Article on “Targeted therapies: speed and collaboration”

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During drug discovery, collaborations can ensure that points in the system where bottlenecks occur can be kept wide open. Programs like ‘fast track designation’, ‘translational research’ as well as ‘public/private collaborations’, all aim to serve the essential needs of manufacturers to speed up the process overall.
Read the full article on “Targeted therapies: speed and collaboration” to find out what makes public/private collaborations so powerful and how the largest collaborations of its type might serve as a model for other huge projects in the future.
Read the full 5-page article here: http://bit.ly/1q0wmaT

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Article on “Targeted therapies: speed and collaboration”

  1. 1. Targeted therapies: speed and collaboration By Al Tuttle, Editor Regulatory bodies get a lot of negative publicity about their treatment of the companies and institutions they regulate. It is a natural outcome of the regulator/regulated relationship: the governmental bodies either overstep their authority or should not have been given such authority in the first place. However, the regulators offer many positive aspects to drug companies and their collaborators. For example, the FDA and EMA are vast research organizations in their own right. They provide guidance and control, but also have support and innovation as public objectives. In a report about innovation and targeted patient therapies (1), the FDA delineates some of its large array of regulatory supported programs. Aimed at expediting the drug development pathway, the agency noted that many targeted early trials get dramatic results because the patients are selected based on the narrow set of criteria expected to produce the best results. The FDA will collect and collate data to help develop wider trial results quickly so that the end result is a drug that can withstand all regulatory scrutiny. In these cases, the agency wanted to publish ways to get the fastest avenue for larger trials started, bring those to good conclusions quicker and certify results more efficiently. Using these proposals as a foundation, in June, 2013, the FDA published a guidance for “expedited programs for serious conditions.” (2) They are aimed at answering several important questions about expedited trials: • • • “Can FDA, drug developers, and investigators agree on a threshold to determine when a treatment poses ‘exceptional promise’ and should thus be treated in an expedited fashion? Can seamless drug development programs be created to utilize natural history data or adaptive trial design concepts to compress drug development time? What are the ethical issues involved in identifying a promising intervention? How should the needs of all patients with the disease be balanced against the need for better therapy for an individual? Can surrogate outcome measures that could be used for accelerated approval be rapidly identified? ---------------------------------------------------------------------------------------------------------------------------IQPC GmbH | Friedrichstr. 94 | D-10117 Berlin, Germany t: +49 (0) 30 2091 3 274 | f: +49 (0) 30 2091 3263 | e: eq@iqpc.de | w: www.iqpc.de Visit IQPC for a portfolio of topic-related events, congresses, seminars and conferences: www.iqpc.de
  2. 2. • Can we arrive at a consensus view of the goal of monitoring commitments companies will make once a product is on the market after such a development program, such as scientific expectations and timelines?” Four to fast track The guidance lists these four programs for expediting approvals: fast track designation, breakthrough therapy designation, accelerated approval, and priority review designation. All are aimed at putting the most critical medicines at the top of the list and hastening the process through channels at all levels. A fast track designation the designation of a drug as a fast track product “if it is intended, whether alone or in combination with one or more other drugs, for the treatment of a serious or life-threatening disease or condition, and it demonstrates the potential to address unmet medical needs for such a disease or condition.” The difference between a fast track designation and a breakthrough therapy designation are few, but is generally used when there is a drug therapy in place which the new therapy would be substantially more effective. Photo courtesy npr.org Accelerated approval can start when a combination of factors has been reached, particularly advanced trials in which the therapy has proven substantially better than an existing drug. The manufacturer is usually required to extend trials beyond the approval, to continue finding favorable results. “A priority review designation means FDA’s goal is to take action on the marketing application 806 within 6 months (compared to 10 months under standard review).” There are several qualifications for priority review that generally mirror those for the other three designations, but review is done at the latest phases of trials. Collaboration and speed Let’s look at the effect of regulations and the accelerated discovery processes on collaboration. Everyone agrees that one of the essential needs of manufacturers is speeding up the process overall. However, collaborations can shorten the time by ---------------------------------------------------------------------------------------------------------------------------IQPC GmbH | Friedrichstr. 94 | D-10117 Berlin, Germany t: +49 (0) 30 2091 3 274 | f: +49 (0) 30 2091 3263 | e: eq@iqpc.de | w: www.iqpc.de Visit IQPC for a portfolio of topic-related events, congresses, seminars and conferences: www.iqpc.de

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