Guest Blogspot: What should we expect from medical and pharmaceutical R&D in the future?

Charities representing patients with different illnesses compete to make the case for research and the availability of the best treatment and care in their areas of interest. The autumn party political conferences probably represent the peak campaigning period.  Many medical charities, healthcare think tanks, official bodies like NICE and other lobby groups take the opportunity to run stalls or are represented at meetings, in an attempt to influence senior politicians and gain publicity. Cancer and neurology are amongst the areas to receive most attention but research into some other illnesses may be just as or more promising.

Future discoveries have not yet been made and therefore we cannot tell exactly what they will be.  However, huge technological advances have been made that should open up important areas for  medical research. If mankind is going to have the best chance of R&D success in the future, we have to manage our R&D effort now. Similar situations are common in many other fields that have nothing to do with medicine. For example, we cannot know the score of future football matches, but a good football manager helps to give us the best chance.

Funding well thought-out medical and pharmaceutical research will always be an excellent use of money. From a scientific standpoint much of the human body can be regarded as a vast number of chemical processes with supporting structures and ways of transporting chemicals to where they are needed. Almost any process in the body could be stopped, slowed down, speeded up or modified by the introduction of new chemicals in the right concentration and in the appropriate place. This concept underlies the discovery of drugs.  The human body is so complex that there will always be vast numbers of drugs awaiting discovery.  The same conclusion can be reached a different way. So long as man is mortal, everyone will eventually die. No matter how much medical progress is made, there will always be new challenges to face.

The idea that mankind is running out of drugs to discover is groundless. In periods of relatively low R&D productivity scientists who work hard without success will understandably say that their task has become harder. A similar situation arose in physics in the late 19th Century. Physics is about energy and forces, which in those days meant heat, light, electricity, magnetism, sound, gravity, mechanics and waves. Many physicists at the beginning of the 20th Century really believed that all the fundamental discoveries in their science had been made. Yet Einstein’s Theory of Relativity (leading to nuclear power and weapons), quantum mechanics, semiconductors (leading to modern computers), radio telescopes (probing the origins of the Universe) and an understanding of radioactivity & subatomic particles were yet to come. History teaches us that scientific advances continue to happen whether or not experts are expecting them. Major medical and pharmaceutical discoveries, like those in other areas of science, have generally involved strong personal drive and dedication and a passion or need to succeed.

Few major pharmaceutical companies existed before the 1950’s. From the 1960’s to the early 1990’s nearly every major pharmaceutical business in the world earned over half of its profit from under six drugs. In many cases companies were dominated by one product. In order to survive in the 1970’s and 1980’s companies had to discover important new drugs to replace those going off patent. They had to succeed and so almost without exception they did. Pharmaceutical companies rarely had profit dips other than when exchange rate movements affected companies that had prepared their accounts in strong currencies. Pharmaceutical firms were so good at discovering drugs when their backs were against the wall that investment analysts used to act on the assumption that many drug businesses would grow at around 15% per annum in perpetuity, a far cry from today’s widespread stagnation.

From the early 1990’s the global pharmaceutical industry has changed. Instead of discovering drugs to sustain growth many companies have been merging. In this way they were able to sustain growth for a period by cutting out duplicated costs and sometimes by plugging gaps in their geographical coverage. The intention was to improve R&D productivity as well by offering improved central services, increasing expertise in key areas and enabling the best features of each of the merging operations to be preserved. In practice, the opposite has happened. R&D productivity in large pharmaceutical businesses has typically declined after mergers because of: increased bureaucracy; R&D staff unsettled by restructuring and the threat of redundancy; R&D departments that are too large for senior decision-takers to be intimately involved with all the science; and management by strategy or policy rather than by the quality of individual ideas and the determination of key scientists to pursue them. The past two decades should have seen an improvement in R&D productivity because of the broadening of the tools available. Huge technological and scientific advances have been made in terms of the human genome, high throughput screening, combinatorial chemistry, the immune system, metabolic pathways and tests for weeding out potentially unsuccessful drug candidates at an early stage.  Tragically large drug companies have failed to reap the rewards and in some cases have lost confidence in their own abilities. The focus of innovative, productive pharmaceutical R&D has largely moved to small, American biotechnology companies. There is also evidence that charities and academia are improving their productivity in pharmaceutical R&D, although they still lag well behind commercial companies.

The cumulative R&D cost in bringing a drug to the market is well over £10m and sometimes above £100m. These figures take no account of the cost of failures, which are important because only about one in ten drugs tested in humans eventually reaches the market. Governments do not need to fear that drug companies will deliberately focus on R&D that is not directed towards fulfilling unmet clinical needs. Companies simply cannot afford to run large R&D programmes aimed at developing products without significant advantages. When companies launch drugs that are little better than competitors the usual reason is that the products have failed to achieve what the scientists had hoped. After a large amount of money has already been spent on a product, bringing it to market may recoup more costs than abandoning it. The extra revenue may help finance other, potentially more successful R&D.

The concept of trying to use a UK drug pricing system to influence the way in which UK R&D budgets are spent is seriously flawed for several reasons.

  1. A company should carry out R&D projects in areas of unmet medical need where it has good ideas and appropriate technology. Where governments or marketing departments would most like to see discoveries should not have too large an influence. Experienced R&D staff, fully familiar with the relevant science, are best placed to determine whether a project has an acceptable chance of achieving its goals. A good R&D operation should involve marketing, regulatory affairs and project management staff at an early stage but not to choose between projects. The marketing, regulatory and project management roles are to ensure compliance with all regulations, to ensure that all helpful data is appropriately collected, to highlight any commercial factors that might otherwise get overlooked and to manage work not directly relevant to the science.
  2. The UK is too small a drug market and has too little pricing flexibility as a result of imports and exports of pharmaceuticals to be able to deflect companies from pursuing the projects most relevant to global markets.
  3. Pharmaceutical companies are important to theUK economy, both as an employer despite recent closures and because of their substantial contribution to exports. Any attempts to dictate R&D strategy will inevitably reduce the UK’s attractions in the eyes of the industry.
  4. Scientists are best motivated if they are actively involved in choosing their projects. What would Einstein have done if he had been directed towards work on semiconductors?
  5. R&D staff must feel free to terminate projects rapidly and cut their losses if the evidence no longer supports their line of approach. At the same time good ideas must be captured and acted on quickly even if they do not fit in with pre-conceived ideas as to what should be researched.

Some of the most important drugs to have been launched since 1990 to help fulfil unmet medical needs have concerned: AIDS, hepatitis, migraine, vomiting in cancer patients, influenza, the risk of arterial blood clots and lupus. Many lists of most wanted future discoveries compiled in the late 1980s contained less than half of these. Probably common cancers came near the top of most wish lists. Important advances have been made in the treatment of cancer with drugs, but huge amounts of money have been wasted by companies that have researched cancer without having had any good ideas about what to do. We need companies to generate good scientific ideas and evaluate them efficiently, Often successes begin with minority opinions and the adoption of a contrarian approach.

In conclusion, governments and the public should continue to encourage medical and pharmaceutical R&D strongly in industry, charities and academia. The focus on R&D in the NHS should be restored. However, support for a project should depend on the quality of the idea and not on dogma about what we would like to discover.  The choice of research projects is an area that governments should generally leave to professionals and not seek to influence. The potential pace of change should never be underestimated, as developments in the Internet, computing and electronics over the past decade demonstrate.

About Barbara Arzymanow

Barbara Arzymanow is a Research Fellow at 2020health and is a founding director of an independent healthcare consultancy firm. She has been an investment analyst specialising in Pharmaceuticals for 25 years, prior to which she carried out academic medical research in university laboratories. Her experience, obtained entirely from outside the pharmaceutical industry, gives her a unique, political perspective independent of commercial lobbies. She has extensive experience in financing the biotechnology industry, which is vital for the long-term standing of medical research in the UK. She has always been inspired by the scientific excellence within the UK and would like to see collaborations between industry, the NHS and academia strengthened. For more information about Barbara's research and writings including submissions to Government Departments please visit . Barbara also tweets as @barbararesearch .
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