The legal imperative for treating rare disorders

Abstract

BackgroundLife-saving orphan drugs are some of the most expensive medicines. European Union governments aim to accommodate their provision within stretched healthcare budgets but face pressure to reduce funding of such treatments. Patients struggle to retain or gain access to them as their special status is questioned, causing distress and in some cases, fears of premature death. In the UK and EU reimbursement and pricing model of drugs, and orphan drugs in particular, is being re-evaluated.MethodsUsing the United Kingdom as a case study we present, for the first time, legal arguments which compel governments to provide orphan medicinal products. These include (i) disability legislation, (ii) national and organisational constitutions, (iii) judicial review, (iv) tort law and (v) human rights legislation. We then address directly potential objections to our analysis and counter arguments which aim to limit provision of orphan drugs to the intended patient recipients.ResultsWe demonstrate that a compelling case can be made that the law demands the treatment of orphan diseases.ConclusionsOur legal framework will assist doctors and patients in ensuring the continued provision of treatments despite significant economic pressure to reduce funding. These legal avenues will empower stakeholders in drafting funding guidelines throughout the EU. The legal right to treatment extends beyond rare diseases and our analysis may therefore affect allocation of healthcare budgets throughout the EU.

Ethical imperatives of timely access to orphan drugs: is possible to reconcile economic incentives and patients’ health needs?

“…Country specific court cases have led to a broad interpretation of national legislation in support of patient’s rights to health care [47] including treatment when no therapeutic alternative exist. Court cases may support patients’ rights to health and health care in cases where clear guidelines are not codified by law and individuals are denied access to treatment.…”

Compassionate use of orphan drugs

“…We contend elsewhere that public and private health providers should offer life-saving treatments once they are thus available in the market [ 4 , 5 ]. However, if a patient has no alternative treatment available in the meantime, they will be deprived effective treatment of their disease.…”

“…We contend elsewhere that human rights and disability legislation require governments to reimburse the costs of orphan medicinal treatments [ 4 ], but these appeals ring hollow if a country is too poor to offer its citizens more than the most rudimentary care. At first, it also seems difficult to argue that a company has a legal obligation to give away drugs for free.…”

“…Simultaneously, safeguards would need to exist to stop companies from being taken advantage of by a care provider’s refusal or delay in paying for treatment. A company might for example be able to bring a judicial review request against a public authority’s refusal to purchase a treatment, although victory in judicial review is very uncertain and the merit of each case would have to be assessed carefully in each case [ 4 ].…”

Availability of Orphan drugs: Basic legal issues of development and financing

“…Agencija izdaje dozvolu za kliničko ispitivanje leka u roku od 60 dana od dana prijema zahteva sa potpunom dokumentacijom. 7 Predlagač po odobrenju zaključuje ugovor sa zdravstvenom ustanovom o sprovođenju kliničkog ispitivanja. Predlagač je dužan da pre početka ispitivanja osigura lica koja učestvuju u ispitivanju za slučaj nastanka štete po zdravlje tih lica izazvane kliničkim ispitivanjem leka, kao i da ugovorom odredi iznos neophodnih troškova koji pripadaju licima koja učestvuju u kliničkom ispitivanju.…”