Opportunities and Challenges for Drug Development: Public–Private Partnerships, Adaptive Designs and Big Data

Abstract

Drug development faces the double challenge of increasing costs and increasing pressure on pricing. To avoid that lack of perceived commercial perspective will leave existing medical needs unmet, pharmaceutical companies and many other stakeholders are discussing ways to improve the efficiency of drug Research and Development. Based on an international symposium organized by the Medical School of the University of Duisburg-Essen (Germany) and held in January 2016, we discuss the opportunities and challenges of three specific areas, i.e., public–private partnerships, adaptive designs and big data. Public–private partnerships come in many different forms with regard to scope, duration and type and number of participants. They range from project-specific collaborations to strategic alliances to large multi-party consortia. Each of them offers specific opportunities and faces distinct challenges. Among types of collaboration, investigator-initiated studies are becoming increasingly popular but have legal, ethical, and financial implications. Adaptive trial designs are also increasingly discussed. However, adaptive should not be used as euphemism for the repurposing of a failed trial; rather it requires carefully planning and specification before a trial starts. Adaptive licensing can be a counter-part of adaptive trial design. The use of Big Data is another opportunity to leverage existing information into knowledge useable for drug discovery and development. Respecting limitations of informed consent and privacy is a key challenge in the use of Big Data. Speakers and participants at the symposium were convinced that appropriate use of the above new options may indeed help to increase the efficiency of future drug development.

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“…In addition, the process of development often takes 15 years at a cost of 2.6 billion U.S. dollars . Safety issues in the laboratories during the clinical application process also present a challenge . With this paradigm, identifying the target protein of bioactive small molecules to reveal the underlying mechanism requires both control of safety issues during pre‐clinical stages, and the synthesis of improved analogs at a reduced cost …”

New Mechanisms to Fund Independent Clinical Trials for Repurposing Off-Patent or Generic Medicines

“…Moreover, several government organisations, such as the Belgian Healthcare Knowledge Center (KCE), the Dutch ZonMw and the UK National Institute for Health Research (NIHR), have included drug repurposing as a focus area in their calls for funding of independent clinical research (44)(45)(46). Some pharmaceutical companies also provide grants to support investigator-initiated clinical research with their approved medicines (47)(48)(49)(50)(51). Bayer even ran a speci c 'Grants4Indications' program that provided grants and further nancial support to explore new therapeutic indications for their own compounds (52).…”

Commentary to “Patients not patents: drug research and development as a public enterprise”

“…“The regulatory requirements are particularly low in cancer, and many hugely expensive cancer drugs have been approved without the existence of a single randomised trial and with only surrogate outcomes, eg disease‐free survival instead of longer life.” The above statement by the author although true appears to overtly underestimate the importance of adaptive trial designs …”

“…The above statement by the author although true appears to overtly underestimate the importance of adaptive trial designs. 7,8 "Trials submitted for obtaining marketing authorization should be large enough and run for sufficient lengths of time to capture rare but lethal harms. .…”