There were numerous articles in March 2017 that covered a wide range of topical issues. Here are five of the best stories you might have missed… (click the headline to finish reading the story)
Whilst the UK has been a world leader in the field of medical research for many years, it became apparent post year 2000 that a change was underway. Central and Eastern Europe (CEE) were developing as attractive centres for clinical research, with a cost base in the order of 30-40 percent of UK costs with rapid patient recruitment. The questions and fears regarding data quality were soon allayed by an extensive audit both by pharmaceutical sponsor companies and regulatory authorities. So, in this region we had recruitment options that were difficult to match in either the UK or Western Europe. Companies (both pharmaceutical and CROs) recognised that they needed some western countries for their key opinion leaders and to show that data generated in the CEE region was of equivalence to that from Western Europe. However, because of cost and time reason, the UK became side-lined predominantly by Germany, Belgium, Italy, Spain and The Netherlands, who whilst they did not have the patient recruitment rates of the CEE region were cheaper with good data quality.
If you have a razor-sharp ear, and you live in Massachusetts and California, on March 10, you may have heard what sounded a lot like a collective sigh of relief. For all the talk about a potential shake-up of the FDA, with President Donald J. Trump nominating Scott Gottlieb to lead the FDA, the president has selected a candidate who appeases the establishment.
For states like Massachusetts and California in particular, two notable pharma and biotech hubs, the consensus appears to be one of approval as Gottlieb is largely viewed as one of the industry’s own.
In 1996, I was hired as a clinical research project manager (CPM) in a full-service clinical research organization known for recruiting young professionals (they’re less expensive). It was expected that I would be working directly with clients, regulatory agencies, statisticians, analytical chemists, FDA auditors and clinical-trial personnel. I was also trained continuously on the job by our supervisors in the intricacies of pharmacokinetics.
I found the work to be varied, scientifically and operationally interesting and an excellent way to learn how to run a clinical trial from contract signature to delivery of the final report. In fact, delivery usually meant going through the satisfying experience of physically packing and carrying the heavy boxes of data-laden binders for pickup by courier (and if we missed the deadline, sometimes delivering these boxes by car to the courier ourselves).
OpenTrials could be the best thing to happen to obtain accessible, usable results since FDAAA 2007 section 801 required the expansion of clinicaltrials.gov to include results.
It’s now just over one year since Ben Goldacre’s enthusiastic rollout of OpenTrials. OpenTrials launched their beta in October 2016. Targeted searching reveals placeholders for much information, such as protocols, clinical study reports and results. Where these items have already been located, the links through to them work neatly. For instance, links from a trial record to the EudraCT results page. What appeals to me though is the potential for linking to usable and detailed clinical trial results from this accessible platform. However, the way in which OpenTrials will enable access to results beyond those available through clinicaltrials.gov and EudraCT have not yet been defined.
G-BA Early Advice: How do we prepare for the requirements that the Early Benefit Assessment in Germany poses for Clinical Trials?
The German AMNOG law (roughly translated as the Pharmaceuticals Market Reorganization Act) has been in place since 2011, and introduced the early benefit assessment. This meant that in addition to regulatory requirements (efficacy, safety and pharmaceutical quality) which an authorized medicine has to meet, it also needs to have a patient-relevant added benefit.
Unlike in other systems, early benefit assessment does not represent a “fourth hurdle.” New medicines are reimbursed as soon as they are available on the market. However, the AMNOG kicks off as soon as the medicine is authorized and available. After one year, there is a negotiated reimbursement price and the added benefit is the basis for price negotiations.
PHOTO CREDIT: Rob Nguyen