Revisions to Brazil's drug price framework are coming - what's included and what's missing? Our analysts provide th… https://t.co/8s17gnKCXf
Ebola – What's the Pipeline?
As the 2014 West Africa Ebola virus outbreak claims its thousandth victim, many questions are being asked over how the disease can be contained, and if not contained, then how best to treat it. The options, as those who are even vaguely familiar with this latest outbreak will know, are very limited. At best, afflicted patients can expect supportive care. The consequences, of course, are dire. Ebola has one of the highest mortality rates of all viral diseases and the latest outbreak has spread across a series of countries - and could spread further afield.
There are plenty of viral diseases that have seen successful treatments, vaccines and containment strategies emerge - HIV/AIDS, smallpox...and most recently we have seen a de facto cure for Hepatitis C come to the market. However, with Ebola, the pharmaceutical industry is being confronted with a familiar tale of criticism over its "moral bankruptcy" - it stands accused of ignoring a neglected disease, a disease which primarily affects poorer countries.
And indeed the research pipeline in Ebola is fairly frugal - there are no clinical trials that are currently registered as ongoing in clinicaltrials.gov, the pre-eminent source for registered clinical trials. And almost all research is at the pre-clinical or Phase I stage, with no major pharmaceutical companies involved; only smaller biotechs, universities and research institutes are conducting the trials.
So far, the recent outbreak has placed a significant amount of attention on Mapp Biopharmaceutical's ZMapp, a preclinical monoclonal antibody (mAb) that is derived from injecting mice with Ebola antigen and produced through tobacco plants. Although human testing was only due to commence at the end of this year, the drug has been given to some foreign aid workers operating in Liberia who had been infected with the virus - and Mapp has indicated that its supply has already been exhausted.
The other main candidate is Tekmira Pharmaceutical's RNAi-based therapeutic TKM-Ebola. The vaccine has received fast-track designation from the US FDA, but its Phase I trial in healthy adults was placed on clinical hold in early July 2014. This was just before the current outbreak, and the clinical hold was modified to a partial hold this month in order to ensure that the product can be used to mitigate the spread of the disease.
Both of these treatments are highly experimental - the technology behind them is advanced and largely unverified, and their safety and efficacy in humans is essentially unknown.
Interestingly, the research behind TKM-Ebola and ZMapp is partly-Canadian-funded, and the Canadian government has stepped in and sought to guarantee broader supplies of the products. ZMapp has been given at no cost to the patients who have received it so far, and the Canadian government is aiming to donate its supplies.
That governments and international organisations are allowing patients to be treated with them - and thus side-stepping a flurry of regulations around clinical testing - highlights the severity of the situation. The World Health Organisation (WHO) has eventually concluded that it is "ethical" to provide unlicensed treatments such as ZMapp and TKM-Ebola.
Predictably, however, Mapp has been criticised for providing ZMapp to Western patients at the expense of patients who are in the eye of the storm in West Africa. Conversely, if patients in West Africa had received the drug, criticisms over Western pharma companies testing their unlicensed drugs in African populations would likely have arisen. Ultimately, in this situation, pharma companies are generally damned if they do and damned if they don't.
And this partly explains why larger pharma companies are not involved in research in this area - but also it is symptomatic of the modern global pharmaceutical industry, which is less involved in R&D and more focused on commercialisation and production. Most of the innovative R&D, across most therapeutic areas, happens in smaller biotechs like Mapp and Tekmira, as these companies have proven themselves to be most adept at bringing early science into clinical trials. Once the technology is validated, larger pharmaceutical companies come in because they are able to design and fund more complicated, larger clinical trials, and subsequently go through the approval and market access process.
Ebola happens to be a particularly difficult case because outbreaks are so infrequent, affecting comparatively small populations, hence making the normal clinical development programme very fragmented and difficult to manage. Plus, it is a volatile disease, and only highly innovative, unproven technologies are proving to be effective (at least in theory) - this only serves to further stretch the development timeline. The latest outbreak hopefully reframes the discussion and will lead to further impetus for development of treatments and vaccines against Ebola - one of the main problems for Tekmira has been to secure funding for its R&D after the US Defense Department asked them to stop working on the candidate in 2012. The "stop-start" nature development programme creates huge uncertainty, and can prevent us from finding the cure we need.
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Major changes to Czech P&R regulations for orphan and high-cost drugs set to expand access. What role will IRP play… https://t.co/bQcVCG5jut