Easy Yes: the value of Early Health Technology Assessment

 Sir Andrew Dillon 

The ASSESS Project Advisor

It’s been estimated that only 32% of biotherapy candidates leaving universities make it to clinical trials and that less than 10% of biotherapies that enter clinical trials make it to market. If these figures are anywhere near correct, we can be confident that over 90% of biotherapies licensed out by universities, never get to change patient outcomes.  

Each failure represents waste of time and effort: in laboratories doing the pre-clinical research, of the patients’ time and effort recruited to the clinical trials, of the expertise and infrastructure required to design and deliver those trials, of regulatory and health technology assessment (HTA) resources, of manufacturing capacity; as well as  a waste of financial investment.  

Given this significant failure rate, it would be good to think that the therapies that do make it are game changers for patients. Some of them are but unfortunately, too many are not. Frequently, HTA agencies search hard to identify the patients in whom the benefit justifies the price. NICE, for example, has had to optimize (in other words, recommend only part of) the clinical indication for over 26% of the appraisals it has conducted since March 2000. It has had to reject outright another 15%. We know that many of the positive recommendations were only achieved through aggressive price negotiations. HTA agencies rarely consider a technology that is an easy yes. 

Better is possible.  Picking winners is challenging, but by considering the potential for a technology to meet the objective standards of value that matter to patients, clinicians and the HTA agencies that act of their behalf, at an early stage in their development, we can help weed out technologies that are unlikely to offer enough benefit to warrant further development. The more these low- or no-value technologies are removed from the investment pool, we will reduce the waste (of people, time and money) involved in attempting to push them into health care systems. HTA agencies are enablers and not barriers to effective new technologies getting to patients. They want to be able to move quickly and confidently to positive recommendations. But for this to happen, they need to be offered robust evidence of value to patients and to the health system. Technologies that are presented for adoption with weak evidence or little potential to improve outcomes simply clog up decision-making processes.  

Early health technology assessment can make a difference.  By identifying the points in the development sequences of new technologies, at which information and opinion from experienced technology assessors can enable decisions that separate technologies with real potential value from those don’t, early HTA can help spot investments that are unlikely to offer enough value to justify their development costs. Real savings can be made. For example, before universities offer technologies to investors, in a single jurisdiction of interest they spend between $10,000 and $20,000 for a patent application and a further $10,000 to $15,000 to prosecute the patent.  Most of those patented technologies do not make it to market and therefore the investment in patent protection is wasted. An early Health Technology Assessment process, to guide tech transfer investment decision-making, could pay for itself within months, by eliminating patent fees for no to low value technologies.  

These are the direct financial savings to the Technology Transfer Offices. But early intervention in the technology transfer process produces benefits to all actors in the commercialization process, as the costs avoided accumulate along the development process. Conversely, technologies with an independently verified potential to meet health systems’ value criteria are both lower risk and higher return investment opportunities.  

Better is possible with The ASSESS Project, which connects university technology transfer offices to experts in early health technology assessment, to offer critical, objective insights into technologies’ clinical and commercial potential, as well as characterizing the time and cost required to generate the evidence to bring the product to market.  The project will help with early identification of technologies that should be a ‘no’, and thereby increase the focus of R&D resources on those with real potential to be the desired “easy yes.”