Pharmaceuticals and Biotechnology

The discoveries and inventions of the pharma/biotech industry have changed human existence. They’ve increased life expectancy, improved survival rates for those suffering from deadly diseases such as cancer and heart disease, allowed physicians to diagnose diseases and disorders more accurately and quickly, and generally improved human health.

Some of the most important breakthroughs of the 19th century include:

• the development of chloral hydrate, the first synthetic sleep-producing drug, in 1869
• the discovery that organic nitrates such as nitroglycerin could be used to treat heart problems, in 1879
• the development of acetanilide, the first analgesic-antipyretic drug (fever and pain relief), in 1886
• the development of phenacetin, a less-toxic alternative to acetanilide, in 1887
• the development in 1899 of aspirin (acetylsalicylic acid), which became the most popular anti-inflammatory, pain- and fever-reducing drug until the 1960s
• the development of benzocaine, an anesthetic that was more efficient and less toxic than previous local anesthetics (such as cocaine), in 1900

As the 20th century dawned, society still faced major challenges from infectious diseases, which were the leading cause of death in most of the world. One major drug breakthrough was the development of arsphenamine to treat syphilis, in 1910. This drug, which was marketed under the name Salvarsan, was used to treat syphilis until the 1940s, when it was replaced by penicillin. Other groundbreaking achievements of the early 20th century include the development of derivatives of sulfonamides, or sulfa drugs, to treat streptococcal infections and other types of infectious diseases, and the discovery of insulin for use in treating diabetes.

The development of penicillin in 1929 is one of the major breakthroughs in the history of the pharmaceutical industry. This “miracle drug” was discovered by the Scottish bacteriologist Alexander Fleming. It took more than 10 years of additional experiments to develop large quantities of penicillin. In 1942 Pfizer became the first pharmaceutical company to produce penicillin in large amounts. Many cite this event as the beginning of the age of modern medicine. Since then there has been a multitude of drug and treatment breakthroughs. Some of the most important are

• 1950s: corticosteroids (for inflammation), antihistamines (for nasal allergies), xanthines (for asthma), antipsychotics (for psychosis), the discovery of DNA, the polio vaccine, the first organ transplant, the first well-tolerated blood pressure medications
• 1960s: the first human heart transplant, birth control pills, the oral polio vaccine, the mumps vaccine
• 1970s: the creation of a modified DNA molecule by recombining DNA from two different organisms, the birth of the first test tube baby, fertility drugs, vaccines for rubella, chicken pox, pneumonia, and meningitis
• 1980s: the eradication of smallpox, the artificial heart, treatments for HIV/AIDS, the hepatitis B vaccine
• 1990s: the first clinical gene therapy trials, HIV protease inhibitors, the first therapy for ischemic stroke, the first mammal was cloned from an adult cell, the first human embryonic stem cell line was produced, vaccines for hepatitis A and Lyme disease
• 2000s: the first face transplant, the emergence of nanomedicine, targeted cancer drugs, stem cell research, integrative medicine, synthetic genomics
• 2010s: the first self-replicating, synthetic bacterial cell; the first therapeutic cancer vaccine approved; the first FDA-approved medicine manufactured using 3-D printing technology; digital health devices and wearables; the discovery of the CRISPR Genome Engineering Tool; the approval of the first human trials for CRISPR; the first gene therapies approved
• 2020s: the use of artificial intelligence to improve screenings for cancer and other diseases; the FDA granted the psychedelic drugs MDMA and psilocybin breakthrough therapy status, which signaled a shift in the future of mental health treatment; major developments in weight-loss medications and treatments; generative AI

Two of the most noteworthy developments in the history of the pharmaceutical industry are the emergence of biotechnology and the mapping of the human genome.

The field of biotechnology emerged in 1972, when scientists created a modified DNA molecule by recombining DNA from two different organisms. In 1982 a biotech company named Genentech began selling the first biotech drug, human insulin. This marked the beginning of the modern use of biotechnology to manufacture products. (Biotechnology concepts such as fermentation have been used for thousands of years to make beer, wine, cheese, and other products.) Today biopharmaceutical companies are using biotechnology to develop thousands of vaccines and medicines. The Biotechnology Innovation Organization reports that more than 250 biotechnology health care products and vaccines are available to patients. In addition, nearly 260 vaccines are in development by biopharmaceutical companies in the U.S. for infectious diseases (such as COVID-19), cancer, allergies, and Alzheimer’s disease.

The U.S. government launched the Human Genome Project (HGP) in 1990. Its mission was to map the entire human genome, or all the genetic material in human beings, with the goal of completing the sequencing by 2005. In 1998 Celera Genomics Corporation (a for-profit company) announced that it would start its own sequencing project to compete with the HGP. By 2001 (four years ahead of schedule) both organizations had completed rough drafts of the human genome. Project results promise new scientific knowledge, medicines, and therapies that can be used to battle diseases such as AIDS, cancer, arthritis, and osteoporosis.

The coronavirus pandemic was a major focal point for the pharmaceuticals and biotechnology industry since it began in Wuhan, China, in late 2019. From pressure to develop vaccines and treatments for the virus, to following pandemic protocols to protects its own workers, the biopharmaceuticals industry worked through disruptions and challenges in 2020 to achieve much in a short period of time. By the end of 2020, the Moderna and Pfizer-BioNTech vaccines had been emergency-use authorized by the Federal Drug Administration for the prevention of COVID-19. In March 2020, the FDA granted emergency use authorization to the Janssen/Johnson & Johnson COVID-19 vaccine. More than 10 drugs and non-vaccine biological treatments were approved in the first few years after the start of the pandemic. The introduction of COVID-19 vaccines in the U.S. is estimated to have saved more than 2.2 million lives and prevented up to 18.2 million hospitalizations between December 12, 2020, and March 31, 2022, according to “Impact of U.S. COVID-19 Vaccination Efforts: An Update on Averted Deaths, Hospitalizations, and Health Care Costs Through March 2022,” at The Commonwealth Fund’s “To the Point” blog.

In early 2023, the Biden Administration rescinded the COVID- focused national emergency and public health emergency declarations and announced that the federal government would treat the coronavirus outbreak as an endemic threat to public health that could be managed through agencies’ normal authorities. Although COVID-19 remains a threat to human health and life, the availability of vaccines and treatments have significantly reduced the number of people who become severely ill or die from the virus.

The Pharmaceutical Research and Manufacturers of America and 100 other organizations collaborated to develop the following recommendations to improve the nation’s disaster preparedness and response to better prepare for the next pandemic:

• Modernize the health care supply chain through digitalization, automation, and predictive analytics
• Improve supply chain resilience through targeted incentives
• Make policy reforms to support rapid response including medical licensure portability, removing barriers to telehealth, and swift access to personal protective equipment and other protective equipment

Key Laws

The U.S. government has regulated the pharmaceutical industry since 1848. That year the Drug Importation Act was passed, banning the importation of substandard or adulterated drugs. Some major legal/regulatory milestones in the history of the U.S. pharma/biotech industry include:

• The Pure Food and Drug Act (1906), which prohibited interstate commerce in adulterated or mislabeled drugs and established The United States Pharmacopeia and The National Formulary as the standard for drugs produced in the United States
• The founding of the Food and Drug Administration (1927) to provide legal enforcement for the Federal Food and Drug Act of 1906
• The Federal Food, Drug, and Cosmetic Act (1938), which required strict and detailed studies in the development of medications before they could be made available to the general public
• The Humphrey-Durham Amendment to the Food, Drug, and Cosmetic Act (1951), which required certain drugs to be sold only by prescription
• The Kefauver-Harris Drug Amendments (1962), which for the first time required drug developers to prove to the FDA the effectiveness of their products before marketing them
• The Fair Packaging and Labeling Act (1966), which required consumer products in interstate commerce to be informatively and honestly labeled
• The Orphan Drug Act (1983), which promoted the development and commercialization of drugs to treat rare diseases (those which affect fewer than 200,000 people)
• The Drug Price Competition and Patent Term Restoration Act (1984), which prompted strong growth in the generic drug industry
• The Prescription Drug Marketing Act (1988), which ensured that drugs sold to the American public were not substandard, ineffective, or counterfeit
• The Patient Protection and Affordable Care Act (2010), which expanded health insurance coverage to 15 to 20 million Americans
• The Leahy-Smith America Invents Act (2011), which transitioned U.S. patent law from a “first to invent” to a “first to file” system—meaning that patents are awarded to the first inventors that apply, rather than the first to invent.