market access strategy for a better fit to emerging markets
For further information, or to discuss your market access, pricing and
reimbursement intelligence requirements, contact US now…
Parioforma Ltd – 55 Prices Gate – Exhibition Road –
London SW7 2PN – United Kingdom
www.parioforma.com 1