Orphan drugs policies: a suitable case for treatment

Abstract

Context Current orphan drug policies are unsatisfactory when viewed from almost all perspectives. Patients find that, although therapies are available for many rare conditions, access to care is sometimes restricted. Pharmaceutical manufacturers have responded to the incentives for research embodied in orphan drug legislation, only to find that funds are not made available to pay for therapies once developed. Those funding health care find that most orphan drugs do not justify funding based on standard value for money criteria, yet that they face political problems if they fail to provide funding for therapy. Methods A literature review was conducted in order to determine the precise nature of the problems and to suggest potential solutions. Results Current orphan drug policies are not fit for the purpose and initiatives need to be taken in the areas of (1) clarifying society's views about the priority to be given to orphan drugs, (2) revising the arrangements for pricing and reimbursement of orphan drugs, (3) defining the priorities for research into rare diseases and (4) developing 'joined up' policies to deal with these issues. Conclusions Without changes in the current policies, pharmaceutical companies will eventually cease responding to the incentives to develop orphan drugs, because they will increasingly be uncertain whether the drugs, if developed, will be reimbursed.

Reimbursed Price of Orphan Drugs: Current Strategies and Potential Improvements

“…Health technology assessment (HTA) [18] , as described by Lampe et al [19] , is intended to identify, appraise, and synthesise all available scientific evidence to inform decisions considering 10 different domains: characterisation of the specific health problem and existing current treatment alternatives; description of the technology under scrutiny; evidence on safety, clinical effectiveness (comparative effectiveness), and costeffectiveness; budget impact analysis; ethical analysis; and considerations of organisational, social, and legal issues. In the case of ODs, several of the above-reported domains become critical [9,12,[20][21][22][23][24][25] , feeding an open worldwide debate about the appropriateness of existing HTA methods for health technologies devoted to RDs [26][27][28][29] . Increasingly, considerations of using an extended set of criteria beyond the conventional core assessment of safety, efficacy, and cost-effectiveness is gaining acceptance even among the HTA community [30][31][32] .…”

Orphan Drugs for Rare Diseases

“…One issue has become relevant given advances in personalised medicine where a subset of the patient population can be defi ned by the existence of a biomarker [ 16 ]. This might lead to the fact that applicants for orphan designation will 'salami slice' treatment indications in order to qualify for orphan status [ 17 ].…”

Scientific and Social Value Judgments for Orphan Drugs in Health Technology Assessment

“…(12) Little evidence in support of a societal preference for rare conditions exists, and the few studies that attempted to elucidate this suggested the contrary when patients with more common diseases were denied treatment in order to treat fewer patients with a rare condition. (12)(13)(14)(15) In such cases, these decisions partly rely on the decision-makers' willingness to accept high ICERs based on additional factors that influence their judgment of (scientific and social) value, such as, for example, disease severity, the treatment's orphan status, or to what extent evidence characterised by uncertainty is acceptable. (16) They also rely on the flexibility of these processes in, for example, their ability to implement managed entry agreements or the availability of separate funding programs (e.g.…”