PRO80 COMPARISON OF EARLY ACCESS PROGRAMMES FOR ORPHAN DRUGS IN THE EU-5 COUNTRIES

The Orphan Drug Act has become a staple of food and drug law in the US. The experience with the US programme continues to serve as a useful reference point as interest in orphan drug incentive programmes expands globally. This article first reviews details of the legislation and orphan drug regulations, and then provides a 13-year overview (1983-1995) of orphan drug activity in the US, including descriptive data on the designated and approved orphan drugs, their indications and sponsors. In light of a recent challenge to the Food and Drug Administration's (FDA's) authority under the Act, we also examine the interplay between the exclusivity provision of the Act and the orphan drug regulations that define when 2 drugs will be considered the 'same' for the purposes of the Act. A recent court decision affirming the FDA's interpretation of the clinical superiority provisions of the regulations suggests that orphan exclusivity may be less predictable and less certain than it has been in the past. Finally, we consider the usefulness to orphan drug sponsors of other initiatives such as FDA's early access and fast-track approval programmes, and the extent to which the FDA's discretion to waive, defer and reduce prescription drug user fees has worked to the benefit of orphan drug sponsors. Over the 13-year analysis period, the FDA granted 631 orphan designations involving 450 different drugs, for which 121 FDA marketing approvals have been granted. Those with both treatment investigational drug designation and fast-track approval status appeared to benefit substantially from shorter development times. The indications targeted by the orphan drugs fall into 8 categories, with 40% of all orphan indications involving cancer and genetic diseases. Evident in the latter part of the analysis period is the increasing share of orphan activity attributable to biotechnology firms. Even though the Prescription Drug User Fee Act of 1992 did not recognise orphan status for the purpose of waiving or reducing the amount of fees payable, almost one-third (32%) of the $US 8,531,550 in user fee revenue foregone through waivers and reductions was attributable to orphan drugs.

BackgroundRare diseases are life-threatening, serious, and chronic conditions that require complex care and have a low prevalence. An estimated one in 15 people worldwide are affected by rare diseases. This study aims to analyze the accessibility, reimbursement status, licensed status, and Anatomical Therapeutic Chemical (ATC) codes of drugs that the European Medicines Agency (EMA) in Turkey considers to be “orphan” pharmaceuticals.MethodsThe drugs included in this analysis were obtained from the list of orphan drugs published by the EMA. Orphan drugs’ accessibility and licensing status in Turkey were obtained from the Health Implementation Communiqué published by the Social Security Institution (SGK) and the List of Abroad Active Substance and List of Licensed Products published by the Turkey Pharmaceuticals and Medical Devices Agency (TİTCK). Descriptive analysis was applied to determine the accessibility status of orphan drugs identified by the EMA in Turkey.ResultsBased on the EMA, 105 pharmaceuticals were approved with “orphan drug” status except for drugs that have lost orphan drug status, decommissioned in the European Union and withdrawn from the European Community Register by January 2020. Of the 105 rare drugs on the EMA list, 34 were inaccessible in Turkey. Of the 71 available drugs, 23 (32%) were licensed and 48 (68%) were unlicensed in Turkey. 17 (74%) of licensed products and 17 (35%) of unlicensed products were covered by reimbursement. When orphan drugs’ ATC codes were examined, the most common ATC group was found to be “L—Antineoplastic and Immunomodulatory” agents.ConclusionAn orphan drug incentive policy is very important to ensure early access to the drugs used to treat rare diseases. Considering the capacity and prices for orphan drugs in Turkey, it can be said that many patients with rare diseases have difficulty in their treatment. It is obvious that such a policy must prepare for the regulation of orphan drugs in Turkey.

This article analyses how the regulatory incentives provided by Regulation 141/2000 affect orphan drug development and generic market entry. It seeks to find out how the regulatory incentives work in combination with patent protection, and whether in combination they foster orphan drug innovation and overall patient access, or rather hinder the latter. The article argues that even though the regulatory incentives are targeted to fostering innovation and early generic market access, when combined with patent protection, the generic entry is likely blocked or delayed.

HPR53 Changes to Early Access Scheme in France: Early Learnings for Orphan Drugs