OBJECTIVES: The number of available orphan drugs to treat rare diseases has significantly increased in the last few years due to various regulatory incentives in the EU and US. This has placed increased pressure on governments in emerging markets to improve access to orphan drugs; however, clear disparity remains across markets and the initiatives implemented. We reviewed the current market access policies and available health technology assessment (HTA) processes to understand the initiatives that impact the P&R of orphan drugs in emerging markets.

METHODS: A structured literature review across multiple databases was conducted to find publications pertaining to P&R initiatives for orphan drugs in emerging markets, based on the MSCI Emerging Markets Index 2019. Searches were limited to English language and articles published between January 2014 and May 2019.

RESULTS: 27 publications were identified reporting on the P&R in emerging markets. Overall, subsidised access is being pursued for orphan drugs more frequently in emerging markets, accompanied by greater pricing controls. Eight of the publications identified reported on China, where rare diseases are recognised as a national priority and although most orphan drugs are not covered by insurance, provincial and city initiatives provide reimbursement for specific rare diseases. In emerging European markets however, orphan drugs are mostly reimbursed by public insurance and initiatives include pre-licensing access and specialised HTA. Alternatively, access in Latin American markets is limited due to the absence of a national plan and in some cases a definition of rare diseases, such as in Peru.

CONCLUSIONS: There is significant variation in the initiatives implemented for orphan drug access across emerging markets: European markets have more established policies opposed to Latin America markets. Despite the shift towards implementing programmes to improve access of orphan drugs, lack of funding may still limit prescribing and patient access in these markets.