Retomis ligomis sergančių pacientų teisinės galimybės gydytis neregistruotais vaistais

LTRetoms ligoms gydyti skirtų vaistų prieinamumo klausimas į Europos Sąjungos (ES) teisės reguliavimo sritį prieš dešimtmetį pateko siekiant paskatinti jų kūrimą ir taip padėti užtikrinti geresnę retomis ligomis sergančių pacientų sveikatos priežiūrą. Tuo tikslu prieš keletą metų ES lygiu priimtos ir teisės normos dėl pacientų galimybių gydytis dar tiriamu – taigi neregistruotu vaistiniu preparatu, jei jie serga retomis, sunkiomis, gyvybei gresiančiomis ligomis ir negali dalyvauti to vaisto klinikiniame tyrime, o kitų gydymo alternatyvų nėra. Straipsnyje taikant istorinį, lyginamąjį, dokumentų analizės, sintezės bei apibendrinimo metodus, remiantis ES ir nacionalinių teisės aktų normomis bei JAV sveikatos teisės doktrina ir jurisprudencija analizuojami retomis ligomis sergančių pacientų teisinių galimybių gydytis neregistruotais vaistais ypatumai, probleminiai aspektai. Siekiama atskleisti neregistruotų vaistų prieinamumo būdų skirtumus, reikšmę, išsiaiškinti, kodėl ne visi iš galimų šių vaistų prieinamumo būdų yra teisėti, svarstoma, kodėl ne visose šalyse jie įteisinti ir veikia. [Iš leidinio]Reikšminiai žodžiai: Farmacijos teisė; Labdaringos vaistų programos; Neregistruoti vaistai; Neregistruotų vaistų prieinamumo reglamentavimas; Pacientų teisės; Retomis ligomis sergantys pacientai; Sveikatos priežiūros teisė; Tiriamieji vaistai; Vaistų prieinamumas; Access to medicines; Compassionate use programmes; Health care law; Investigational drugs; Legal regulation of unauthorized medicines; Patients suffering from rare diseases; Patients' rights; Pharmaceutical law.

ENIn the European Union (EU), medicines can be marketed only after they have been authorised. However, some medicines need to be accessible before they have been authorised. This especially applies to medicines, which are expected to benefit severely ill patients who cannot be treated with authorised medicines or who have a disease for which no medicine has been authorised yet. The treatment with unauthorised medicines may be the last chance for patients who suffer from rare diseases and cannot enrol in an ongoing clinical trial or be treated with a potentially life-saving medicine. The aim of this article is to analyse the aspects of the legal regulation of access to unauthorised medicines for such patients. The fact that 27-36 million patients with rare diseases live in the EU and there are approximately 200,000-240,000 of such patients in Lithuania underlines the relevance of the study. The first part of the article analyses the EU legal provisions, which govern the so called “named patient” programmes for single patient use under the Article 5 of Directive 2001/83/ EC (ex Article 3.1 of Directive 1989/341/EEC) and “compassionate use” programmes for a group of patients set out in the Article 83 of Regulation (EC) No 726/2004. Upon the introduction of these legal exceptions into the national law, medicines intended for the treatment of rare diseases can reach patients more quickly and easily.The national legal regulation of patient access to unauthorised medicines differs by country, because EC regulations define only the general principles. Therefore, access to new investigational medicines depends on the country where the patient resides. This means that patients are not always able to access medicines that would be of benefit to them. As a consequence, their lives can be endangered. Such a situation is inequitable. The second part of the article reviews the legal framework of access to unauthorised medicines in Lithuania. Patients are able to access medicines approved in other countries for a genuine, unmet medical need before the drugs are licensed in Lithuania. The treatment, however, with investigational drugs for individual patients and for groups of patients who are unable to participate in clinical trials and suffer from rare, life-threatening diseases is not regulated. According to the author, the Law on Pharmacy of the Republic of Lithuania must be supplemented with such important exceptions. The third part of the article addresses the question why patients have no access to potentially life saving post-Phase I investigational new drugs. The article considers the margins of personal autonomy principle in the light of the problem of accessibility to unapproved medicinal products intended for treatment of rare diseases. In the summary of the findings of the article, the author emphasize the need for joint approach of EU Member States to availability of unauthorised medicinal products for orphan patients. [From the publication]