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Starting with the launch of the Human Genome Project three decades ago, and continuing after its completion in 2003, genomics has progressively come to have a central and catalytic role in basic and translational research. In addition, studies increasingly demonstrate how genomic information can be effectively used in clinical care. In the future, the anticipated advances in technology development, biological insights, and clinical applications (among others) will lead to more widespread integration of genomics into almost all areas of biomedical research, the adoption of genomics into mainstream medical and public-health practices, and an increasing relevance of genomics for everyday life. On behalf of the research community, the National Human Genome Research Institute recently completed a multi-year process of strategic engagement to identify future research priorities and opportunities in human genomics, with an emphasis on health applications. Here we describe the highest-priority elements envisioned for the cutting-edge of human genomics going forward—that is, at ‘The Forefront of Genomics’. In this Perspective, authors from the National Human Genome Research Institute (NHGRI) present a vision for human genomics research for the coming decade.

Background Randomised controlled trials (RCTs) are the gold standard assessment for health technologies. A key aspect of the design of any clinical trial is the target sample size. However, many publicly-funded trials fail to reach their target sample size. This study seeks to assess the current state of recruitment success and grant extensions in trials funded by the Health Technology Assessment (HTA) program and the UK Medical Research Council (MRC). Methods Data were gathered from two sources: the National Institute for Health Research (NIHR) HTA Journal Archive and the MRC subset of the International Standard Randomised Controlled Trial Number (ISRCTN) register. A total of 440 trials recruiting between 2002 and 2008 were assessed for eligibility, of which 73 met the inclusion criteria. Where data were unavailable from the reports, members of the trial team were contacted to ensure completeness. Results Over half (55%) of trials recruited their originally specified target sample size, with over three-quarters (78%) recruiting 80% of their target. There was no evidence of this improving over the time of the assessment. Nearly half (45%) of trials received an extension of some kind. Those that did were no more likely to successfully recruit. Trials with 80% power were less likely to successfully recruit compared to studies with 90% power. Conclusions While recruitment appears to have improved since 1994 to 2002, publicly-funded trials in the UK still struggle to recruit to their target sample size, and both time and financial extensions are often requested. Strategies to cope with such problems should be more widely applied. It is recommended that where possible studies are planned with 90% power.

The aim of the study was to compare response proportions and research costs of telephone calling vs. continued emailing nonresponding authors of studies included in a systematic review. Key features of included studies were poorly reported in a systematic review of diabetes quality improvement interventions. We developed a survey to request additional information from contact authors. After three email contact attempts, only 76 of 279 authors (27%) had completed the survey. In this study, we randomly assigned nonresponding authors to contact by telephone calling vs. continued emailing to compare the effect of these strategies on response proportions and research costs. We randomized 87 authors to telephone and 89 to email contact. Telephone contact increased survey completion (36.7% vs. 20.2%; adjusted risk difference of 15.6% [95% confidence interval: 2.90%, 28.4%]; adjusted odds ratio 2.26 [95% confidence interval: 1.10, 4.76]) but required more time to deliver (20 vs. 10 hours in total; 14 vs. 7 minutes per randomized author; 26 vs. 4 weeks), and cost more (total intervention cost of $504 Canadian dollars vs. $252 for the telephone and email arm, respectively). Contacting nonresponding authors of included studies by telephone increased response compared with emailing but required more investigator time and had higher cost.

ObjectivesThe Breathing for Life Trial (BLT) was a multicentre randomised controlled trial testing the hypothesis that a fractional exhaled nitric oxide-based intervention to guide asthma therapy in pregnancy improves perinatal outcomes. While BLT was negative based on selected outcomes, the conduct of the trial over 7 years showed potential for assessing the broader research impacts and returns on investment in BLT. The aim of this study was to retrospectively assess and report on the impact and value of BLT to show accountability for the research investment in what was deemed a ‘negative’ trial.MethodsThe Framework to Assess the Impact from Translational health research (FAIT) was selected as the preferred method. FAIT combines three validated methods, including a modified Payback framework, an economic analysis of return on investment and a narrative account of the impact generated from the research. Data collection was done via document analysis of BLT administrative and research records and review of relevant websites/databases.ResultsBLT delivered a return on investment of $6.7 million in leveraged grants, fellowships and consultancies and conservatively returned $2.44 for every dollar invested. The research trained and upskilled 18 midwives and obstetricians in evidence-based asthma management in pregnancy and improved research capability of six PhD students. Specialised equipment purchased by BLT is now being repurposed to undertake other research in regional Australia, saving further research investment. Of the 1200 mothers who were part of BLT, 508 now have written asthma plans, 268 had a clinically significant improvement in their asthma control score and the proportion who improved their asthma plan knowledge increased by 58 percentage points from 12 to 70%.ConclusionThis case example in the developing field of impact assessment illustrates how researchers can use evidence to demonstrate and report more broadly on the impact of and returns on research investment in a clinical trial.Trial registration numberACTRN12613000202763; Post results.

A €30 million initiative bringing together academic centres, industry, charities and patient groups provides an unprecedented opportunity for translational research on autism.A €30 million initiative bringing together academic centres, industry, charities and patient groups provides an unprecedented opportunity for translational research on autism.

Quantifying the value of investment in medical research can inform decision-making on the prioritisation of research programmes. Existing methodologies to estimate the rate of return of medical research are inappropriate for early-phase translational research due to censoring of health benefits and time lags. A strategy to improve the process of translational research for patient benefit has been initiated as part of the UK National Institute for Health Research (NIHR) investment in Biomedical Research Centres (BRCs) in England. By providing a platform for partnership between universities, NHS trusts and industry, successful BRCs should reduce time lags within translational research whilst also providing an impetus for local economic growth through industry collaboration. We present a novel contribution in the assessment of early-phase biomedical research by estimating the impact of the Oxford Biomedical Research Centre (OxBRC) on income and job creation following the initial NIHR investment. We adopt a macroeconomic assessment approach using Input-Output Analysis to estimate the value of medical research in terms of income and job creation during the early pathway towards translational biomedical research. Inter-industry linkages are assessed by building a model economy for the South East England region to estimate the return on investment of the OxBRC. The results from the input-output model estimate that the return on investment in biomedical research within the OxBRC is 46%. Each £1 invested in the OxBRC generates an additional £0.46 through income and job creation alone. Multiplicative employment effects following a marginal investment in the OxBRC of £98m during the period 2007-2017 result in an estimated additional 196 full time equivalent positions being created within the local economy on top of direct employment within OxBRC. Results from input-output analyses can be used to inform the prioritisation of biomedical research programmes when compared against national minimum thresholds of investment.

Background Realising the economic potential of research institutions, including medical research institutes, represents a policy imperative for many Organisation for Economic Co-operation and Development nations. The assessment of research impact has consequently drawn increasing attention. Research impact assessment frameworks (RIAFs) provide a structure to assess research translation, but minimal research has examined whether alternative RIAFs realise the intended policy outcomes. This paper examines the objectives presented for RIAFs in light of economic imperatives to justify ongoing support for health and medical research investment, leverage productivity via commercialisation and outcome–efficiency gains in health systems, and ensure that translation and impact considerations are embedded into the research process. This paper sought to list the stated objectives for RIAFs, to identify existing frameworks and to evaluate whether the identified frameworks possessed the capabilities necessary to address the specified objectives. Methods A scoping review of the literature to identify objectives specified for RIAFs, inform upon descriptive criteria for each objective and identify existing RIAFs. Criteria were derived for each objective. The capability for the existing RIAFs to realise the alternative objectives was evaluated based upon these criteria. Results The collated objectives for RIAFs included accountability (top-down), transparency/accountability (bottom-up), advocacy, steering, value for money, management/learning and feedback/allocation, prospective orientation, and speed of translation. Of the 25 RIAFs identified, most satisfied objectives such as accountability and advocacy, which are largely sufficient for the first economic imperative to justify research investment. The frameworks primarily designed to optimise the speed of translation or enable the prospective orientation of research possessed qualities most likely to optimise the productive outcomes from research. However, the results show that few frameworks met the criteria for these objectives. Conclusion It is imperative that the objective(s) for an assessment framework are explicit and that RIAFs are designed to realise these objectives. If the objectives include the capability to pro-actively drive productive research impacts, the potential for prospective orientation and a focus upon the speed of translation merits prioritisation. Frameworks designed to optimise research translation and impact, rather than simply assess impact, offer greater promise to contribute to the economic imperatives compelling their implementation.

Background Building on an approach applied to cardiovascular and cancer research, we estimated the economic returns from United Kingdom public- and charitable-funded musculoskeletal disease (MSD) research that arise from the net value of the improved health outcomes in the United Kingdom. Methods To calculate the economic returns from MSD-related research in the United Kingdom, we estimated (1) the public and charitable expenditure on MSD-related research in the United Kingdom between 1970 and 2013; (2) the net monetary benefit (NMB), derived from the health benefit in quality adjusted life years (QALYs) valued in monetary terms (using a base-case value of a QALY of £25,000) minus the cost of delivering that benefit, for a prioritised list of interventions from 1994 to 2013; (3) the proportion of NMB attributable to United Kingdom research; and (4) the elapsed time between research funding and health gain. The data collected from these four key elements were used to estimate the internal rate of return (IRR) from MSD-related research investments on health benefits. We analysed the uncertainties in the IRR estimate using a one-way sensitivity analysis. Results Expressed in 2013 prices, total expenditure on MSD-related research from 1970 to 2013 was £3.5 billion, and for the period used to estimate the rate of return, 1978-1997, was £1.4 billion. Over the period 1994–2013 the key interventions analysed produced 871,000 QALYs with a NMB of £16 billion, allowing for the net NHS costs resulting from them and valuing a QALY at £25,000. The proportion of benefit attributable to United Kingdom research was 30% and the elapsed time between funding and impact of MSD treatments was 16 years. Our best estimate of the IRR from MSD-related research was 7%, which is similar to the 9% for CVD and 10% for cancer research. Conclusions Our estimate of the IRR from the net health gain to public and charitable funding of MSD-related research in the United Kingdom is substantial, and justifies the research investments made between 1978 and 1997. We also demonstrated the applicability of the approach previously used in assessing the returns from cardiovascular and cancer research. Inevitably, with a study of this kind, there are a number of important assumptions and caveats that we highlight, and these can inform future research.

How the NIH Can Help You Get Funded is an insider's guide to planning and preparing competitive grant applications. The book demystifies the NIH and the process of crafting the proposal, how award decisions are made, and next steps after their review. Readers learn how and when to interact with NIH staff and how to use available data in developing their personal grant strategy.

The long-held but erroneous assumption of never-ending rapid growth in biomedical science has created an unsustainable hypercompetitive system that is discouraging even the most outstanding prospective students from entering our profession—and making it difficult for seasoned investigators to produce their best work. This is a recipe for long-term decline, and the problems cannot be solved with simplistic approaches. Instead, it is time to confront the dangers at hand and rethink some fundamental features of the US biomedical research ecosystem.