In the past the pharmaceutical companies have always been anti-cyclic businesses and were not affected by the “wows” of the global or local markets. This has changed since 2008 with the start of the financial crisis. Although some say that sufficient money is still available for start-ups and R&D, those in the business know better. But if we have a closer look at the situation, one can see there is more than the financial crisis causing the current problems.
While the dust of the explosion of regulations is starting to settle, currently most guidelines and directives are in place, society is faced with a system crisis in the pharmaceutical industry. The empty pipelines of big pharma urged them to rethink their business model. Some say the easy grapes have been picked. Others think the new computer techniques for candidate drug discovery have failed. This has been labeled as another factor in the ever rising costs for drug development. However, not only the overregulated environment or the high attrition rate is responsible for the high costs.
Certainly, the development over the last twenty years from an ill regulated environment, where statisticians were doing everything from data entry to reporting, to an over regulated situation we have to date, attributed to this price rise. And yes of course the increasing prices are hindering the development of new medicines. Big pharma is more reluctant than ever to finance risky, albeit promising projects. Instead they leave the high risk work more and more to the small biotech, which being funded by private investors are more inclined to take risks.
From a societal perspective there is still a demand for new medicines. In many areas, and certainly in oncology, there is an unmet medical need for better or simply non-existing medicines. How to produce and develop these medicines considering the continually staggering development costs?
A glimpse of a solution can be read between the lines in the draft guideline on ‘Risk based monitoring’ that has been issued for review by the American FDA. This guideline is a clear break with the past which was always characterized by increasing work or scrutiny of the data, which consequently pushed the prices for clinical drug development to the top. The key element of this guideline is the risk based approach for monitoring, meaning that companies no longer have to do a 100% source data verification (SDV) but are allowed to a SDV guided by a risk based approach, which could mean e.g. that non-pivotal data might be handled without complete SDV. In general, SDV and monitoring encompasses a large portion of the operational costs of a clinical trial. Typically the clinical research associates (CRAs) have to travel to the sites and check all CRF data and the site files.
Another trend, although progressing at a slower pace than previously anticipated, is electronic data capture (eDC). The combination of eDC and risk based monitoring could really become an answer to the cost question. Less travel, less SDV would have a clear impact. eDC allows the CRA to do a lot of checking with the aid of a PC or laptop from their home or office, or at least without the necessity to travel to the sites. Some frontline companies already started to speak of a data coordinator, as a hybrid between a data manager (DM) and a CRA. Logically, it seems more and more likely that both functions (DM and CRA) will merge, up to the stage that they are being done by one and the same person.
The future is to the data, that is the pivotal data, at the expense of less critical data. Risk based monitoring combined with eDC deserves our serious attention as a potential way to reduce drug development costs and provide better and cheaper medicines to the world.