There are a host of examples where medical breakthroughs have changed the face of a disease – saving thousands if not millions of lives. The process by which these breakthroughs occur is complex and multi-faceted. But if we can get to grips with how they happen, it could help us make similar discoveries for other diseases. In this blog post, Dr Matthew Lam, Communications Manager at the Policy Institute at King’s, draws on the findings of a recent report to examine the variety of factors that contribute to medical breakthroughs.
At the end of August this year a report commissioned by Alzheimer’s Disease International, and led by Professor Martin Prince at King’s College London, revealed that the total estimated worldwide cost of dementia has reached $818 billion. Perhaps even more shocking is the reality that by 2018 it will have become a trillion dollar disease. To put this in perspective, a report in 2011 from Harvard School of Public Health and the World Economic Forum stated that new cases of cancer worldwide cost around $290 billion, and the global cost of cardiovascular disease was estimated to be around $863 billion.
It is quite clear that dementia is rapidly on its way to becoming a health and economic burden that can’t be ignored. As people across the world continue to live longer, it’s now more important than ever that medical research breakthroughs occur for dementia to not only improve treatment of future patients, but to also reduce the number of new cases emerging each year.
A recent publication by PRiSM (Policy Research in Science and Medicine) – a partnership between RAND Europe and the Policy Institute at King’s – has recognised this urgent need and compiled lessons that could be learned from medical breakthroughs for other health conditions. There’s no doubt that investment in innovative science has led to incredible discoveries that have changed the face of human health. But laboratory and clinical research is not the only area that needs consideration. The PRiSM report showcases the importance of social, institutional and technological factors, alongside the science, working in concert to enable medical breakthroughs.
One of the breakthroughs pulled out in the report as an example was the introduction of highly active antiretroviral therapy (HAART) as a treatment for HIV/AIDS. This treatment uses a combination of antiretroviral drugs, each targeting a specific part of the virus, in an attempt to reduce the patients HIV infection as much as possible. The success of HAART is measurable – with mortality rates dropping dramatically since its introduction – and in many countries, transforming HIV into a chronic, manageable disease.
Tackling the science
The success of HAART, as assessed in the PRiSM report, was due to several parallel advances that enabled the research to be translated into a public health benefit. Firstly, scientists from all over the world contributed to major advances in the basic understanding of the biology behind the disease. This ultimately led to the identification of drug targets and the development of early antiretroviral drugs, such as azidothymidine.
There was also a global, coordinated effort in the scientific community to push forward in HIV/AIDS research. Scientists and clinicians with different expertise worked together with leading pharmaceutical companies to turn basic research into a viable treatment. Driven by large amounts of public and industry finances the medical research community were enticed, and able, to make the necessary breakthroughs. This co-ordinated effort also extended beyond the scientific community. Alongside the medical breakthroughs, prevention and education about the disease was also paramount, as well as ensuring that health services were ready to make treatments accessible and affordable.
Innovation was also identified as a key driver of success in the development of HAART. The need for rapid translation of research into therapies, the wide spread epidemic of HIV/AIDS and the variation in effects of both the disease and drugs on different populations meant that bold and innovative steps needed to be taken. For example, the clinical trials for HAART were innovative in the way that patients were engaged with the research. Feedback from patients on their experiences during treatment helped industry develop better dosing regimens and an understanding of drug side effects.
Another important factor in the development of HAART was the flexibility in which the regulatory bodies operated to try and reduce barriers in the drug development pipeline. As a result of regulators fast tracking their standard review processes, new scientific breakthroughs moved through to trials in patients more rapidly. This reduction in regulatory barriers and the clear commitment to spend public money on clinical trial infrastructure made it an attractive prospect for pharmaceutical companies who were able to rapidly develop new, more effective treatments.
One area that can’t be ignored is that of a social movement in HIV/AIDS. During the development of new treatments the AIDS community became more united and committed to helping the cause. The public disclosure and death of high profile celebrities with HIV/AIDS such as Rock Hudson, Liberace, Ryan White, Freddie Mercury and Greg Louganis, kept the media’s focus on the disease for over three decades. Campaigns and advocacy groups for people with HIV/AIDS sprung up all over the world – keeping the disease high on political agendas. Ultimately, this sustained momentum for better care of patients with HIV/AIDS, and meant more was done across all sectors to help develop more effective treatments.
Advances such as these have also been necessary for breakthroughs in other conditions. The PRiSM report highlights case studies in breast cancer, coronary heart disease and Parkinson’s disease that all show similar patterns to the HAART breakthrough for HIV/AIDS. The authors are clear that while they have identified areas of action likely to be important in addressing the dementia challenge, they also raise questions about how these could be implemented in practice and what the immediate next steps would need to be.
What can be taken away from this report is just how much work needs to go into a medical breakthrough. The science is, without a doubt, incredibly important. But without the public, patients, politicians, doctors and industry pulling together to ensure money is available and unnecessary bureaucratic and regulatory barriers are removed, the science can never be turned into that discovery destined for the history books.
Dr Matthew Lam – Communications Manager at the Policy Institute at King’s
This blog by Policy Institute at King’s is licensed under a Creative Commons Attribution-NonCommercial 4.0 International License.