On average, a new drug brought to market in the United States requires $1 billion spent over 10 years in order to meet the stringent standards of the U.S. Food and Drug Administration (FDA). In 2012, however, the FDA sought out ways to ameliorate these high costs and lengthy delays of bringing breakthrough drugs to pharmacies across the country, creating a new program that expedites the process for medicines that show evidence of having major benefits for people with serious injuries and illnesses. Since then, more than 50 drugs have been admitted to the program, including new treatments for blood cancers, cystic fibrosis, and hepatitis C.
For pharmaceutical companies and patients alike, this has been a major boon in many cases. One drug spent only six years between its initial trials and ultimate approval after inclusion in the new FDA program. Some observers have criticized the degree to which the FDA’s new process has complicated both pricing considerations and marketing concerns, but the FDA itself has declared the program an unqualified success. Industry experts consider it likely that in the coming years, the effects of the FDA’s new rules will make it much easier for promising drug candidates to reach American hospitals.
In the field of pharmaceutical research, clinical trials represent one of the most important stages in a drug’s life cycle. Conducted in a series of phases and carefully monitored by the United States Food and Drug Administration, clinical trials answer important research questions surrounding the safety and efficacy of pharmaceutical drugs.
Typically, clinical trials encompass four phases. In the first phase, or Phase I, researchers administer a drug to a small group of people to gain an understanding of safe dosage and possible side effects. In Phase II, scientists expand the research group from several hundred people to several thousand to further test the drug’s safety and efficacy. In Phase III, researchers again increase the scope of the trial and begin learning about the potential therapeutic effects of the treatment. Phase IV—the final phase of the clinical research process, commonly referred to as post-marketing surveillance—involves careful monitoring of patients to determine the effects of the drug in the long term.
For prospective investors in the health care sector, pharmacy and biotechnology represent two of the most popular areas of interest. Each possesses its own advantages and disadvantages in terms of risk tolerance. Here is a quick look at the two types of stocks.
Biotechnology: In general, biotechnology stocks carry a significant amount of risk, due to the possibility of regulatory obstacles, competition, and failed clinical trials. If a technology falls short of its objectives, the project often folds within days and the stock loses the majority of its value. However, if a drug or technology succeeds in all clinical trials, stocks can experience rapid growth in a relatively short period of time.
Pharmaceuticals: Compared to biotechnology, the pharmaceutical sector requires investors to assume a relatively small amount of risk. The factors determining the price of pharmaceutical stocks typically include reimbursement charges, new drug pipelines, strategic alliances, and prescription data. Many of these are fairly easy to predict, which results in less volatility.
Acute promyelocytic leukemia (APL) is a cancer of the white blood cells that affects individuals of a wide range of ages. Unlike many other leukemias, APL is a relatively treatable condition, which features a 12-year, progression-free, survival rate of 70 percent.
Treatment for APL usually begins with the administration of the non-chemotherapy drug known as all-trans-retinoic acid (ATRA), which possesses a structure closely related to vitamin A. Used in conjunction with an anthracycline chemotherapy drug such as idarubicin or daunorubicin, ATRA produces results in a considerable number of patients. Alternatively, physicians may choose to prescribe the drug arsenic trioxide, particularly for patients with an intolerance to anthracycline drugs.
After undergoing initial treatment, many patients receive post-remission treatment with combinations of several drugs and begin a maintenance therapy regimen that involves ATRA administration for at least one year. In some cases, doctors may also prescribe low doses of the chemotherapy drugs 6-MP and methotrexate.
In the drug discovery process, finding a lead compound represents one of the most important tasks for researchers. Once scientists find a compound that affects the target molecule and passes a series of rigorous safety tests, they begin altering the structure of the compound to enhance certain biological and chemical properties. Often referred to as lead optimization, the alteration process results in safer and more effective compounds.
Researchers often begin the lead optimization process by identifying interactions with other biological systems in the body and making the drug less likely to interact with them. By testing hundreds or even thousands of analogues, scientists can greatly decrease the likelihood of side effects. Lead optimization typically involves a significant back-and-forth between biologists, who test the compounds on living systems, and chemists, who make the desired alterations to the drug. At this stage, chemists also begin to consider logistics such as the delivery mechanism, inactive binding agents, and large-scale manufacturing.
In the field of drug discovery, researchers typically begin the process by identifying a target molecule and validating it as a major cause of disease. Once they find a suitable target, researchers must then begin the search for a promising drug molecule, often referred to as a “lead compound.” Following are three ways scientists find lead compounds:
- Nature: One of the oldest sources of medicinal compounds, nature plays host to a wide range of bioactive substances. While the large majority of nature-based research has focused on the medicinal properties of plants, researchers have discovered promising compounds in bacteria and mold.
- Biotechnology: Over the past few decades, scientists have discovered many ways to genetically engineer living systems and cause them to produce specific biological molecules.
- High-throughput screening: One of the most common drug discovery methods, high-throughput screening can test a large volume of compounds against the target molecule in a relatively short period of time.
As Johnson & Johnson’s sales in the consumer and medical devices/diagnostics sectors have dipped, the company has experienced significant increases in its pharmaceutical sales. Johnson & Johnson maintains a strong portfolio of high-performing drugs, including immunology drugs like Simponi and Stelara, the hepatitis C treatment Olysio, and two oncology drugs, Velcade and Zytiga. Analysts predict that these five drugs will help drive Johnson & Johnson’s estimated 5 percent growth in overall revenue for the next year.
Zytiga will make an especially critical contribution to this growth rate. The blockbuster prostate cancer drug surpassed Velcade as Johnson & Johnson’s top-selling oncology drug, and it experienced a 48.8 percent revenue increase in its quarter-one revenues. In the first quarter, Zytiga generated $512 million, over $100 million more than Velcade. Additionally, sales of Zytiga jumped 77 percent from 2012 to 2013, resulting in $1.7 billion for the pharmaceutical giant. The drug does face upcoming competition, however, from such therapies as Medivation and Astellas’ Xtandi. Investors will be watching closely as the companies report on these drugs’ quarterly sales.
One of the most studied and least-understood psychiatric disorders, schizophrenia manifests itself in a variety of ways, and researchers have yet to identify a single underlying cause. Researchers have, however, identified three factors that correlate with the onset of schizophrenia.
Family history: One of the most reliable risk factors of schizophrenia, family history has been shown to correlate strongly with schizophrenia diagnoses. A child of a parent with schizophrenia has a 10 percent chance of developing the disorder, while an individual whose identical twin has been diagnosed with schizophrenia has a 40 percent to 65 percent chance.
Chemical factors: In recent years, schizophrenia research has focused on the role of neurotransmitters. In particular, research has shown that schizophrenia may be related to imbalances in compounds such as glutamate and dopamine.
Physical abnormality: With the arrival of advanced neuroimaging techniques, researchers have discovered that people with schizophrenia often have abnormal brain structures, such as larger ventricles, less gray matter, and abnormal activity in certain areas.
Seasonal influenza, which is especially prevalent during the fall and winter months, affects millions of people, infecting as much as one-fifth of the American population annually. According to the Centers for Disease Control and Prevention, the most common way for flu germs to spread is via respiratory secretions: the droplets of fluid expelled when someone coughs or sneezes. Contact with these droplets can result in infection from as far as 6 feet away. In some cases, an individual can contract the flu virus by touching an object handled by a contagious person and then touching his or her own mouth or nose.
Individuals with flu should make every attempt to avoid spreading it to others by staying home while they are ill. A generally healthy adult is able to transmit the virus to others a full day before displaying any symptoms. In addition, he or she may continue to be contagious for as much as one week afterward.
To prevent the spread of flu, remember to disinfect surfaces that an infected person has touched. Thoroughly wash dishes used by anyone who is sick with flu, and launder linens and towels before another person uses them. To avoid contracting flu, children and adults should wash their hands often and avoid touching their mouths and noses, especially when they are in well-frequented public places.
Furthermore, the CDC advises the influenza vaccine, targeted to the current season’s strain of the virus, for everyone 6 months of age and older, unless an individual’s physician recommends otherwise.
Fibromyalgia, a musculoskeletal condition characterized by increased sensitivity to pain throughout the body, is often associated with sleep disorders, lowered energy levels, and difficulty remembering. Experts believe the condition affects some 5 million American adults, women more frequently than men. Though patients living with fibromyalgia can often find pain relief with analgesic medications, the condition can become a chronic problem.
While no researcher has yet posited a definitive cause for fibromyalgia, several theories have developed. Some experts believe that the condition results from a chemical or endocrine problem affecting the brain’s neurotransmitting system, from nerve cells made overly sensitive to pain, or from an emotionally traumatic event.
Decreased levels of the calm-inducing neurotransmitter serotonin may result in heightened sensitivity to painful stimuli, as may an excess amount of substance P, which boosts the body’s amplification of painful sensations. In addition, some theorists have pointed to lower levels of human growth hormone as a contributor to the development of fibromyalgia.
In addition, certain infections may be at the root of the condition, or may initiate flare-ups. Many researchers have proposed that fibromyalgia has a genetic component, as the condition does tend to occur in family members. However, most agree that the likeliest scenario involves several factors in combination.