Contract Manufacturing Organizations
(CMOs):
CMOs specialize in manufacturing pharmaceutical products on behalf of pharmaceutical companies, providing expertise in manufacturing processes, scale-up, and production of drug substances and finished products.
Patient Advocacy Groups, Patient
Foundations, and Individual Patients:
Patients provide insights into the experience of living with a disease, articulate their needs and desires to drug developers, and take on significant risk by participating in clinical trials. Their input helps in shaping drug development strategies and ensuring patient-centric approaches to care. Patient Advocacy Groups and Foundations can also provide funding for drug development and may participate in lobbying and advocacy as well.
For example, one of the primary focuses of the Alzheimer’s Association is funding research into the causes, treatments, and potential cures for Alzheimer’s disease. They provide grants to scientists and researchers working on various aspects of dementia to advance knowledge about the disease and improve care for those affected. Similarly, the Cystic Fibrosis Foundation invests significantly in funding research aimed at finding new treatments and therapies for cystic fibrosis. Both organizations advocate for policies and legislation that support people with these diseases.
Health Care Providers and Clinicians:
Physicians, hospitals, and healthcare professionals conduct clinical trials, prescribe approved drugs, and provide feedback on drug efficacy and safety in real-world settings.
The U.S. Food and Drug Administration (FDA):
The FDA (and similar regulatory bodies in different countries) oversees the drug approval process. They evaluate the safety, efficacy, and quality of drugs before granting approval for marketing and distribution.
Supply Chain and Distribution
Networks:
Companies involved in drug distribution, logistics, and supply chain management ensure that approved drugs reach patients through pharmacies, hospitals, and healthcare facilities.
Public and Private Insurance Providers
(Payers):
Medicare, Medicaid, the VA, private (employer-sponsored) insurance plans, and ACA exchange plans collectivize healthcare costs for participants, ideally spreading the cost of expensive care over the majority of our society. With pharmacy benefit managers (PBMs), they negotiate net prices with drug companies and control which treatments plan members can access. Behind-the-scenes negotiations starring opaque middlemen can misalign incentives, leading to untoward behavior and higher out-of-pocket costs for patients. (More on this in Chapter 5!)
As you complete this chapter, it’s hopefully become clear that the vast majority of drugs never make to market, and that drug development is a very expensive and lengthy endeavor. So where does the cash to run this process come from? Our next chapter takes a deeper look into what you’re probably wondering about: the money!