Tuesday, December 17, 2019

A Year of Too Little Too Late

Looking back on 2019, it has been a year of climate change for antibiotics. If you are easily depressed – maybe you don’t want to continue reading this “grim-gram.”

The regulators are working hard to come to grips with how to manage small data sets.  This is especially a problem for the FDA where “adequate and well controlled” is the law. Have we made progress here? I certainly don’t know. But without progress, pathogen specific products, microbiome products and others will never make it to market. 

Of course, even if these products do make it, will they be welcomed? The bankruptcy of Achaogen either woke us from sleep, deepened our nightmare or demonstrated that the market works – or maybe all of the above.  Depending on your point of view, no good deed goes unpunished or Achaogen got what it deserved (see this blog). Regardless of your viewpoint here, it is clear that several other biotechs will face Achaogen’s fate in 2020 baring some miracle. 

And what miracle might that be? There is the UK’s effort to provide value-based pricing with a pre-purchase of needed new antibiotic(s).  This will be based on the UKs perceived need in terms of patients requiring such treatment over time. But there are not enough such patients to actually make a market and the UK’s contribution to the market will not be enough to provide a return on investment for companies brining needed new therapies forward. What if every country did what the UK is doing or at least something similar such as the DISARM act?  (DISARM would provide for payment for high priced antibiotics for US patients in need).  But even if this became a global effort, there are still not enough such patients to support more than one or two products. These are baby steps, not miracles. As I keep saying, we need substantial pull incentives to “pull” this off. 

The view of various countries seems to be that they somehow should only pay their share based on their needs.  But we know that will not be sufficient – so what’s the point? What we need here is true leadership and sacrifice.  We need a country or region (think Europe or even North America) to provide the entire pull incentive for the world – and then bring in the followers through negotiation later. Unfortunately, like the situation with climate change, I see no such leadership emerging. 

And these considerations bring me to the issue of support for research and development. Why, I ask, are we doing this? Why do we support research designed to bring products for extremely limited markets forward when we know that without large pull incentives plus a regulatory path forward, such products and their sponsors are doomed? I have posed this question to funders.  They reply with hope for (and a great deal of work to achieve) a better future. How long should we continue along this path? Private funders are voting with their feet. Venture capital and public market investors alike are abandoning antibiotics faster than you can shake a dollar at them. In the absence of sufficient pull incentives in the near future, collapse of these small companies and the pipeline of products they support is inevitable. 

As with climate change, we as a global society (is there such a thing?) seem unable to believe existing data and invest now to provide a better tomorrow. We prefer to close our eyes and ears, stick our head in the sand and await either an apocalyptic event or the slow arrival of the inevitable consequences of our inaction. 

In the past, this would be the point where I would exhort you to be calling your political representatives to push pull incentives. But my experience over the past year convinces me that there is nobody home. Politicians are all focused on reducing health care costs and drug prices and spending additional monies now to save money in the future is not part of their thought process. Certainly none are interested in providing the kind of global leadership we need. 

One way forward, although, again, this might be too little too late, would be to establish a entirely publicly funded antibiotic R&D institute with sufficient funding to bring needed products to market and to support them once they are approved.  This would not be a small investment, but it would occur over time. Politicians do seem interested in supporting R&D. While I have never been enthusiastic about this sort of government approach to antibiotic R&D given their track record, it may be that we have little choice at this point. 

1 comment:

  1. I think the publicly funded Institute would be great - if a good group of scientists could be recruited to work there. I do, however, question your global warming analogy - as I believe most of the globe agrees about those facts - excepting our President [and his fossil fuel henchmen].