orphan drugs in emerging markets

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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

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Market access issues for orphan drugs in Emerging Markets

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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

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