By Andrew MacGarvey

One question I am asked frequently in my role as commercial director of a CRO is, “How do you see the sector changing?” This question has become particularly relevant over the last 18 months as the economic environment has tightened up. As attention has turned to the cost of healthcare around the world, the need for a change in approach has become stronger.

I have always had a great interest in data and how it is collected and handled; my career has included roles in statistical programming, database design, systems implementation, and electronic data capture (EDC). Anyone who works in these disciplines knows that as technology has advanced, more data has become available from increasingly diverse sources. The technical component of the role of a Clinical Data Manager has increased greatly during the last 10 years and is set to continue to do so. For complex studies, the planning required to bring the data together in a reportable state is a big job, but in assessing the risks of the overall study, teams spend much less time examining this area than say, patient recruitment rates. That may be absolutely correct in the current development model; after all, in terms of budget overrun, the biggest risk might be slow recruitment, and so much effort is devoted to protecting against it. The biometrics portion of the project is relegated down the agenda, and there may be some talk of “discussing the back end services further down the line.” This may well be about to change.

The Need For A Change
I was lucky to see Steven Burrill (see the “What’s Next?” article in the June issue of Life Science Leader online) present his view on the life sciences sector earlier this year, and his vision inspired me to do some further research on what is happening in the sector at a macroeconomic level. What are governments looking to achieve? Where is money being spent in healthcare? How will the market collapse of 2008 affect the industry? I did this because Burrill outlined his vision of the R&D environment over a 10-year horizon. His view: the development time for new drugs will drop from around 10 years to about 2 years. Most of the audience probably felt such a change was impossible until he went on to outline his thinking.

For those of you not familiar with Steven Burrill, he is the CEO of Burrill and Company, which is heavily involved in the funding of life sciences companies and corporate activities (e.g. M&As) in the sector. The company also publishes a great deal of research on the life sciences sector.

All change needs a driver. In the case of the current healthcare model, this driver is an economic imperative. The world cannot afford to treat everyone; the current system does not function now, and yet the number of new drugs approved is slowing down each year, and the population is growing. Even if it was felt that things were OK now, the model is not scalable in its current form. This problem is illustrated very well by the healthcare system in the United States, and not surprisingly, reform is very high on President Obama’s agenda. Influential stakeholders are also crucial to successful change. Obama is certainly influential and has the energy that comes with a new appointment to drive transformation. He addresses the problem at a very good time. The 2008 economic “correction” (which is ongoing) has dramatically affected the pharma/biotech companies and changed the way their business is viewed. With diminishing pipelines and increasing R&D costs, pharma had considered partnering with or acquiring biotechs as a way to boost product pipelines, and in fact examples of this are seen on a regular basis, including some high profile acquisitions this year. The problem going forward is that with a tightening credit market, a very high proportion of biotechs will fail over the coming months. This is bound to have some effect on the pipeline and will undoubtedly affect the rate of joint ventures and acquisitions over at least the next 12 months.

As pharma companies (and the large biotechs) seek to improve margins, the next option is cost reduction. One way this can be achieved is through merger (another way of boosting individual portfolios, but of course not increasing the overall pipeline), and the current activity in terms of mergers will continue. The problem is that much work already has been done in terms of cost reduction; a lot of efficiency gains already have been taken. Current activities will not get the sector where it needs to be.

A Shift To Wellness Management
So, there is political will in a major market (other nations are facing the same problem — think about the job the National Institute for Health and Clinical Excellence does in the United Kingdom) and economic conditions having a real effect on the sector and the model for R&D. These pressures on their own might be enough to force change, but probably not a reduction from 10 years to 2 years for approvals. Burrill thinks he can see a third strand, and I think this might be the most important factor. His belief is that there will be a shift away from “sickness management” to “wellness management,” and it is this that will reshape R&D.

Burrill says the paradigm will move from doctors reacting to illness and fixing the problem, to you spending more money on preventing illness and maintaining your health. In this new world, you go to the supermarket to do the weekly shopping and have several diagnostic checks run as you enter the store. While you are shopping, the results will be produced along with any necessary prescriptions. As you complete your shopping, you pick up your medication (for both prevention and cure), and your electronic health record is updated. You may or may not be referred for secondary care. The point is that blood pressure, cholesterol, insulin, and others can be tested and managed in this way, and as more sophisticated diagnostics become available, increasingly complex testing and treatment regimens can be deployed. This world is actually not very hard to imagine; we are seeing in-store treatment rooms appearing already and not just in traditional pharmacies and healthcare stores. If you think the leap to seeing this in supermarkets is far-fetched, then take a look at www.walmart.com/clinics to see some of the conditions they can deal with. Walmart is also investing significantly in the electronic health records market working with Dell to provide a cost-effective solution. In other words, the shift has started. The industry is taking notice too; J&J was very close to establishing a “wellness and disease-prevention” business unit, but has now announced it will be folded into its consumer business. Anticipated revenues? $20 billion a year.

The Biggest Obstacle: A Cultural Shift
This whole approach requires patient-centric systems and thinking. This means a cultural shift, known to be one of the most difficult transformations to effect. Again, much work has already been done, and the emergence of social networking sites has played a significant role. The age of “privacy” may well be past. Social networking sites have encouraged people from all over the world, from every demographic, to disclose all manner of information (in many cases, to anyone who cares to read it). There is some way to go, but the evidence is that a shift from withholding personal information to publicizing it is well under way with the associated “comfort level” growing all the time. In researching this article, I looked at Google Health for the first time. If you want to see one way to manage wellness (and indeed sickness), visit www.google.com/health.

This online resource allowed me to log details related to my demographics, medical history, and so on. I could also import my electronic health records, prescription records, and lab tests from participating sites. If I had paper records, the site would have linked me to service companies that can convert them to electronic records. I found the terms and conditions very interesting, particularly the clause:

“I hereby authorize Google to share the health information contained in my Google Health profile(s) in its entirety, to only those entities and individuals I designate, for the purpose of providing me with medical care and for the purpose of sharing my information with others that I choose.”

Google will be using my information to provide me with medical care! So, our corporations are working away to make this new paradigm a reality; there are many other large retailers and internet companies aiming for market share. All of this should be good for us, the patients. A wellness management approach detects conditions early, and hence the level of reversibility is high; the current, reactive model has lower reversibility. It seems like everyone wins. Burrill notes that if you wonder if populations will be willing to pay for more expensive diagnostics and for managing their wellness, you only have to look at what they will pay for nutraceuticals. A recent report in Nutraceuticals World quoted Global Industry Analysts (GIA) whose projections suggest that sales in the global nutraceutical industry will reach $187 billion by 2010. I think this is a valid indicator. After all, the money spent here is spent to maintain or improve well-being.

An Increased Focus On Health Data
How does this all lead to an increasing focus on data? The answer is in what becomes of “sickness management” in the new paradigm, once you are all dealing with your own “wellness management.”

With the onset of an environment of prediction, pre-emption, and personalization, the clinical study process is set to fundamentally change. With drugs being targeted to specific populations, the importance of in-silico modeling will increase and become more widely accepted. Programs will then run more quickly, and the FDA and other regulators will approve drugs much earlier once a response in the target population has been proven. It is because of this that Burrill sees the clinical phase reducing in time. Given the economics, it is clear that this will be very acceptable to healthcare providers, as the cost of development will be dramatically reduced, allowing for cheaper drugs. The win for pharma/biotech (and Burrill sees most of these treatments being from biotech) is one of improved cash flow with revenues coming far earlier than they do now. If patent protection rulings are static, then the negative effect on those revenues by generics or biosimilars is reduced by virtue of prolonged exclusivity. It is fairly easy to see how all of the stakeholders could be satisfied by this model. However, the key to all of this working is the design of the shorter clinical development program; that is where the skill sets of biometricians come to the fore.

In the new model, the importance of intelligent design, conduct, and reporting of studies will be crucial. The regulators will require increasing amounts of data, which in turn will be more complex and come from more sources. How biometricians collect and use this data will be vital to a successful outcome, and expertise in the biometrics arena will become a focal point. Attention will shift from patient recruitment issues to data issues. It is in biometrics (and pharmacovigilance) that the risks will be weighed.

Is it realistic to bring down the time to market of new drugs even allowing for targeted therapies? I wonder if a parallel can be drawn with off-label use of drugs. With sophisticated in-silico modeling providing credible information with respect to how the drug might act and intelligently designed trials gathering extended volumes of useful data, the regulators may well be comfortable approving drugs into targeted populations. In 2008, a report in Nature Biotechnology estimated that “off-label use of cancer drugs runs from 50%-70% of total usage, and perhaps higher.” In critical care, there is always additional pressure to get new treatments to the patient. When sickness management is addressed in 10 years, if the technology in diagnostics has advanced sufficiently and more is known about how the drug will affect its population, the pressure to get new treatment to the patient will only increase. With the economic and demographic challenges ahead, the industry may have to rely on the benefit from a new approach. They can focus on accelerating the delivery of targeted therapies to specific populations. The main concern surrounding targeted medicine has been the cost — can a return on investment be made when the market is limited? Under a model where sales begin at the two- or three-year point and where preclinical work also has been accelerated, it may be possible.

The data has always been important. My view is that more attention will be placed on mitigating the risk of bad data in our studies going forward, and this can only be a good thing, not just for the biometrics profession, but for development programs as a whole.

This article was first published in the Association for Clinical Data Management newsletter.

About the Author
Andrew MacGarvey is the president of Quanticate Inc., a global CRO delivering scalable clinical data management, statistical programming, statistical analysis, medical writing solutions, and consultancy across all these services. He can be reached at
andrew. This email address is being protected from spambots. You need JavaScript enabled to view it. .