34 No. 5
The pharmaceutical industry has made important contributions to quality of life, longevity, economic growth, and education, and is a key component of the economic growth strategy for many countries. However, the industry is now under considerable pressure as the number of new chemical entities has not increased over the past decade, and there have been significant revenue losses as important drugs lost patent protection. Consequently, the sector has experienced major reductions in R&D budgets, closure of research sites, and the loss of thousands of skilled jobs. A new and sustainable funding model with public-sector participation is urgently required for world-class scientists to invent and develop innovative medicines that meet the medical needs of the 21st century and contribute to economic growth. This perspective considers how these objectives might be achieved, and aims to stimulate discussion.
First, we should build consensus among a group of expert stakeholders with the common objective of informing and influencing future development of world-class healthcare innovation. Learned societies and professional bodies have obviously contributed to the government reviews of life sciences, but we now need to work together to ensure proper focus on drug discovery. Governments have stated a firm commitment to life sciences as a catalyst for growth, but emphasis appears to be on clinical trials, biologics, and cell therapies rather than on cost-effective and orally delivered small molecules that are the bedrock of any healthcare system.
|. . . the sector has experienced major reductions in R&D budgets, closure of research sites, and the loss of thousands of skilled jobs.
Second, future R&D should focus on therapeutic areas of significant medical need where transformative new drugs will improve quality of life, and bring economic benefit. For example, a recent survey showed that 38 percent of Europeans suffer from mental disorders, with depression being the single greatest burden of all human diseases. Brain disorders cost Europe almost 800 billion Euros per year, which is more than cancer, cardiovascular disease, and diabetes combined. Yet, Pharma is withdrawing from neuroscience research. To encourage investment in such challenging areas, it will be important to define efficacy/safety criteria early in drug discovery programs so that effective new agents can expect fair reimbursement and an acceptable return. This would minimize negative regulatory decisions—which often are made after a company has invested 10 years or more in discovery and development—and would help lower costs.
Third, we must address attrition as current failure rates during discovery and development are creating higher R&D costs that are simply unsustainable. One approach would be to expand precompetitive collaborations between industry/academia to focus on target selection/validation, predictive toxicology, and to identify patient subgroups that respond to agents with novel mechanisms of action. These initiatives would reduce risk, simplify clinical trials, and lower costs, which would make the sector more attractive for investment, and would also deter parallel and wasteful pursuit of nonvalidated targets. Companies would create intellectual property through innovative, but distinctive, medicinal chemistry programs.
Fourth, as Pharma contracts and Biotech struggles for new investment, we must consider alternative models of drug discovery that capitalize on our outstanding record of innovation and productivity. Networks of therapeutic centers of excellence should be established where expert medicinal chemists released by industry can work within multidisciplinary environments to exploit significant investments in biomedical research. Unused assets from Pharma could be included, and a flow of spin-off companies would revitalize the biotech sector. Such an arrangement would help educate and train of the next generation of research scientists and help create new jobs.
Fifth, universities should be encouraged to appoint experienced industrial scientists as professors and researches in their world-class biomedical centers since chemists play a pivotal role in transforming biological discoveries into innovative new medicines. There is also a pressing need to invest in fundamental new chemistries to address challenging biological targets such as protein/protein and protein/nucleotide interactions that are currently beyond traditional drug templates. It is important to realize that the majority of medicinal chemistry “muscle” was located in Pharma and the significant expertise recently lost due to layoffs should be re-captured within therapeutic centers of excellence, or similar initiatives. If world-class Pharma/Biotech talent fades away, it will be difficult to rebuild it in the future.
Finally, any new funding model may require reallocation of current budgets rather than additional monies considering current deficit constraints. However, the European Union recently announced “Horizon 2020,” which is investing 80 billion Euros in R&D to improve long-term competitiveness. Of this amount, 8 billion has been set aside for health care, which is considered to be a “major concern.” (see http://ec.europa.eu/research/horizon2020) Public-private partnerships involving universities, research centers, and industry will play a key role, and therapeutic centers of excellence make a natural and synergistic fit. Governments, funding bodies, and charities should invest now for future growth, as economic benefits will largely depend on strong drug discovery capabilities that lead to new medicines for worldwide commercialization. Investment in medicinal chemistry will translate biological discoveries into commercial products, consistent with the EU’s commitment to life sciences, and will provide economic gains.
The positive benefits of such exciting initiatives include the following:
- new biology would be translated by expert medicinal chemists into innovative new medicines to help meet the healthcare challenges of the 21st century
- quality of life would be improved within constrained healthcare budgets
- important disease areas abandoned by Pharma would receive proper attention, with consequent patient benefits
- assets not being pursued by Pharma would be revived and developed
- economic benefits would flow as innovative medicines enter world markets
- the science base would be strengthened with exciting career opportunities for the next generation of scientists
- the biotech sector would be revitalized as an additional source of innovation/discovery with a sustainable return on investment
last modified 5 September 2012.
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