Industry focus on niche drugs means nothing in pipeline for major diseases

By Nicola Garrett

14 Aug 2019

The focus on rare diseases by the pharmaceutical industry is a dangerous trend that is stifling innovation and neglects major causes of death and disability such as cardiovascular disease, the President of the European Society of Cardiology has warned.

Speaking at the Cardiac Society of Australia and NZ meeting in Adelaide, Professor Babara Casadei who is also a British Heart Foundation Professor and Honorary Consultant Cardiologist at the John Radcliffe Hospital in Oxford, UK, told delegates there was a common misconception that cardiovascular disease had been conquered. 

“We have witnessed a dramatic reduction in cardiovascular deaths over the last 50 years but there is this idea that the job is mostly done, which is clearly not the case. Cardiovascular disease is still the number one killer in Europe and the projection in the next 25 years is that this will still be the case,” she told delegates.

The other problem is that while there are many people with cardiovascular disease, the number of people who will benefit from novel treatments being developed by industry is going to be modest.

“The industry is answering to this by saying that the trials are too expensive, expected benefits are of moderate size and payers are reluctant to fund new drugs because many people would be eligible to use them,” she said.

This business mindset is driving industry focus towards costly treatments for rare diseases where large trials are not needed and profits are high, and away from treatments for the major causes of morbidity and mortality, like cardiovascular disease, Prof. Casadei explained.  

In the year 2000 the top 10 drugs in the United States had a revenue of $34 billion and benefited 413 million patients. In 2015 revenue was $84 billion but only 54 million patients benefited.

“This represents a 19-fold increase in cost per patient … this is a major problem because the pipeline for cardiovascular disease is really dry and this is a very obvious, particularly if you compare it to other diseases such as cancer,” Professor Casadei told the audience.

“This is a situation that is extremely bad for cardiovascular disease or any other commonly acquired disease,” she said.  

According to Professor Casadei the reaction to this “dangerous trend” has been to “bark up the wrong tree” by looking at observational data because it is cheaper and quicker than gold standard RCTs.

“Observational  data has its merit and use but it is not suitable for the actual efficacy of treatment, you still need randomisation,” she said.

The European Cardiology Society has made moves to tackle the issue by focusing and investing in developing and educating people in using and building platforms for pragmatic streamlined clinical trials that use local data.

“One of the most amazing benefits of doing [research] this way is really in the efficient recruitment of patients, there are enormous costs in going through several centres to identify patients to recruit to a trial, this way you can do it electronically,” she explained. 

Other benefits included the effective assessment of safety and efficacy using routine data and complete and comprehensive follow-up.

“The results are the same [as a traditional RCT] but the price isn’t so you can run the same trial, better, more efficiently  at a fraction of the price,” she added.  

“This is an intervention that as a society we felt we needed to actively promote because it is going to be impactful in the progress of cardiovascular medicine and our ability to test new treatments and new devices.”

“We need to look at electronic health records not just as headaches but as infrastructures that can bring wealth and innovation to individual countries.”

One such initiative is the EUROHEART project which aims to develop continuous quality of care based on individual patient data. 

“We would like you to consider thinking about doing something similar and compare your data with European data to find together the solution,” she concluded. 

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