Conceptual clarifications regarding Chilean Act 20850 on public funding of high-cost diseases.

In 2015, Chile enacted the 20850 law, providing public funds for rare and costly diseases that demanded high diagnostic and therapeutic expenditures. The law modifies the Chilean Sanitary Code regulation of research with human beings, aiming at the protection of subjects by securing post-investigational medical benefits and insurance coverage for damage imputable to the research they participated in. Due to ambiguous phrasing, a polemic rose for fear that these protective measures applied to all clinical research, although a careful reading of the law in its context clearly suggests that it refers to phase I therapeutic trials. This paper stresses the distinction between compassionate use and genuine phase I/II therapeutic trials aimed at both pharmacodynamics and an intended therapeutic effect for severe and progressive diseases that are therapeutically orphaned, emphasizing the ethical and medical duty of providing post-trial beneficial medication.

En 2015 se publica en Chile la Ley 20850, cuyo objetivo declarado es el financiamiento público de enfermedades raras y de aquellas de alto costo diagnóstico y terapéutico. Inserto en la ley hay un articulado a introducir en el Código Sanitario, que exige de las investigaciones clínicas que mantengan los beneficios médicos determinados por el estudio, para los pacientes investigados, por todo el tiempo que sea médicamente necesario; amparado por extensos seguros para cubrir eventuales complicaciones y efectos indeseados. La redacción de la ley había motivado intensas polémicas, debido a su imprecisa redacción que permite interpretar que la protección exigida es extensible a todo estudio clínico; siendo que la lectura atenta y el contexto de este articulado claramente lo refieren a terapias experimentales. Este artículo distingue entre uso compasivo y terapias experimentales genuinas, que enlazan Fase I (delimita dosis máximas no tóxicas en individuos sanos) y Fase II (estudia efectividad en pequeños grupos de pacientes), investigando tanto farmacodinamia como efectos terapéuticos para enfermedades graves, en deterioro progresivo y huérfanas de tratamiento, con el objetivo ético y médico de la disponibilidad de efectos benéficos, más allá de terminado el estudio.

Quantifying the magnitude and cost of collecting extraneous protocol data.

Although most research professionals believe that protocol designs contain a growing number of unnecessary and redundant procedures generating unused data, incurring high cost, and jeopardizing study success, there are no published studies systematically examining this issue. Between November 2011 and May 2012, Tufts Center for the Study of Drug Development conducted a study among a working group of 15 pharmaceutical companies in which a total of 25,103 individual protocol procedures were evaluated and classified using clinical study reports and analysis plans. The results show that the typical later-stage protocol had an average of 7 objectives and 13 end points of which 53.8% are supplementary. One (24.7%) of every 4 procedures performed per phase-III protocol and 17.7% of all phase-II procedures per protocol were classified as "Noncore" in that they supported supplemental secondary, tertiary, and exploratory end points. For phase-III protocols, 23.6% of all procedures supported regulatory compliance requirements and 15.9% supported those for phase-II protocols. The study also found that on average, $1.7 million (18.5% of the total) is spent in direct costs to administer Noncore procedures per phase-III protocol and $0.3 million (13.1% of the total) in direct costs are spent on Noncore procedures for each phase-II protocol. Based on the results of this study, the total direct cost to perform Noncore procedures for all active annual phase-II and phase-III protocols is conservatively estimated at $3.7 billion annually, not including the indirect costs associated with collecting and managing Noncore procedure data and the ethical costs of exposing study volunteers to unnecessary risks associated with conducting extraneous procedures.