At the end of August and the beginning of September, the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA) approved the marketing of around a dozen medicines that could change the course of treatment for diseases that have so far been considered incurable. Patients suffering from various types of malignant and hematological diseases, rare diseases, epilepsy, and HIV are gaining new treatment options, and with them a greater chance of recovery and a better quality of life. Since the beginning of the year, more than one hundred new therapies have been approved on the European and U.S. markets, all as a result of years of research, the expertise of hundreds of top scientists, and the desire to provide patients with a new beginning through innovative medicines.
Medicines are among the most effective and widely used tools for treating and curing diseases. Numerous innovations throughout history have improved survival rates for certain diseases and overall human health, yet significant unmet medical needs remain. Scientists and researchers working on the discovery and development of new medicines listen closely to patients’ needs. Sometimes small innovations, such as making a medicine easier to administer—for example, allowing a patient to take a pill instead of receiving intravenous therapy—can greatly improve a patient’s quality of life. Therapies that can be administered at home also directly relieve the burden on healthcare systems, allowing resources to be focused on patients who truly require hospitalization. When it comes to medicines that keep certain diseases under control or even cure them completely, such discoveries are revolutionary.
In recent years, innovative medicines have led to a reduction in mortality from cardiovascular diseases by approximately 37% between 2002 and 2012. Today, two out of three people worldwide diagnosed with cancer survive at least five years, and mortality rates from all types of cancer have fallen by 21% since 1991. Currently, around 15 million people in Europe live with hepatitis C. The first generation of medicines (1999–2010) successfully treated the disease in 41% of cases over 48 weeks, whereas today’s fourth-generation therapies cure hepatitis C in 95–96% of cases within 8–12 weeks. Effective therapy and vaccination have contributed to the fight against infectious diseases, and effective therapeutics have transformed HIV/AIDS from a death sentence into a manageable disease.
Medicines being developed by the innovative pharmaceutical industry will change approaches to treating many diseases and are expected to improve treatment outcomes. Research and development efforts open up new potential areas of use in new indications, offering greater benefits and broader application than were known at the time of a medicine’s initial approval. For example, over the past 60 years, improved understanding of the molecular basis of disease has transformed what was once known as a single “blood disease” into multiple subtypes of leukemia and lymphoma, which can now be treated with personalized medicines.
How will we treat diseases in the near future?
By applying new knowledge and technologies, over the next five to ten years innovative medicines could address many currently unmet needs in oncology, cardiovascular and metabolic diseases, hematology, central nervous system disorders, infectious diseases, and other conditions affecting large segments of the population, thereby helping patients and society. Within the next five years alone, many promising medicines are expected to become available to patients.
Scientists have found ways to harness the body’s own immune system by directing immune cells to target specific types of blood cancer. CAR-T cell therapy involves genetically modifying T cells so that they can recognize and destroy tumor cells. This treatment approach could replace chemotherapy and has the potential to save the lives of people with blood cancers. In fact, this is not a distant prospect—two such medicines have already been approved in Europe.
For cancer patients, improved treatment outcomes could be achieved through combination therapy—the use of multiple medicines with different mechanisms of action to increase the likelihood of keeping cancer under control. Combination therapy could extend patients’ lives, improve their quality of life, and even offer hope to patients whose diagnosis is currently terminal. Each year, combination therapy could prevent the deaths of 30,000 people with non-small cell lung cancer.
Treating genetic diseases is extremely challenging, and gene therapy helps replace damaged or missing genes in cells by introducing DNA to treat genetic disorders, thereby correcting the root cause of the disease. This approach could cure lifelong and life-threatening conditions such as hemophilia.
Type 1 diabetes is one of the diseases that currently requires lifelong treatment. This may soon change with the use of cell therapy. Cell therapy involves the introduction of living cells into patients to replace or repair damaged tissue, thereby restoring organ or tissue function. In practice, this means patients may no longer require constant insulin therapy to control blood sugar levels. Cell therapies could help regulate blood glucose levels in nearly four million Europeans living with type 1 diabetes.
As antimicrobial resistance becomes an increasingly serious problem, antibacterial treatments could reduce the pressure to find new antibiotics by neutralizing highly pathogenic bacterial surface proteins or secreted toxins and activating the immune system to kill bacteria directly. Antibacterial monoclonal antibodies offer new ways to combat antibiotic resistance and bacterial infections. With more than 1,000 medicines in development, antibacterial antibodies could save 25,000 lives each year in Europe.
While current therapies for Alzheimer’s disease focus on treating symptoms, new treatments are aimed at early diagnosis and prevention by delaying the onset and/or progression of the disease through breaking down or preventing the formation of protein plaques. Delaying the onset or progression of Alzheimer’s disease allows patients to live independently for longer. Scientists are researching more than one hundred new medicines that could enable patients to lead more independent lives.
Therapies in development for non-alcoholic steatohepatitis (NASH) could reduce fat accumulation in the liver, potentially sparing up to 12,000 Europeans each year from the need for liver transplantation. New therapies under development will also alleviate migraine attacks and enable patients to enjoy a better quality of life.
The possibilities for developing and applying the treatment approaches described here to other diseases are countless. The guiding principle of those involved in research and development of new medicines is better disease management and reducing long-term treatment, which exhausts patients, their families, and the healthcare system as a whole. Every disease reduces the quality of life and productivity of those affected, both within their families and in the workplace. The primary goal for all stakeholders is improved treatment outcomes. Better health outcomes and more treatment options tailored to each patient’s needs lead to improved quality of life, allowing patients to live longer, healthier, and more productive lives.
References:
- EFPIA Annual Report 2017
- EFPIA: Value od Medicines. Medicines in Context. Dostupno na: https://www.efpia.eu/about-medicines/use-of-medicines/value-of-medicines/
- U.S. Food & Drug Administration
- European Medicines Agency
