Ask the expert: the clinical trials questionnaire

AL. We always need to keep questioning processes, to continually see how we can do better. It is true to say that those with an innovative mindset will, generally, always be willing to accept innovations and those who do not, will not. As with all new things there should be a compelling reason to introduce something new – after all a smoothly running and efficient older system may actually do a better job than a new, buggy system that requires continual maintenance and “tweaking”.

In many ways embracing a new approach is like volunteering for a phase I study – those following are more likely to reap the benefits! So, in order to benefit the innovator has to stay the course and see the new approach through to its conclusion and not give up halfway through. A difficult thing sometimes and like most things, it requires a degree of courage and belief in the new process.

NGP. Biomarkers can be used to track the efficacy of a drug and, according to experts, shorten clinical trials. Is this an opportunity to save time and cost?

AL. Not necessarily. To be useful, biomarkers have to be developed and validated so that they become acceptable by the mainstream including Regulatory Authorities. Physicians (and agencies) will always wish to see the demonstrated connection between the biomarker and a clinical benefit and/or outcome. Until this happens the value of (new) biomarkers can always be questioned.

The development of a biomarker is not necessarily cheap and implementing it in a clinical study can also be expensive – especially where the testing has to be centralised and the shipping costs have to be considered.

Nevertheless, new biomarkers will help us understand diseases better and, as a consequence, better understand their treatment and help follow the course of the disease – and ultimately be of benefit.

NGP. British scientists have warned that an impending government decision that may ban stem cell research using animal eggs will jeopardize finding treatment and cures for degenerative diseases. Does this affect your company?

AL. Research should always be managed in a highly ethical, moral and legal way. As a CRO, we would, perhaps, not be directly affected by such a decision but as a company committed to helping innovative life science companies develop today’s new medical concepts into quality medical treatments of tomorrow we would be disappointed if we could not participate in the development of new ground breaking therapeutic options because of an unnecessarily restrictive regulatory environment.

NGP. How does Hesperion save costs? And what other benefits do you offer to your customers?

AL. As a mid-sized CRO, Hesperion has the necessary geographic spread and the tools in place to run most clinical studies. It focuses on establishing a partnership style of collaboration with its sponsors, in which joint senior management steering committees re-enforce the bilateral commitment and also ensure the provision of project staffing through longer term planning. Net results can be discount or milestone payment structures for volume.

In addition, continuous project teams ensure high levels of experience and competence, and add to the efficiency of the Hesperion-sponsor teamwork. Moreover, planning at all levels and working together as a team allows to establish the optimal times to start up and execute studies and eliminate, or at least minimise, the steep learning curve that invariably happens when CRO and sponsor first work together.

Other advantages are stable project teams, well-managed budgets and timesavings between and during projects.

NGP. The number of people willing to participate in clinical trials is dropping. Is outsourcing a solution to this problem? Do you think that there are ethical issues involved?

AL. Clinical trial participation is always a personal decision and there are many factors that a person considers before entering a trial. Many of these influences are not necessarily scientific – for example, negative press reports of problems in clinical trials. Outsourcing can be part of the solution – it enables a third part to present the trial rather than the sponsor perhaps limiting bias. It does add resources and potentially allows for a larger population to be canvassed for participation – especially in the case of smaller drug developers.

I do believe that study participants wish to feel that the sponsor has a personal interest in them and does not see them as just part of a production line to get a new drug approved. Participation in a study always involves ethics and the ethics of participation do not change because a sponsor outsourced recruitment – ethical standards should always be maintained at the highest level at all times by everyone concerned.

NGP. People are looking beyond genomics and proteomics into higher-content information. HCS (high-content screening) in cell-based assays is a strong trend for the future. Others see a growing trend in gene expression analysis from the perspective of RNA-focused research. Where do you think the future is taking us?

AL. All these strategies are trying to identify or demonstrate the functional links to disease and providing possible ways to assess (measure, if you like) the status and progress of the disease in question. Being able to better understand the mechanisms operating in a disease can give us a better understanding of where to target our treatments – and in some cases at a very fundamental level of cellular biological functions. Although there are many new strategies being explored at the moment, there are in my opinion no clear leaders, and it would be a mistake to exclude from our consideration any of these techniques new or old. In fact some would say that it is only the integration of genomics, proteomics and DNA profiles that would give exploitable data to identify new drug targets. Therefore, I would not exclude any approach that enhances our level of understanding, which will inevitably expand our treatment options.

Dr Alexander Leigh was born and raised in the UK. Apart from his qualification as medical practitioner, he has a BSc in Psychology and is a Member of the Faculty of Pharmaceutical Medicine. After working in general medicine in the UK NHS hospital service, he joined the pharmaceutical industry in 1985 and has held positions of increasing seniority with Lederle (Gosport, UK), Bristol-Myers Squibb (Brussels, Belgium), Parke-Davis (Freiburg, Germany), Sanofi-Synthélabo and Biogen (Paris, France). Dr Leigh has gained extensive experience in clinical research, however, he has also held more commercial positions and has published a number of papers concerning his work in the anti-infective area. Currently he lives near Freiburg, Germany with his wife and three children.