Zeeshaan Arshad

Open Access Could Transform Drug Discovery: A Case Study of JQ1.

Abstract Introduction: The cost to develop a new drug from target discovery to market is a staggering

Cell assisted lipotransfer in breast augmentation and reconstruction: A systematic review of safety, efficacy, use of patient reported outcomes and study quality.

1.8 billion, largely due to the very high attrition rate of drug candidates and the lengthy transition times during development. Open access is an emerging model of open innovation that places no restriction on the use of information and has the potential to accelerate the development of new drugs. Areas Covered: To date, no quantitative assessment has yet taken place to determine the effects and viability of open access on the process of drug translation. This need is addressed within this study. The literature and intellectual property landscapes of the drug candidate JQ1, which was made available on an open access basis when discovered, and conventionally developed equivalents that were not are compared using the Web of Science and Thomson Innovation software, respectively. Expert opinion: Results demonstrate that openly sharing the JQ1 molecule led to a greater uptake by a wider and more multi-disciplinary research community. A comparative analysis of the patent landscapes for each candidate also found that the broader scientific diaspora of the publically released JQ1 data enhanced innovation, evidenced by a greater number of downstream patents filed in relation to JQ1. The authors’ findings counter the notion that open access drug discovery would leak commercial intellectual property. On the contrary, JQ1 serves as a test case to evidence that open access drug discovery can be an economic model that potentially improves efficiency and cost of drug discovery and its subsequent commercialization.

Apert syndrome: A consensus on the management of Apert hands

Background Cell assisted lipotransfer serves as a novel technique for both breast reconstruction and breast augmentation. This systematic review assesses the efficacy, safety and use of patient reported outcome measures in studies involving cell assisted lipotransfer. We also carry out an objective assessment of study quality focussing on recruitment, follow-up and provide an up-to-date clinical trial landscaping analysis. Methods Key electronic databases were searched according to PRISMA guidelines and pre-defined inclusion and exclusion criteria. Two independent reviewers examined the retrieved publications and performed data extraction. Results 3980 publications were identified. Following screening, 11 studies were included for full review, representing a total of 336 patients with a follow-up time ranging from six to 42 months. A degree of variation was noted in graft retention and reported satisfaction levels, although there were only three comparative studies with conflicting results. Complications occurred at a rate of 37%. Additionally, there was a paucity of objective outcomes assessments (e.g. 3D assessment modalities or validated patient reported outcome measures) in the selected studies. Conclusions Cell assisted lipotransfer is a surgical technique that is currently employed sparingly within the plastic & reconstructive surgery community. Presently, further technical and outcome standardization is required, in addition to rigorous randomized controlled trials and supporting long-term follow-up data to better determine procedural safety and efficacy. Routine use of more objective outcome measures, particularly 3D assessments and validated patient reported outcome measures, will also help facilitate wider clinical adoption and establish procedural utility.

Regulatory barriers to the advancement of precision medicine

INTRODUCTION Apert Syndrome is a congenital condition characterised by primary craniosynostosis, midfacial malformations and complex symmetrical malformations of the hands and feet. The hands demonstrate one of the most complex collections of congenital upper limb deformities, posing a significant challenge for the paediatric hand surgeon. This study examines the extant literature and current practice of the four UK specialist craniofacial units regarding the management of Apert hands in order to provide a basis for guideline development. METHODS The current literature was reviewed. Survey-type questionnaires were distributed to the four UK specialist craniofacial units and responses analysed. RESULTS Management of the Apert hand is largely dictated by the degree of malformation present. Although all units aim to achieve a five digit hand, variation in the timing of surgery, operative protocols and mobilisation policies exist. CONCLUSION The results of this study provide an interesting snapshot of the current management of Apert hands across four UK craniofacial surgery units. The four UK units remain congruent on most areas surrounding the management of Apert hands although some minor inter-unit variation exists. A multidisciplinary approach to management remains fundamental in optimising the regain of function and aesthetically acceptable hands.

21st Century Cures Act: An Act of Cure or Diagnosis?

ABSTRACT Precision medicine utilizes tailored diagnostic, prognostic and therapeutic strategies based on an individual’s molecular profile. Although it is gaining considerable traction and high-level political endorsement, it must overcome a number of translational hurdles, including regulatory barriers. At the core of precision medicine lies diagnostic tests and devices, however the regulatory classification of such products varies on a global basis. Navigating these convoluted regulatory pathways can be challenging – exacerbated by asymmetric technological advancement and regulatory progression. Both the EU and US are attempting to address such issues and newer concerns relating to direct-to-consumer testing. Flexible solutions are required to establish regulatory compliance across multiple countries and coordinated cross-collaboration initiatives need to empower technological development and globally harmonized regulation. The wider infrastructure, spanning beyond regulation, must also accommodate these changes and support subsequent clinical adoption, in order to firmly establish precision medicine in modern day medical practice.

An assessment of the factors affecting the commercialization of cell-based therapeutics: a systematic review protocol

The 21st Century Cures Act1 is a positive step for biomedical innovation. However, despite a rare bipartisan approval in the House of Representatives with an overwhelming vote of 344–77, public opinion is divided; naysayers have already vocally claimed that the reforms do not go far enough, while others have criticized the bill for promoting increased approval speeds at the expense of patient safety.2 As biomedical researchers and patients wanting novel, and safe, therapies to reach the market, it is difficult not to sympathize with negative factions; but is this fair or productive?

Evidence of insufficient quality of reporting in patent landscapes in the life sciences

BackgroundCellular-based therapies represent a platform technology within the rapidly expanding field of regenerative medicine and are distinct from conventional therapeutics—offering a unique approach to managing what were once considered untreatable diseases. Despite a significant increase in basic science activity within the cell therapy arena, alongside a growing portfolio of cell therapy trials and promising investment, the translation of cellular-based therapeutics from “bench to bedside” remains challenging, and the number of industry products available for widespread clinical use remains comparatively low. This systematic review identifies unique intrinsic and extrinsic barriers in the cell-based therapy domain.Methods/designEight electronic databases will be searched, specifically Medline, EMBASE (OvidSP), BIOSIS & Web of Science, Cochrane Library & HEED, EconLit (ProQuest), WHOLIS WHO Library Database, PAIS International (ProQuest), and Scopus. Addition to this gray literature was searched by manually reviewing relevant work. All identified articles will be subjected for review by two authors who will decide whether or not each article passes our inclusion/exclusion criteria. Eligible papers will subsequently be reviewed, and key data extracted into a pre-designed data extraction scorecard. An assessment of the perceived impact of broad commercial barriers to the adoption of cell-based therapies will be conducted. These broad categories will include manufacturing, regulation and intellectual property, reimbursement, clinical trials, clinical adoption, ethics, and business models. This will inform further discussion in the review. There is no PROSPERO registration number.DiscussionThrough a systematic search and appraisal of available literature, this review will identify key challenges in the commercialization pathway of cellular-based therapeutics and highlights significant barriers impeding successful clinical adoption. This will aid in creating an adaptable, acceptable, and harmonized approach supported by apposite regulatory frameworks and pertinent expertise throughout the respective stages of the adoption cycle to facilitate the adoption of new products and technologies in the industry.

Adipose-Derived Stem Cells in Aesthetic Surgery: A Mixed Methods Evaluation of the Current Clinical Trial, Intellectual Property, and Regulatory Landscape

Despite the importance of patent landscape analyses in the commercialization process for life science and healthcare technologies, the quality of reporting for patent landscapes published in academic journals is inadequate.