Trends in clinical development timeframes for antiviral drugs launched in the UK, 1981-2014: a retrospective observational study.

OBJECTIVES: Recent decades have witnessed the development of highly innovative new antiviral drug therapies. However, there are concerns that rising costs and lengthening development times could have implications for future patient access to innovative new drugs. We sought to establish whether the time taken for the clinical development of new antiviral drugs launched in the UK had increased since the 1980s. DESIGN AND SETTING: Retrospective observational study of all new antiviral drugs licensed for use in the UK. PRIMARY AND SECONDARY OUTCOME MEASURES: Duration of clinical development (from initiation of studies in humans to receipt of Marketing Authorisation), subdivided into clinical trial and regulatory approval periods by the date of Marketing Authorisation Application. RESULTS: 48 new antiviral drugs were licensed for use in the UK between 1981 and 2014 (inclusive), over half (54%) initially for HIV infection. The overall mean duration of clinical development was 77.2 months, of which 64.6 months was spent in clinical trials before regulatory submission. The total time in clinical development increased from 41.7 months for drugs licensed 1981-1992 to 91.7 months for drugs licensed 2004-2014. This increase was accounted for by an increase in the clinical trials period and not the regulatory approval period, for which there was no observable trend. Drugs initially licensed to treat hepatitis C had a longer duration of clinical development than those indicated for other viral infections. However, the, initially shorter clinical development durations of drugs indicated for HIV infection increased more rapidly across the study period than those indicated for other viral infections. CONCLUSIONS: The time spent by antiviral drugs in clinical development has increased markedly in recent decades despite many initiatives to speed access to innovative new drugs. However, this represents only one part of the translational research pathway, and a complete picture of development timeframes is lacking.

The future role of genetic screening to detect newborns at risk of childhood-onset hearing loss.

OBJECTIVE: To explore the future potential of genetic screening to detect newborns at risk of childhood-onset hearing loss. DESIGN: An expert led discussion of current and future developments in genetic technology and the knowledge base of genetic hearing loss to determine the viability of genetic screening and the implications for screening policy. RESULTS AND DISCUSSION: Despite increasing pressure to adopt genetic technologies, a major barrier for genetic screening in hearing loss is the uncertain clinical significance of the identified mutations and their interactions. Only when a reliable estimate of the future risk of hearing loss can be made at a reasonable cost, will genetic screening become viable. Given the speed of technological advancement this may be within the next 10 years. Decision-makers should start to consider how genetic screening could augment current screening programmes as well as the associated data processing and storage requirements. CONCLUSION: In the interim, we suggest that decision makers consider the benefits of (1) genetically testing all newborns and children with hearing loss, to determine aetiology and to increase knowledge of the genetic causes of hearing loss, and (2) consider screening pregnant women for the m.1555A> G mutation to reduce the risk of aminoglycoside antibiotic-associated hearing loss.

Emerging point of care tests for influenza: innovation or status quo.

BACKGROUND: Point of care tests (POCTs) for influenza potentially offer earlier diagnosis, enabling specific treatment, infection control measures and greater patient convenience and satisfaction. Current POCTs have limited sensitivity, some cannot distinguish influenza types, none differentiate subtypes and are relatively expensive. AIMS: To identify and characterise influenza POCTs expected to be available for clinical use in the U.K. by mid-2013, highlighting those with potential benefits over existing tests. METHODS: Potential developers of influenza POCTs were identified through known manufacturers' websites, Medical Technology trade associations, the EuroScan International Network, an expert advisory group and by searching relevant online sources. Identified companies were asked to provide standard information on relevant technologies. RESULTS: Fifty-six companies were identified, and 29 (52%) responded, identifying 57 potentially relevant technologies. Of these, 40 (70%) were already available or had undetermined status and 5 (9%) were excluded as time to results took over 60 minutes. Of the remaining 12 emerging POCTs, 10 (83%) reportedly enabled differentiation of influenza types and eight differentiation of A subtypes. Nasopharyngeal swabs were the most commonly acceptable sample type; the sample volume ranging from 80 µl to 1.4 ml. DISCUSSION: Most identified emerging influenza POCTs offered differentiation of influenza type and subtype. Tests claiming this capability include several incorporating reverse transcription polymerase chain reaction assays; though, these also had the longest time to result. However, whilst some identified POCTs exhibit high sensitivity and specificity, most lack published clinical data for assessment, and the overall costs of these technologies remains largely unknown.

Prioritisation criteria for the selection of new diagnostic technologies for evaluation.

BACKGROUND: Currently there is no framework for those involved in the identification, evaluation and prioritisation of new diagnostic technologies. Therefore we aimed to develop prioritisation criteria for the assessment of new diagnostic technologies, by gaining international consensus on not only which criteria should be used, but also their relative importance. METHODS: A two-round Delphi process was used to generate consensus amongst an international panel of twenty-six experts on priority criteria for diagnostic health technology assessment. Participants represented a range of health care and related professions, including government, industry, health services and academia. RESULTS: Based on the responses to the first questionnaire 18 criteria were placed into three categories: high, intermediate and moderate priority. For 16 of the 18 criteria, agreement with the categorisation of the criteria into the high, intermediate and moderate categories was high at > or = 70% (10 had agreement > or = 80%). A further questionnaire and panel discussion reduced the criteria to 16 and two categories; seven were classified as high priority and nine intermediate. CONCLUSIONS: This study proposes an objective structure of prioritisation criteria to use when assessing new diagnostic technologies, based on an expert consensus process. The value of these criteria is that no one single component should be used as the decisive driver for prioritisation of new diagnostic technologies for adoption in healthcare settings. Future studies should be directed at establishing the value of these prioritisation criteria across a range of healthcare settings.