Cancer is a principal cause of mortality worldwide, accounting for around 13% of all deaths in 2008. The number of cancer deaths is expected to rise further to reach over 11 million in 2030. Today, about 6% of total US health expenditure are allocated to cancer care. With respect to per-patient costs, cancer is the most expensive health condition. Compared to other therapeutic areas, cancer drugs obtain higher shares of FDA priority review ratings, of orphan drug designations, and are more often granted inclusion in FDA's expedited access programs. According to the EMEA, one third of new chemical or biological entities under clinical development are currently aimed at the treatment of malignancies, which reflects the need for improved cancer therapies.
New pharmacological approaches, targeted and patient-tailored treatments, and innovative radiation/surgical techniques have substantially improved survival and quality of life of cancer patients. However, a particular challenge arises today from recurrent malignancies and new clinical research is needed to focus on cancer as a chronic disease.
Osteoarthritis and Cartilage Injury, Imaging
Cartilage injuries represent an unmet medical need, because these defects poorly heal in adults and to date, there is no treatment available for partial defects. In contrast to osteoarthritis (OA), where the development of disease-modifying drugs is long and difficult, treatment of patients suffering from acute, focal cartilage injury should be short and curative. Therefore, the probability of success might be higher. However, both indications are often treated by the same specialists and cartilage injury may ultimately lead to secondary OA. Thus, both indications ideally complement each other and might be seen as extremes of a disease continuum. Therefore, the development of novel treatments for acute cartilage injury can support the search for disease-modifying OA drugs.
A large number of studies have been undertaken to establish the role of magnetic resonance imaging (MRI), which holds great promises in diagnosis, monitoring of treatment response, and follow-up of these conditions.
COPD, asthma, and smoking cessation
Chronic obstructive pulmonary disease (COPD) is a major cause of disability and death. Besides symptom-relieving therapies, phosphodiesterase 4-inhibitors are now included in COPD treatment guidelines. These drugs treat the underlying chronic airway inflammation and may help to slow down the progressive structural damage of the lung. Specific anti-inflammatory approaches and new classes of bronchodilators, such as smooth muscle myosin inhibitors, may also lead the way to more effective therapies of asthma.
Smoking causes both COPD and asthma. Current pharmacological treatments for nicotine addiction can double smoking cessation rates. New compounds that selectively interact with nicotinic receptor subtypes should allow to resolve nicotine dependency with less systemic side effects.
The complexity of the immune system offers a multitude of therapeutic approaches against autoimmune disease, allergy, and infection. Defects in the immune system that make the body attack its own healthy tissues cause autoimmune disease. The example of anti-cytokine treatments for rheumatoid arthritis shows how scientific advances in immunology may bring relief to millions of patients. However, other potentially devastating autoimmune diseases are still treated with old, non-specific drugs that have considerable side effects. Despite significant progress in the understanding of allergic diseases, the increasing incidence of asthma, eczema, drug and food allergies poses a major clinical challenge. The role of infections in the pathophysiology of allergies is particularly intriguing, since they may either trigger off the disease or have a protective effect against allergy.
In the treatment of infectious disease, the limits of antibiotics have become obvious. While vaccination may be the only way to overcome some of the world's most deadly plagues, the protection against pathogens adapted to evade immunity remains in many cases an elusive goal.
Orphan and neglected diseases
Diseases may be designated orphan status if they are either rare or if they are by far most prevalent in developing countries. Currently, about 6’000 of mostly chronic diseases fall into this category. Definition criteria vary in different regions of the world, as do incentives for pharmaceutical companies to develop therapies for those diseases. Although diverse, overall those incentive programs were successful in fostering drug development in rare indications. Solely in the US, more than 300 orphan-designated products received marketing approval for 200 different diseases since the release of the US Orphan Drug Act in 1983, with a median time span of only 4 years between granting orphan designation and approval. Also in Europe the trend continues upwards.
It is noteworthy, that for orphan diseases (as well as for drugs with well established use) not only regulations are different, but also the common CTD format to apply for marketing authorization has to be considerably modified. Regarding orphan diseases, we have worked on leishmaniasis, Peyronie’s disease, and phenylketonuria.