Therapeutic areas: Strategically important diseases of the future

Society and industry—still the only provider of approved medicine—share the desire to treat important diseases. Governments too, since some diseases’ high prevalence affect the economic output of society. Yet the decisions as to which drugs will be developed are strictly in private hands. The risks required to treat some really important, and often increasingly prevalent, diseases mean that the desires of society and the industry are diverging. This is not boding well for our society. The understanding and willingness of governments to step in as a partner and financier is not apparent. Will governments recognize their role in this? Since the economic cost of these diseases, in terms of reduced productivity and medical costs to treat the disease, is enormous, understanding should be forthcoming. Government has invested for many decades in an enormous program of cancer research, which now shows dividends. The importance of further government investment in basic research is apparent when industry does not want to or cannot anymore afford it. As Big Pharma drops out of important therapeutic areas, it is not promising to assume that government can step in to the vacancy. There has to be a serious effort to find a formula by which the government gently or not so gently forces the industry to continue its efforts because it is so important. Perhaps the most important of these neglected diseases is Alzheimer’s disease (AD) because of its dehumanizing nature and its predictably massively increasing prevalence. Yet, none of the currently approved Alzheimer drugs can be used to prevent, cure, or even significantly slow the progression of this disease. It is predicted that by 2030 50% of the over 80s will have the disease. Almost 100% of people with an inheritable trait will develop the disease unless we succeed in finding a preventive therapy. Only recently has a preventive trial been announced. It is often thought that insulin-dependent diabetes is a well-treated disease and needs no further diagnostic and drug development efforts. This could not be further from the truth. The long-term complications of insulin-treated diabetes add to the economic burden, especially as the growth in prevalence of the disease expands with the waistlines of the world’s population. Three hundred million may have type 2 diabetes by 2030. Pharmaceutical control of weight loss has so far been vexed by serious side effects. When it comes to diabetes drugs, we start to recognize that the control of blood glucose can be further improved; the biggest contribution is not by drugs, but by self-diagnosis with point of care glucose measuring devices. Morphine is still the most effective medicine against pain. It has been around for 5,000 years and we haven’t come up with anything better. But it can be ineffective against neuropathic pain and cancer pain. New pain medicines are difficult to find because of the subjective measurement and poor animal models. Since many current pain killers are abused, there are other hurdles. Despite the many difficulties, society needs new pain killers at the same time that notable pharma are pulling out, despite the economic opportunity. Drugs against cancer have had many high-profile and expensive failures in recent years, with all major companies having at least one failed candidate. Biotech companies have made much progress, however, and they are either collaborating with or being taken over by Big Pharma. Oncology drug development has been crucially enabled by decades of government investment. The key is the many characterized drug targets that other disease areas do not have. Government intervention in oncology and HIV provide the model for treatment of AD, neuropathic pain, and schizophrenia, among other therapeutic areas. Increasing governmental direct funding of translational research is not the solution many hope it will be.