Orphan Drug Production
Rare Diseases
Drug Approval
Economics, Pharmaceutical
United States Food and Drug Administration
Tropical Medicine
Ethics, Pharmacy
Drug Industry
Off-Label Use
Is orphan drug status beneficial to tropical disease control? Comparison of the American and future European orphan drug acts. (1/70)
OBJECTIVES To quantify past outcomes of tropical pharmacology research and development (R & D) and to assess past benefits of the American orphan drug act and potential benefits of the future European orphan drug regulation on tropical diseases. METHODS: This paper presents two analyses: a 1983-97 retrospective study of the United States Orphan Drug Act concerning rare diseases and a prospective study of the European Proposal for a Regulation Concerning Orphan Drugs and its possible impact on tropical diseases. RESULTS: Different programmes have in the past tried to stimulate R & D in this area, but results remain limited. Of 1450 new chemical entities marketed between 1972 and 1997, 13 were specifically for tropical diseases and considered as essential drugs. Between 1983 & 1997, the US Orphan Drug Act approved 837 drugs and marketing of 152 new molecular entities (NMEs). Three NMEs have been designated for malaria and human African trypanosomiasis. Seven others, already commonly used in tropical diseases, received either orphan designation or an orphan approval for another indication. Pharmaceutical companies benefit from the US framework only when the US market exclusivity clause was applicable. Future European orphan drug regulation appears to be similar to the US Orphan Drug Act. CONCLUSION The orphan drug programmes relating to rare diseases have met with some success. Considering tropical diseases rare diseases seems inadequate to boost pharmaceutical R & D. However, some provisions of the European text may be relevant to tropical diseases, admitting the need for a more specific rule for evaluations of this kind of drug and recognizing the existence of 'diseases of exception'. (+info)Development of orphan vaccines: an industry perspective. (2/70)
The development of vaccines against rare emerging infectious diseases is hampered by many disincentives. In the face of growing in-house expenditures associated with research and development projects in a complex legal and regulatory environment, most pharmaceutical companies prioritize their projects and streamline their product portfolio. Nevertheless, for humanitarian reasons, there is a need to develop niche vaccines for rare diseases not preventable or curable by other means. The U.S. Orphan Drug Act of 1983 and a similar proposal from the European Commission (currently under legislative approval) provide financial and practical incentives for the research and development of drugs to treat rare diseases. In addition, updated epidemiologic information from experts in the field of emerging diseases; increased disease awareness among health professionals, patients, and the general public; a list of priority vaccines; emergence of a dedicated organization with strong leadership; and the long-term pharmacoeconomic viability of orphan products will be key factors in overcoming the complexity of orphan status and the limited need for vaccine. (+info)Functional foods: the Food and Drug Administration perspective. (3/70)
Because the Federal Food, Drug, and Cosmetic Act (FFDCA) does not provide a statutory definition of functional foods, the Food and Drug Administration has no authority to establish a formal regulatory category for such foods. The primary determinant of the regulatory status of a food is its intended use, which is determined largely by the label and labeling information accompanying the product. This information includes nutrient information, nutrient content claims, and various types of health claims. In marketing these foods, manufacturers may come under one of several existing regulatory options. The first decision manufacturers will make that will help determine their product's regulatory status is whether the product is a food or a drug. Thus, manufacturers and retailers have a range of legal and regulatory categories in which their products may be classified. This article describes the definitions provided in the FFDCA for a drug and a food, the safety and labeling requirements of various food categories, and types of possible claims for dietary supplements. (+info)What are today's orphaned vaccines? (4/70)
Development costs for new biological agents are increasing, and the time span from laboratory research to introduction of a product on the world market is becoming ever longer. Complex regulatory requirements add barriers and additional costs to early introduction abroad. This results in reluctance by manufacturers to undertake development of a vaccine that will be used for a tropical disease in only the public sector of a poor country. The chances of recovery of huge investment costs before patents expire are not good, unless such a new vaccine can also be sold at high cost in North America and Europe. These are some of the reasons that we still do not have a modern Japanese encephalitis vaccine or products against malaria and dengue fever. Many tropical countries must find a way to develop their own vaccine production facilities. Innovative help for technology transfer will have to be forthcoming, or many new life-saving products will never bridge the gap between research unit and production. (+info)Initial lessons from public-private partnerships in drug and vaccine development. (5/70)
In recent years, venture capital approaches have delivered impressive results in identifying and funding promising health discoveries and bringing them to market. This success has inspired public sector experiments with "social venture capital" approaches to address the dearth of affordable treatment and prevention for diseases of the developing world. Employing the same focus on well-defined and measurable objectives, and the same type of connections to pool and deploy resources as their for-profit counterparts, social venture capitalists seek to use the tools and incentives of capitalism to solve one of its biggest failures: the lack of drugs and vaccines for diseases endemic to low-income populations. As part of a larger trend of partnerships emerging in health product donation and distribution, public-private partnerships for pharmaceutical development have led research and development (R&D) efforts to generate more accessible and efficacious products for diseases such as malaria, tuberculosis, and AIDS. In this article, three R&D-focused partnerships are explored: the International AIDS Vaccine Initiative; the Medicines for Malaria Venture; and the newly formed Global Alliance for TB Drug Development. The article highlights key elements essential to the success of these ventures. (+info)Building local research and development capacity for the prevention and cure of neglected diseases: the case of India. (6/70)
This paper examines the proposal to build research and development (R&D) capabilities for dealing with neglected infectious and tropical diseases in countries where they are endemic, as a potentially cost- and time-effective way to fill the gap between the supply of and need for new medicines. With reference to the situation in India, we consider the competencies and incentives needed by companies so that their strategy can be shifted from reverse engineering of existing products to investment in R&D for new products. This requires complex reforms, of which the intellectual property rights agreement is only one. We also consider whether Indian companies capable of conducting research and development are likely to target neglected diseases. Patterns of patenting and of R&D, together with evidence from interviews we have conducted, suggest that Indian companies, like multinational corporations, are likely to target global diseases because of the prospect of much greater returns. Further studies are required on how Indian companies would respond to push and pull incentives originally designed to persuade multinational corporations to do more R&D on neglected diseases. (+info)Gleevec for the treatment of chronic myelogenous leukemia: US. Food and Drug Administration regulatory mechanisms, accelerated approval, and orphan drug status. (7/70)
Gleevec (imatinib mesylate), a highly promising new drug for the treatment of chronic myelogenous leukemia in blast crisis, in accelerated phase, and in chronic phase after interferon failure or intolerance, received orphan drug status from the U.S. Food and Drug Administration (FDA) Office of Orphan Products Development on January 31, 2001, and accelerated approval from the FDA for the above three indications on May 10, 2001. The purpose of this report is to summarize FDA regulatory mechanisms, i.e., accelerated approval and orphan drug regulations, that have permitted patients to receive this drug as rapidly as possible. (+info)U.S. Food and Drug Administration drug approval summaries: imatinib mesylate, mesna tablets, and zoledronic acid. (8/70)
The purpose of this report is to summarize information on drugs recently approved by the U.S. Food and Drug Administration. Three drugs have recently been approved: Gleevec (imatinib mesylate) at a starting dose of 400 or 600 mg daily for the treatment of malignant unresectable and/or metastatic gastrointestinal stromal tumors; Mesnex (mesna) tablets as a prophylactic agent to reduce the incidence of ifosfamide-induced hemorrhagic cystitis, and Zometa (zoledronic acid) for the treatment of patients with multiple myeloma and for patients with documented bone metastases from solid tumors, in conjunction with standard antineoplastic therapy. Prostate cancer should have progressed after treatment with at least one hormonal therapy. The recommended dose and schedule is 4 mg infused over 15 minutes every 3-4 weeks. These three drugs represent three different types of drug approval: Gleevec is an accelerated approval and supplemental new drug application (NDA); Mesnex tablets represent an oral formulation of a drug approved 14 years ago as an intravenous formulation, and Zometa represents a standard NDA for a noncytotoxic, supportive-care drug. Information provided includes rationale for drug development, study design, efficacy and safety results, and pertinent literature references. (+info)An "Orphan Drug" is a pharmaceutical agent that is developed to treat a rare medical condition, disorder, or disease that affects a small number of people in comparison to other conditions. In the United States, this is defined as a condition or disease that affects fewer than 200,000 people nationwide. Due to the limited market for these drugs, pharmaceutical companies are often reluctant to invest in their development and production.
"Orphan Drug Production," therefore, refers to the manufacturing process of these rare disease treatments. To encourage the development and production of orphan drugs, governments and regulatory agencies offer incentives such as tax credits, grants, and exclusive marketing rights for a certain period of time. These measures help offset the higher costs and lower profit margins associated with developing and producing orphan drugs, ultimately benefiting patients with rare diseases who often have few or no treatment options available to them.
A rare disease, also known as an orphan disease, is a health condition that affects fewer than 200,000 people in the United States or fewer than 1 in 2,000 people in Europe. There are over 7,000 rare diseases identified, and many of them are severe, chronic, and often life-threatening. The causes of rare diseases can be genetic, infectious, environmental, or degenerative. Due to their rarity, research on rare diseases is often underfunded, and treatments may not be available or well-studied. Additionally, the diagnosis of rare diseases can be challenging due to a lack of awareness and understanding among healthcare professionals.
"Drug approval" is the process by which a regulatory agency, such as the US Food and Drug Administration (FDA), grants formal authorization for a pharmaceutical company to market and sell a drug for a specific medical condition. The approval process is based on rigorous evaluation of clinical trial data to ensure that the drug is safe and effective for its intended use.
The FDA's approval process typically involves several stages, including preclinical testing in the lab and animal studies, followed by three phases of clinical trials in human subjects. The first phase tests the safety of the drug in a small group of healthy volunteers, while the second and third phases test the drug's efficacy and side effects in larger groups of patients with the medical condition for which the drug is intended.
If the results of these studies demonstrate that the drug is safe and effective, the pharmaceutical company can submit a New Drug Application (NDA) or Biologics License Application (BLA) to the FDA for review. The application includes data from the clinical trials, as well as information about the manufacturing process, labeling, and proposed use of the drug.
The FDA reviews the application and may seek input from independent experts before making a decision on whether to approve the drug. If approved, the drug can be marketed and sold to patients with the medical condition for which it was approved. The FDA continues to monitor the safety and efficacy of approved drugs after they reach the market to ensure that they remain safe and effective for their intended use.
Pharmaceutical economics is a branch of economics that focuses on the production and distribution of pharmaceutical products and services. It involves the analysis of various factors that influence the development, pricing, and accessibility of medications, including issues related to healthcare policy, regulation, reimbursement, and market competition.
Pharmaceutical economists study topics such as:
1. The research and development (R&D) process for new drugs, including the costs, risks, and uncertainties associated with bringing a new drug to market.
2. The pricing of pharmaceuticals, taking into account factors such as production costs, R&D expenses, market competition, and the value that medications provide to patients and society.
3. The impact of government regulations and policies on the pharmaceutical industry, including issues related to intellectual property protection, drug safety, and efficacy testing.
4. The role of health insurance and other third-party payers in shaping the demand for and access to pharmaceuticals.
5. The evaluation of pharmaceutical interventions' cost-effectiveness and their impact on healthcare outcomes and patient well-being.
6. The analysis of market structures, competitive dynamics, and strategic decision-making within the pharmaceutical industry.
7. The assessment of globalization, international trade, and cross-border collaboration in the pharmaceutical sector.
Pharmaceutical economics plays a crucial role in informing healthcare policy decisions, improving patient access to essential medications, and promoting sustainable and innovative practices within the pharmaceutical industry.
'Drug legislation' refers to the laws and regulations that govern the production, distribution, sale, possession, and use of medications and pharmaceutical products within a given jurisdiction. These laws are designed to protect public health and safety by establishing standards for drug quality, ensuring appropriate prescribing and dispensing practices, preventing drug abuse and diversion, and promoting access to necessary medications. Drug legislation may also include provisions related to clinical trials, advertising, packaging, labeling, and reimbursement. Compliance with these regulations is typically enforced through a combination of government agencies, professional organizations, and legal penalties for non-compliance.
The United States Food and Drug Administration (FDA) is a federal government agency responsible for protecting public health by ensuring the safety, efficacy, and security of human and veterinary drugs, biological products, medical devices, our country's food supply, cosmetics, and products that emit radiation. The FDA also provides guidance on the proper use of these products, and enforces laws and regulations related to them. It is part of the Department of Health and Human Services (HHS).
Tropical medicine is a branch of medicine that deals with health problems that are prevalent in or unique to tropical and subtropical regions. These regions are typically characterized by hot and humid climates, and often have distinct ecological systems that can contribute to the spread of infectious diseases.
The field of tropical medicine encompasses a wide range of health issues, including:
1. Infectious diseases: Many tropical diseases are caused by infectious agents such as bacteria, viruses, parasites, and fungi. Some of the most common infectious diseases in the tropics include malaria, dengue fever, yellow fever, chikungunya, Zika virus, leishmaniasis, schistosomiasis, and Chagas disease.
2. Neglected tropical diseases (NTDs): A group of chronic infectious diseases that primarily affect poor and marginalized populations in the tropics. NTDs include diseases such as human African trypanosomiasis (sleeping sickness), leprosy, Buruli ulcer, and dracunculiasis (guinea worm disease).
3. Zoonotic diseases: Diseases that are transmitted between animals and humans, often through insect vectors or contaminated food and water. Examples of zoonotic diseases in the tropics include rabies, leptospirosis, and Rift Valley fever.
4. Environmental health issues: The tropical environment can pose unique health challenges, such as exposure to toxic chemicals, heat stress, and poor air quality. Tropical medicine also addresses these environmental health issues.
5. Travel medicine: As global travel increases, there is a growing need for medical professionals who are knowledgeable about the health risks associated with traveling to tropical destinations. Tropical medicine physicians often provide pre-travel consultations and post-travel evaluations for international travelers.
Overall, tropical medicine is an essential field that addresses the unique health challenges faced by populations living in or traveling to tropical and subtropical regions.
Pharmacy ethics refers to the principles that guide the behavior and decision-making of pharmacists and other healthcare professionals involved in the provision of medications and related services. These principles are based on values such as respect for autonomy, beneficence (doing good), non-maleficence (doing no harm), justice, and fidelity (faithfulness to commitments).
Pharmacy ethics encompass a wide range of issues, including:
* Informed consent: ensuring that patients understand the risks and benefits of medications and make voluntary decisions about their use.
* Confidentiality: protecting patient information and respecting privacy.
* Professional competence: maintaining knowledge and skills to provide safe and effective care.
* Conflict of interest: avoiding situations where personal or professional interests could compromise patient care.
* Allocation of resources: making fair and equitable decisions about the distribution of medications and related services.
* End-of-life care: respecting patients' wishes and providing appropriate care at the end of life.
Pharmacy ethics are guided by professional codes of conduct, legal regulations, and ethical theories and principles. Pharmacists and other healthcare professionals are expected to uphold these standards and engage in ongoing reflection and education to promote ethical practice.
The "drug industry" is also commonly referred to as the "pharmaceutical industry." It is a segment of the healthcare sector that involves the research, development, production, and marketing of medications or drugs. This includes both prescription and over-the-counter medicines used to treat, cure, or prevent diseases and medical conditions in humans and animals.
The drug industry comprises various types of organizations, such as:
1. Research-based pharmaceutical companies: These are large corporations that focus on the research and development (R&D) of new drugs, clinical trials, obtaining regulatory approvals, manufacturing, and marketing their products globally. Examples include Pfizer, Johnson & Johnson, Roche, and Merck.
2. Generic drug manufacturers: After the patent for a brand-name drug expires, generic drug manufacturers can produce and sell a similar version of the drug at a lower cost. These companies must demonstrate that their product is bioequivalent to the brand-name drug in terms of safety, quality, and efficacy.
3. Biotechnology companies: These firms specialize in developing drugs using biotechnological methods, such as recombinant DNA technology, gene therapy, or monoclonal antibodies. Many biotech companies focus on specific therapeutic areas, like oncology, immunology, or neurology.
4. Contract research organizations (CROs): CROs provide various services to the drug industry, including clinical trial management, data analysis, regulatory affairs support, and pharmacovigilance. They work with both large pharmaceutical companies and smaller biotech firms to help streamline the drug development process.
5. Drug delivery system companies: These organizations focus on developing innovative technologies for delivering drugs more effectively and safely to patients. Examples include transdermal patches, inhalers, or long-acting injectables.
6. Wholesalers and distributors: Companies that purchase drugs from manufacturers and distribute them to pharmacies, hospitals, and other healthcare providers.
The drug industry plays a crucial role in improving public health by discovering, developing, and delivering new treatments for various diseases and medical conditions. However, it is also subject to criticism and regulation due to concerns about high drug prices, marketing practices, and the potential for conflicts of interest between industry and healthcare professionals.
Off-label use refers to the practice of prescribing or using pharmaceutical drugs for purposes, dosages, patient populations, or routes of administration that are not included in the approved labeling of the drug by the regulatory authority, such as the U.S. Food and Drug Administration (FDA). It is not illegal or unethical for physicians to prescribe medications off-label when they judge that it is medically appropriate for their patients. However, manufacturers are prohibited from promoting their drugs for off-label uses.
A patent, in the context of medicine and healthcare, generally refers to a government-granted exclusive right for an inventor to manufacture, use, or sell their invention for a certain period of time, typically 20 years from the filing date. In the medical field, patents may cover a wide range of inventions, including new drugs, medical devices, diagnostic methods, and even genetic sequences.
The purpose of patents is to provide incentives for innovation by allowing inventors to profit from their inventions. However, patents can also have significant implications for access to medical technologies and healthcare costs. For example, a patent on a life-saving drug may give the patent holder the exclusive right to manufacture and sell the drug, potentially limiting access and driving up prices.
It's worth noting that the patent system is complex and varies from country to country. In some cases, there may be ways to challenge or circumvent patents in order to increase access to medical technologies, such as through compulsory licensing or generic substitution.
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