The value of clinical trials

What is supposed to be an objective scientific process designed to assess whether product work, whether they are better than products already on the market and whether their side effects are a price worth paying for any benefits they might convey s routinely being corrupted.  Companies bury clinical trials which show bad results and publish only those that show a benefit.  Trials are often run on small numbers of unrepresentative patients, and the statistical analyses are massaged to give as rosy a picture as possible. Entire clinical trials are run not as trials at all, but as under-the-counter advertising campaigns. That is a scandal.

Economist, reviewing Ben Goldacre’s new book.

This was my jumping-point for a talk on Clinical Trials Strategy that I gave to an industry audience at Medtec Ireland this week.

My first point was a straightforward solution: Clinical trials must inform and conform.  To inform, trials must be designed to answer a significant question honestly, with the possibility of a negative result.  To conform, trials must be executed using hypothesis-driven scientific methods, following rules to assure patient safety and privacy.

My second point was strategic: Recognize that clinical trial results serve many other audiences.  Publication in peer reviewed literature is obvious.  But regulators, payers, professional clinical associations, industry risk / usability standards,  and investors also have particular needs for information and evidence.  If the planning process doesn’t take account of them, then trials may need to be repeated.  As our Business Development director reminds me, 10% more investment broadening a trial's data collection strategy returns 30x savings later when the right information is available as needed.

I’m in Washington DC now for the Anesthesiology Society of America meeting. We’re presenting five abstracts (here’s one) describing the results of clinical studies that demonstrate safety and baseline characteristics of a new surgical monitoring device in normal subjects. 

The first two day’s experiences has really driven home both points.

These are High Science presentations: hypothesis-driven, rigorous methodology, direct comparison to the  commercial gold standard, formal registration, peer-reviewed publication.  These studies establish facts and promote open discussion, first through formal presentation and then through hours of informal walk-by discussions with clinicians. Our next set of results, accepted for December, will move to clinical experiences describing efficacy and usability.

But more broadly, the presentations also connected with multiple audiences.  We brought a prototype of the monitoring device that illustrated the likely instantiation of the idea.  Doctor who understood the need and the evidence could comment directly on the product.   They told friends; friends talked but it on the trade floor.  Industry rep’s dropped through to inquire and to  fret.  We were featured in n article in the ASA Daily News (p.3).

We hoped for a few hours of comment by our medical advisory group.  We got very full 12-hour days of intense discussion by a diverse audience of people from academics, clinical practice, and industry.  The prototype got a month of shakeout in days.

To be honest, the momentum built so fast that I got a bit worried.  Was it being oversold?  Could we meet expectations for performance and delivery time?  Was awareness building too quickly among competitors?  Were we losing control of the process?

There’s no question of the value that connecting with customers through clinical trials  has brought to the process of developing the product and the business.   Context, credibility, awareness, and access have all accelerated; optimism and enthusiasm have infused the founding team.  It’s driving late night discussions over nibbled tapas and glowing tablets about planning and funding.  It’s exciting.

Are we losing sight of the simple maxims that I advocated in Ireland just days before?  I think it’s really fortunate that I presented on these issues immediately before the conference: so far I think we’re wholly on the side of the angels and headed in directions that will both inform and conform. 

In directions that should, ultimately, benefit patients and the business.