Do we need authorized orphan drugs when compounded medications are available?

WHAT IS KNOWN AND OBJECTIVE: Orphan drugs are used to diagnose, prevent or treat a rare disease. This Commentary aims to present a number of case studies questioning the need for designating compounded medications with a long history of effective use, which is well-supported by published clinical evidence. COMMENT: Prior to the market introduction of orphan drugs, medication compounding was done in our hospital pharmacy for several rare diseases. Examples include amifampridine for the treatment of Lambert-Eaton myasthenic syndrome (Firdapse(®)), ibuprofen for the treatment of neonatal patent ductus arteriosus (Pedea(®)) and zinc acetate for the treatment of Wilson's disease (Wilzin(®)). Several 'non-orphan' pharmaceutical products, used off-label for the treatment of rare diseases, that became orphan medicinal products include Hydrea(®) for the treatment of sickle-cell syndrome (Siklos(®)) and Viagra(®) for the treatment of pulmonary arterial hypertension (Revatio(®)). WHAT IS NEW AND CONCLUSION: In our opinion, as indicated by our examples, a better balance should be struck between the development of orphan drugs along the recently established regulatory pathways and the pragmatic use of pharmacy-compounded products and evidence-based off-label use of already available commercial products. Societal needs would be best met by focusing orphan drug development on rare diseases for which there is a high unmet medical need.

Interpretative reporting and alarming based on laboratory data.

The utilisation of laboratory services for patient diagnosis and management involves many steps with both clinical and laboratory components. The clinical components include the decision to order a test, interpretation of the test results and actions taken on the basis of the results. The laboratory components on the other hand include receipt of the request, specimen collection, preparation and analysis, result entry, test result validation and verification and reporting of the results. In this paper, which is part of the OpenLabs project, we concentrate on the post-analytical applications which include interpretation and reporting of the laboratory results to the users in primary care and in high dependency care units. The final objective of the work described is to develop generic modules which can be integrated both with an Open laboratory information system architecture and existing laboratory information processing environment.

Computer aided interpretation of acid-base disorders.

This paper describes an expert system for the interpretation of acid-base disorders. The target users are residents in training in internal medicine, anaesthesia and intensive care medicine. The program is written in PROLOG and runs on a SUN 3/160 minicomputer. Evaluation of a learning set (N = 202) and a test set (N = 194) has proved that the system's accuracy is acceptable. As a result, the program has recently been put in routine clinical practice.

Computer aided phenotyping of dyslipoproteinemia.

An expert system was developed in order to obtain uniform and fast phenotyping and reporting of dyslipoproteinemia. PROLOG is used as knowledge representation formalism. The program is in daily use on an IBM PC AT, in the Laboratory of Clinical chemistry of the University Hospitals in Leuven. Evaluation has proved the system to be reliable and useful for the interpretation of lipoprotein disorders. Indeed accuracy figures of 98.4% and 95.2% were obtained in the learning (N = 315) and test set (N = 126) respectively.