A Proposal to Improve the Supply of Orphan Drugs

The US Orphan Drug Act: Rare disease research stimulator or commercial opportunity?

“…In Japan, pharmaceutical manufacturers are mandated to pay a one-percent sales tax on orphan drugs with annual profits exceeding 100 million yen until government subsidies received by manufacturers have been repaid [2]. This clause does not appear to have negatively affected orphan drug development in Japan since the Japanese Orphan Drug Act has resulted in the approval of nearly 100 orphan products in the 12 years following its 1993 enactment [23,33,34]. Considering that both Japan and the EU offer 10 year market exclusivity, the Japanese Orphan Drug Act appears to be more successful in stimulating orphan drug R&D, as deduced by drug approvals, than its European counterpart which has threatened to revoke orphan drug status for profitable drugs.…”

Reimbursed Orphan Medicines in Bulgaria and the Share of Biotechnology-Derived Products

“…We have applied a three-step methodology in order to assess the availability of orphan drugs in Bulgaria: (1) Review of the list of orphan medicines in europe (with european market authorisation and with or without prior orphan designation in Europe) for orphan medicines clarification (16); (2) Review of Bulgarian PDL and identification of orphan drugs included (19); (3) Crossing the medicines identified in step (2) with the list of orphan medicines in europe (with european market authorisation and with or without prior orphan designation in europe) to evaluate the level of orphan drug availability in Bulgaria. the methods of manufacturing for each reimbursed orphan medicine were checked in the Summary of Product characteristics and european public assessment reports (33).…”

“…the Bulgarian Positive Drug list (19) consists of four annexes which are divided based on the sources of funding. For the purpose of this publication we studied the orphan medicines, which are included in Annexes 3 and 4 and are fully reimbursed by the state budget.…”

“…there are chapters dealing with rare diseases in the health Act and with orphan medicines in the law on Medicinal Products but a lot of other elements are missing as are the cooperation with marketing authorisation holders and full harmonisation with the european regulatory policy. thus patients suffering from rare diseases are also underserved in Bulgaria (19,25,27). the latter conclusion is supported by the limited availability of the orphan medicines in the Bulgarian PDl.…”

“…Rare diseases are presented as one of the priorities in the current european Union Public health Program running till 2013 and in addition to the initiatives on the eU level, the member-states develop further regional programmes to ensure adequate access to pharmacotherapy (12,14,15,17,18,19). the commission recommends that Member States put in place strategies organized around national plans for rare diseases and orphan medicines, adequate mechanisms for definition, codification and inventory of rare diseases and production of good practice guidelines, fostering research on rare diseases, including cross-border cooperation, ensuring access to high-quality healthcare, in particular through identifying national and regional centres of expertise (5,18,22).…”