Priority Medicines for Europe and the World
"A Public Health Approach to Innovation"
“Opportunities to Address Pharmaceutical Gaps”
By Saloni Tanna, Pharm.D. MPH
7 October 2004
1. Introduction 4
2. Size and Nature of Disease Burden 4
Incidence and prevalence 4
Country impact 5
3. Control Strategy 7
Table 1. Therapeutic options in osteoarthritis Error: Reference source not found, Error: Reference source not found, Error: Reference source not found, Error: Reference source not found, , 9
Non-pharmacological treatment 9
Pharmacological treatment 9
Intra-articular treatment 9
Non-pharmacological therapy review 9
Pharmacological therapy review 10
Affordability, feasibility and sustainability 16
4. Major Problems and Challenges for Disease Control (Why Does the Disease Burden Persist?) 16
Risk factors for incidence and progression of osteoarthritis 17
5. Past/Current Research into Pharmaceutical Interventions for OA 18
6. Current Pharmaceutical Product “Pipeline” for OA Treatment 18
7. Opportunities for Research into New Pharmaceutical Intervention 20
8. Gaps Between Current Research and Potential Research Issues that Could Make a Difference 20
9. Conclusion 22
10. References 23
Osteoarthritis (OA) is the most common type of arthritis or degenerative joint disease.1 It is a leading cause of chronic disability. The disease most commonly affects the middle-aged and elderly, although younger people may be affected as a result of injury or overuse. Age is the strongest predictor of the disease and therefore increasing age and extended life expectancy will result in a greater occurrence of the disease. Patients affected by this disease suffer from pain and loss of function.
OA is regarded as a complex disease whose cause is not completely understood. Furthermore, effective biomarkers, diagnostic aids and imaging technology are not available to assist in the management of OA. There are also several areas where information is still lacking; these include: epidemiology, pathophysiology, environmental risk factors, genetic predisposition and lifestyle factors.2, 3
At present, there is no cure for OA. The management of OA is broadly divided into non-pharmacological, pharmacological, and surgical treatments. Surgical management is generally reserved for failed medical management where functional disability affects a patient’s quality of life. Pharmacological management includes control of pain and improvement in function and quality of life while limiting drug toxicity. Experts in this field suggest that appropriate therapy for OA combines one or more pharmacological agents with exercise, weight loss and physical therapy (i.e. non-pharmacological therapy).
There are a number of drugs under development for symptomatic and disease modification, and several studies are also evaluating alternative therapies. There are several drugs on the market whose clinical effectiveness and long-term safety still need to be determined. This assessment is especially important since OA requires long-term disease management and the disease primarily affects people over the age of 60 who are most prone to drug toxicity, and for whom the potential for drug interactions are high. Information on the impact of the disease to society and the cost of disease management (including pharmacological and non-pharmacological treatments) needs to be re-evaluated. Finally, most experts emphasize that more research efforts need to be directed towards new diagnostics, biomarkers and imaging technology. This is an essential area of research in OA since it will help to determine who is likely to get OA; severity and progression of disease; patient response to drugs, and the development of disease modifying drugs that have the potential to halt or reverse the disease.Error: Reference source not found, Error: Reference source not found
There are more than 100 different types of arthritis.Error: Reference source not found The most common type of arthritis is osteoarthritis (OA) or degenerative joint disease. It is a common chronic, progressive musculoskeletal disorder characterized by gradual loss of articular cartilage. The disease most commonly affects the middle-aged and elderly, although it may begin earlier as a result of injury or overuse. It is often more painful in weight bearing joints such as the knee, hip, and spine than in the wrist, elbow, and shoulder joints. All joints may be more affected if they are used extensively in work or sports, or if they have been damaged from fractures or other injuries.Error: Reference source not found, 4
Musculoskeletal conditions are a major burden on individuals as well as health and social care systems, with significant indirect costs.
Incidence and prevalence
Literature is limited on the incidence and prevalence of OA because of the problems of defining it and determining its onset. Worldwide estimates indicate that 9.6% of men and 18% of women 60 years have symptomatic OA.5
OA is a major cause of impaired mobility. In 1990, OA was estimated to be the eighth leading non-fatal burden of disease, accounting for 2.8% of total years of living with disability.Error: Reference source not found
OA is the highest-ranking disease among the musculoskeletal diseases and contributes to approximately 50% of the disease burden in this disease group (See Background Chapter 5).
Overall disease burden ranking according to this compiled data shows a ranking of 12 for combined 25 EU countries; 15th ranked for old EU and 9th rank for the 10 EU accession countries.
igure 1 Burden of disease of OA by age groups and regions
Note from Figure 1, the peak in the burden of disease (DALYs) in each of the three regions: Global, EU15, EU25, and EU10 are different. In EU 10, the onset of OA is at an earlier age, perhaps resulting in more disability, loss of productivity and increased health care costs.
Knee OA is likely to become the fourth most important global cause of disability in women and eighth most important in men.Error: Reference source not found
OA contributes to a higher disease burden in men below the age of 50 and in women over the age of 50.
According to expert opinions presented in the EULAR committee report, radiographic evidence of knee OA in men and women over 65 is found in 30% of patients.Error: Reference source not found
Figure 2 shows the prevalence of OA of the knee by age group, sex and region.Error: Reference source not found In general OA is more prevalent in Europe and USA than in other parts of the world.Error: Reference source not found