Topics - Innovation

Research & Development

The fruits of Research and Development, or R&D, can add years to life, and life to years. An investment in R&D is an investment in patient wellbeing. In 2011 alone, the industry invested €27.5 billion in research and development in Europe. The new research has enabled us to make progress in areas such as personalised medicines, orphan drugs and biologics – notably biologics using monoclonal antibodies, laboratory-produced molecules engineered to attach to specific defects in cancer cells. Biological drugs have been developed for such serious illnesses as cystic fibrosis and asthma. Investment in R&D also has a positive economic impact: Research initiatives nurture collaboration among diverse stakeholders, from SMEs to academia, create new jobs in Europe, and can help reverse the European “brain drain”.

European pharmaceutical companies manage the R&D process from a molecule, where it is identified as a potentially suitable medicinal product, to a medicine, where it is licensed and available for a doctor to prescribe to their patient. This can take up to 13 years and is a long, expensive and complex process, necessary to ensuring medicines meet the standards of quality, efficacy and safety.

Investment into R&D has always been essential to improving the wellbeing of patients. Without it, we would not have the treatments that help patients now – and would have no new medicines in the future. Today, investment in R&D is more imperative than ever, as scientists and researchers have conquered many of the low-hanging fruits of innovation and are tackling some of medicine’s more complex challenges: cancer, diabetes, HIV, and more.

These are complex problems requiring complex solutions. We already have seen success in the cases of personalised medicines for certain breast and skin cancers. Such personalised medicines offer unique treatments developed on the basis of a specific tumour’s molecular makeup and the patient’s normal tissue. The R&D processes required to develop such treatments are complicated, costly, and time-consuming – but the benefits are worth the investment, as we are already seeing.

Looking at the big picture, R&D in the pharmaceutical industry is moving towards open innovation, embracing increased collaboration and expertise sharing. The Innovative Medicines Initiative (IMI) is one example of this, as a public-private partnership bringing together diverse stakeholders across the EU, from patient groups to academics. Such collaborative models have spread among several EU countries, and most recently to Japan, with the establishment of the Global Health Innovative Technology Fund.

The Medicine Development Process: The long path from petri dish to patient

It can take up to 13 years to take a medicine from its origins as a molecule to a treatment with tangible benefits for patients. A look at the many steps needed to complete this process can help explain why it is so time-consuming.

  • Pre-discovery: Based on a disease focus, scientists work to better understand the disease.
  • Drug Discovery: Researchers select a “target” such as a gene or protein, then search for a molecule, or compound, which may act on the target to alter the disease.
  • Pre-clinical testing: Early safety and efficacy tests are undertaken using computer models, cells and animals.
  • Phase I Clinical Trial: The candidate medicine is tested in people for the first time. Studies are usually conducted on a small number (20 to 100) of healthy volunteers. The dose is gradually increased during these clinical trials to allow the investigator to measure the participant's clinical response to the medicine, such as whether the medicine is sufficiently absorbed, how long the medicine remains in the bloodstream, and which dosage levels are safe and well tolerated.
  • Phase 2 Clinical Trial: Researchers evaluate the potential medicines efficacy in treating an illness or medical condition, in about 100 to 500 patients with the disease. In this phase, researchers work to determine the most effective dosages and the best method of delivery – for example in tablet form or by injection.
  • Phase 3 Clinical Trial: Researchers study the potential medicine in about 1,000 to 5,000 patients to generate data about safety, efficacy and the overall benefit-risk relationship of the medicine. Phase 3 is used to test the results of earlier trials in larger populations and gather additional information about the effectiveness and safety of the medicine. This phase generally provides the basis for the benefit-risk assessment for the new medicine and much of the core information about the medicine that will be described on the labelling.
  • Licensing approval: Information and results from all the studies as well as description of the medicine's manufacturing process is compiled and submitted to the regulatory agencies in order to demonstrate the safety and effectiveness of the new medicine.
  • Health Technology Assessment: Once it has been concluded that a medicine is safe and effective, its value and cost-effectiveness must be assessed. Health Technology Assessment (HTA) processes are used by the relevant regulatory body to assess the added-value of medicines and inform decisions on access. Depending on the given healthcare system, HTAs can be used to determine pricing, reimbursement status, and/or prescribing, for instance.
  • Medicines available for patients: Once a medicine is licensed for use, and pricing and reimbursement measures determined, it may be made available for patients.
  • Post marketing studies: Even after a drug is on the market, it is being scrutinized: post-approval or post-marketing studies are necessary to monitor a drug’s long-term effects.