The pharmaceutical industry invests heavily in research and development, with biopharmaceutical companies spending $65.2 billion annually on R&D. Developing a new drug takes an average of 10-15 years and costs $2.6 billion. Only about 20% of experimental drugs make it through clinical trials to receive FDA approval. After approval, many drugs fail to become commercial successes. However, post-approval research on existing drugs often leads to new uses that provide important medical advances. The industry and government play complementary roles in supporting medical research.
The orphan drug area is relatively new but fast growing. Over the next weeks, Black Swan Consulting will summarise information on this class of drug products. Please also see http://black-swan-consulting.com/what-is-cooking/Orphan-drugs.
Please share this webinar with anyone who may be interested!
Watch all our webinars: https://www.youtube.com/playlist?list=PL4dDQscmFYu_ezxuxnAE61hx4JlqAKXpR
In this webinar:
● Primer for attendees attending the November 15-16 Drug Pricing Policy Summit
● Broad conceptual blueprint of federal and provincial/territorial public health policy structures across Canada
● Description of legal frameworks, government responsibility centres and their mandates for treatment access, with reference to specific opportunities for patient engagement
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Why is the orphan drug area receiving increasing attention? In this presentation you will find the primary reasons explained.
Over the next weeks, Black Swan Consulting will summarise information on this class of drug products. Please also see http://black-swan-consulting.com/what-is-cooking/Orphan-drugs.
The orphan drug area is relatively new but fast growing. Over the next weeks, Black Swan Consulting will summarise information on this class of drug products. Please also see http://black-swan-consulting.com/what-is-cooking/Orphan-drugs.
Please share this webinar with anyone who may be interested!
Watch all our webinars: https://www.youtube.com/playlist?list=PL4dDQscmFYu_ezxuxnAE61hx4JlqAKXpR
In this webinar:
● Primer for attendees attending the November 15-16 Drug Pricing Policy Summit
● Broad conceptual blueprint of federal and provincial/territorial public health policy structures across Canada
● Description of legal frameworks, government responsibility centres and their mandates for treatment access, with reference to specific opportunities for patient engagement
View the video: https://youtu.be/X9AB70om-Dw
Follow our social media accounts:
Twitter - https://twitter.com/survivornetca
Facebook - https://www.facebook.com/CanadianSurvivorNet
Pinterest - https://www.pinterest.com/survivornetwork
YouTube - https://www.youtube.com/user/Survivornetca
Why is the orphan drug area receiving increasing attention? In this presentation you will find the primary reasons explained.
Over the next weeks, Black Swan Consulting will summarise information on this class of drug products. Please also see http://black-swan-consulting.com/what-is-cooking/Orphan-drugs.
The Biologics Market was worth USD 267.58 billion in 2014 and is expected to reach approximately USD 373.66 billion by 2023, while registering itself at a compound annual growth rate (CAGR) of 3.78% during the forecast period. Biologics are medicates as hereditarily built proteins, got from human qualities. According to the US FDA, biologics can be made out of sugars, proteins, or nucleic acids or complex blends of these substances, or might live elements, for example, cells and tissues. The biologic medications are taken from an assortment of regular sources, for example, people, creatures, or microorganisms and comprise of items, for example, antibodies, blood and blood parts, allergenic, substantial cells, quality treatment, tissues, and recombinant remedial proteins. Propelled biotechnology strategies and complex procedures are utilized to produce biologics. They are at the cutting edge of biomedical research.
Regulatory requirements for orphan drugs delivery, Prof. Dr. Basavaraj K. Nanjwade, KLE University College of Pharmacy, Belgavi/Belgaum, Karnataka, India.
Mastering Regulatory Approval in New Orphan Drug MarketsLewis Lau
Presented at DIA 2015 Annual Meeting. A symposum titled "A Global Update on Orphan Drug" chaired by Mr Noriaki Murao
http://www.diaglobal.org/en-US/Flagship-Meetings/DIA-Annual-Meeting/Meeting-Program/Find-Sessions-and-Presentations/Event-Details.aspx?productID=3803687&eventType=Symposium&title=A%20Global%20Update%20on%20Orphan%20Drugs
This symposium addresses the current status and forthcoming activities related to orphan drugs in North America, EU and Japan. Orphan drug development is considered essential in these regions, and the various provisions to accelerate the development of orphan drugs have been implemented. However, some challenges still remain for the companies and the agencies wishing to pursue development and approval of orphan drugs in these regions.
Orphan Drugs – the Challenges and Benefits of Navigating FDA’s Regime Governi...Michael Swit
Webinar sponsored by The Weinberg Group on Orphan Drugs, covering these topics:
The Basics of the Orphan Drug Act
Benefits of Orphan Drug status
Exclusivity
Protocol assistance, tax credits, and research grants
When is an indication is “rare”?
Orphan Drug Designation Requests – ensuring yours
robust and persuasive
Approval criteria for orphan products – how they
compare to non-orphan products
Challenges in the Orphan Drug Process
The annual 2013 Pharmaceutical Industry Profile provides an overview of the sector, highlighting the latest medical advances, the impact of biopharmaceutical companies on the economy and the future of innovation.
2016-11-28 Mentlife seminar: Pharmaceutical Drug Development; An Overall Pers...MentLife
This seminar provided an understanding of modern pharmaceutical drug development – the different phases of drug development and insight into different jobs.
Connecting the Dots for Fast-Track Approval for Rare Disease and Orphan DrugMedpace
Managing a rare disease or orphan drug clinical trial has several challenges. Be prepared to understand and take away real world tactical lessons to expedite these studies:
•What are the new promising techniques in clinical studies for Rare Disease research? How are the innovative areas of adaptive strategies and translational medicine being employed?
• How do patient registries and natural history studies provide insights and patients for these studies?
• What quality of life issues play into patient retention in these studies?
• How can regulatory challenges from submissions to drug approvals be met using integrated global knowledge and systems to keep a project on track?
Over 30 years after the Orphan Drug Act was passed, orphan drugs continue to be a lucrative market for pharma companies. Although orphan diseases affect small populations, these treatments address a high unmet need and also benefit from commercially attractive pricing structures and additional regulatory benefits.
Full graphic: http://www.isrreports.com/free-resources/5408/
2014 Profile: Biopharmaceutical Research IndustryPhRMA
Biopharmaceutical science is a complex, collaborative, resource-intensive enterprise. It requires a highly skilled workforce, sustained investment, and long-term vision. Critical to its success are policies and regulations that foster innovation and broad access to new medicines. By working together—on the science, the research and the policies—we
can help ensure that medicines live up to patients’ hope for new solutions to our greatest health care challenges.
The IOSR Journal of Pharmacy (IOSRPHR) is an open access online & offline peer reviewed international journal, which publishes innovative research papers, reviews, mini-reviews, short communications and notes dealing with Pharmaceutical Sciences( Pharmaceutical Technology, Pharmaceutics, Biopharmaceutics, Pharmacokinetics, Pharmaceutical/Medicinal Chemistry, Computational Chemistry and Molecular Drug Design, Pharmacognosy & Phytochemistry, Pharmacology, Pharmaceutical Analysis, Pharmacy Practice, Clinical and Hospital Pharmacy, Cell Biology, Genomics and Proteomics, Pharmacogenomics, Bioinformatics and Biotechnology of Pharmaceutical Interest........more details on Aim & Scope).
All manuscripts are subject to rapid peer review. Those of high quality (not previously published and not under consideration for publication in another journal) will be published without delay.
The Biologics Market was worth USD 267.58 billion in 2014 and is expected to reach approximately USD 373.66 billion by 2023, while registering itself at a compound annual growth rate (CAGR) of 3.78% during the forecast period. Biologics are medicates as hereditarily built proteins, got from human qualities. According to the US FDA, biologics can be made out of sugars, proteins, or nucleic acids or complex blends of these substances, or might live elements, for example, cells and tissues. The biologic medications are taken from an assortment of regular sources, for example, people, creatures, or microorganisms and comprise of items, for example, antibodies, blood and blood parts, allergenic, substantial cells, quality treatment, tissues, and recombinant remedial proteins. Propelled biotechnology strategies and complex procedures are utilized to produce biologics. They are at the cutting edge of biomedical research.
Regulatory requirements for orphan drugs delivery, Prof. Dr. Basavaraj K. Nanjwade, KLE University College of Pharmacy, Belgavi/Belgaum, Karnataka, India.
Mastering Regulatory Approval in New Orphan Drug MarketsLewis Lau
Presented at DIA 2015 Annual Meeting. A symposum titled "A Global Update on Orphan Drug" chaired by Mr Noriaki Murao
http://www.diaglobal.org/en-US/Flagship-Meetings/DIA-Annual-Meeting/Meeting-Program/Find-Sessions-and-Presentations/Event-Details.aspx?productID=3803687&eventType=Symposium&title=A%20Global%20Update%20on%20Orphan%20Drugs
This symposium addresses the current status and forthcoming activities related to orphan drugs in North America, EU and Japan. Orphan drug development is considered essential in these regions, and the various provisions to accelerate the development of orphan drugs have been implemented. However, some challenges still remain for the companies and the agencies wishing to pursue development and approval of orphan drugs in these regions.
Orphan Drugs – the Challenges and Benefits of Navigating FDA’s Regime Governi...Michael Swit
Webinar sponsored by The Weinberg Group on Orphan Drugs, covering these topics:
The Basics of the Orphan Drug Act
Benefits of Orphan Drug status
Exclusivity
Protocol assistance, tax credits, and research grants
When is an indication is “rare”?
Orphan Drug Designation Requests – ensuring yours
robust and persuasive
Approval criteria for orphan products – how they
compare to non-orphan products
Challenges in the Orphan Drug Process
The annual 2013 Pharmaceutical Industry Profile provides an overview of the sector, highlighting the latest medical advances, the impact of biopharmaceutical companies on the economy and the future of innovation.
2016-11-28 Mentlife seminar: Pharmaceutical Drug Development; An Overall Pers...MentLife
This seminar provided an understanding of modern pharmaceutical drug development – the different phases of drug development and insight into different jobs.
Connecting the Dots for Fast-Track Approval for Rare Disease and Orphan DrugMedpace
Managing a rare disease or orphan drug clinical trial has several challenges. Be prepared to understand and take away real world tactical lessons to expedite these studies:
•What are the new promising techniques in clinical studies for Rare Disease research? How are the innovative areas of adaptive strategies and translational medicine being employed?
• How do patient registries and natural history studies provide insights and patients for these studies?
• What quality of life issues play into patient retention in these studies?
• How can regulatory challenges from submissions to drug approvals be met using integrated global knowledge and systems to keep a project on track?
Over 30 years after the Orphan Drug Act was passed, orphan drugs continue to be a lucrative market for pharma companies. Although orphan diseases affect small populations, these treatments address a high unmet need and also benefit from commercially attractive pricing structures and additional regulatory benefits.
Full graphic: http://www.isrreports.com/free-resources/5408/
2014 Profile: Biopharmaceutical Research IndustryPhRMA
Biopharmaceutical science is a complex, collaborative, resource-intensive enterprise. It requires a highly skilled workforce, sustained investment, and long-term vision. Critical to its success are policies and regulations that foster innovation and broad access to new medicines. By working together—on the science, the research and the policies—we
can help ensure that medicines live up to patients’ hope for new solutions to our greatest health care challenges.
The IOSR Journal of Pharmacy (IOSRPHR) is an open access online & offline peer reviewed international journal, which publishes innovative research papers, reviews, mini-reviews, short communications and notes dealing with Pharmaceutical Sciences( Pharmaceutical Technology, Pharmaceutics, Biopharmaceutics, Pharmacokinetics, Pharmaceutical/Medicinal Chemistry, Computational Chemistry and Molecular Drug Design, Pharmacognosy & Phytochemistry, Pharmacology, Pharmaceutical Analysis, Pharmacy Practice, Clinical and Hospital Pharmacy, Cell Biology, Genomics and Proteomics, Pharmacogenomics, Bioinformatics and Biotechnology of Pharmaceutical Interest........more details on Aim & Scope).
All manuscripts are subject to rapid peer review. Those of high quality (not previously published and not under consideration for publication in another journal) will be published without delay.
Chapter 19Clinical Trials Clinical TrialsThe history EstelaJeffery653
Chapter 19
Clinical Trials
Clinical Trials
“The history of the last 20 years is one of crises with drugs and medical devices, many approved despite the objections of the FDA’s own scientists.”
— Sidney M. Wolfe
Lecture Overview
Research and Development Investments Fund a Complex Multistage Pathway
Clinical Trials of Generic Drugs
Health Risk Assessments
Expanded Access Protocols
Termination of Clinical Trials
Observational Studies
International Clinical Trials
Informed Consent in General
Transparency and Full Disclosure in Clinical Testing
Financial Conflicts of Interest
Commitment to the Life Sciences
Source: Hammaker, D. K., & Knadig, T. M. with Tomlinson, S.J. (2017). Clinical trials. In Health care management and the law: Principles and applications (pp. 389-412). (2nd Ed.) Burlington, MA: Jones & Bartlett Learning.
Research and Development Investments Fund a Complex Multistage Pathway
Some major expenses are research materials, advanced computers, and other highly sophisticated machines that support research activities, and salaries of scientists. Stage specific activities include:
Drug discovery
Preclinical testing
Clinical trials
Approval by the FDA
Post marketing surveillance
Research and Development Investments Fund a Complex Multistage Pathway
Drug Discovery:
While most compounds will never be approved for use, each one is evaluated to determine its potential value compared to existing therapies, complexity of large scale manufacturing, and other factors.
Preclinical Testing:
Candidate drugs from the discovery stage receive 1 to 3 years of extensive testing to assess safety and show biological activity against a disease.
Chemical tests establish the purity, stability, and shelf life of a compound.
Manufacturing tests determine mass production of the drug.
Pharmaceutical development studies explore dosing, packaging, and formulation of the drug.
Research and Development Investments
Fund a Complex Multistage Pathway: Clinical Trials
For drugs in development, there are low odds of reaching the market.
While Phase I, II, and III of studies are taking place, research investigators are also conducting toxicity tests and other long-term safety evaluations, evaluating dosage forms, planning for mass production, designing packaging, and preparing the extensive application required for FDA approval.
One out of five drugs that enter clinical testing is never approved by the FDA:
20% of the drugs that enter Phase I are approved to enter Phase II
30% of the drugs that enter Phase II are approved to enter Phase III
60% of the drugs that enter Phase III are approved for a new drug application
80% of the new drug applications are approved by the FDA for market entry
Research and Development Investments Fund a Complex Multistage Pathway
Approval by the U.S. Food and Drug Administration
According to the FDA, the documentation required in a new drug application is supposed to tell the whole story of a ...
Global gene therapy market & pipeline insightKuicK Research
“Global Gene Therapy Market & Pipeline Insight” Market Highlight:
Gene Therapy Market Overview
Significance of Gene Therapy in Cancer Therapeutics
Current Applications of Gene Therapy to Cancer Treatment
Gene Therapy Market Dynamics: Drivers, Challenges & Future Outlook
FDA & AMA Guidelines for Gene Therapy
Gene Therapy Pipeline by Phase, Indications, Country & Company
Gene Therapy Pipeline: 246 Drugs
Marketed Gene Therapy: 2 (Gendicine & Rexin-G)
Cancer Gene Therapy Pipeline: 69 Drugs
Medication nonadherence cost and noncompliance in clinical trialsSynegys
Drug development has reached over $2.6 B and is driven by a clinical trial's success rate, out-of-pocket study costs and study timescales. However, medication nonadherence is a hidden cost which heavily influences these cost drivers. We discuss how medication nonadherence introduces data variability, requiring trial managers to enrol more patients to maintain statistical power, which in turn extends trial timelines. Cost savings are described based on improving study noncompliance with a compliance tool such as Synegys' mComply. This mHealth tool reduces costs as a result of improved statistical power, lower enrollment and shorter trial duration.
Determinants of New Molecular Entity Approval by United States Food & Drug Ad...inventionjournals
This paper analyzes the relationship between research-based pharmaceutical companies’ R&D productivity, patent, pivotal trial and drug development strategy with the number of NME approval by U.S. FDA. The model was estimated using annual data, gathered from ten large pharmaceutical companies in the world. The regression analysis used pooled regression with Estimated Generalized Least Squares (EGLS) method. The result showed that R&D productivity, patent, pivotal trial, and drug development strategy are statistically significant in increasing the number of NME approval in research-based pharmaceutical companies. The relative order of significance in influencing the number of NME approval was patent, development strategy, R&D productivity, and pivotal trial.
Global gene therapy market & pipeline insightKuicK Research
“Global Gene Therapy Market & Pipeline Insight” Market Highlight:
Gene Therapy Market Overview
Significance of Gene Therapy in Cancer Therapeutics
Current Applications of Gene Therapy to Cancer Treatment
Gene Therapy Market Dynamics: Drivers, Challenges & Future Outlook
FDA & AMA Guidelines for Gene Therapy
Gene Therapy Pipeline by Phase, Indications, Country & Company
Gene Therapy Pipeline: 246 Drugs
Marketed Gene Therapy: 2 (Gendicine & Rexin-G)
Cancer Gene Therapy Pipeline: 69 Drugs
1. Biopharmaceutical Companies’ Investment in R&D Increasing Steadily Total Biopharmaceutical Company R&D and PhRMA Member R&D: 1995–20081 The pharmaceutical industry is one of the most research-intensive industries in the United States. Pharmaceutical firms invest as much asfive times more in research and development, relative to their sales, than the average U.S. manufacturing firm.2 — Congressional Budget Office Sources: 1Burrill & Company, analysis for PhRMA, 2005–2009; Pharmaceutical Research and Manufacturers of America, PhRMA Annual Member Survey (Washington, DC: PhRMA, 1981-2009); 2CBO, Research and Development in the Pharmaceutical Industry, 2006.
2. Clinical Research Clinical Research Translational Research Translational Research Basic Research Basic Research Federal and Industry Roles in Research and Development Government and biopharmaceutical industry research are complementary Private Sector – $65.2B1 There is an ecosystem of science and biotechnology. Public organizations, patient organizations, universities, Congress, FDA, all of this is an ecosystem that is envied in the rest of the world. – E. Zerhouni, Director of NIH NIH3– $29.4B total – $20.1B research Sources: 1Burrill & Company, analysis for PhRMA, 2005–2009 (Includes PhRMA research associates and nonmembers) in PhRMA, “Profile 2008, Pharmaceutical Industry;” PhRMA, “PhRMA Annual Membership Survey,” 1996-2009; 2Adapted from E. Zerhouni, Presentation at Transforming Health: Fulfilling the Promise of Research, 2007; 3NIH Office of the Budget, “FY 2009 President’s Budget Request Tabular Data”, http://officeofbudget.od.nih.gov/ui/2008/tabular%20data.pdf
3. Medical Research in the U.S. Outpaces the Rest of the World …in the late 1980s only 41% of the top 50 innovative drugs were of American origin, in the late 1990s…[it had] climbed to 62%.... In 1990, the pharmaceutical industry spent 50% more on research in Europe than in the U.S. In 2001, the situation was reversed with 40% more spent in the U.S.2 –Gunter Verheugen, Vice-President of the European Commission for Enterprise and Industry Number of Compounds in Development, by Region,* 1997–2007 *Note: Reflects the number of compounds in clinical trials or awaiting approval as of June of each year. Compounds in development for multiple regions are counted in each region for which regulatory approval is sought, and multiple indications are counted only once. Sources: 1Adis R&D Insight, February 2009; 2G. Verheugen, “Address to the Concluding Session of the European Track” (Lyon) 2005.
4. Post-MarketingSurveillance Drug Discovery Preclinical Clinical Trials FDA Review Scale-Up to Mfg. 5 250 ~ 5,000 – 10,000 COMPOUNDS ONE FDA-APPROVED DRUG PRE-DISCOVERY PHASE 3 PHASE 1 PHASE 2 NDA SUBMITTED IND SUBMITTED NUMBER OF VOLUNTEERS 20–100 100–500 1,000–5,000 0.5 – 2 YEARS INDEFINITE 6 – 7 YEARS 3 – 6 YEARS Drug Development takes longer that it did in the past Developing a new medicine takes an average of 10–15 years; the Congressional Budget Office reports that “relatively few drugs survive the clinical trial process” Sources: Drug Discovery and Development: Understanding the R&D Process, www.innovation.org; CBO, Research and Development in the Pharmaceutical Industry, 2006.
5. The Cost of Developing a New Drug Has Greatly Increased Cost to Develop One New Drug1 Billions (Constant Dollars, Year 2000) Sources: 1J. DiMasi and H. Grabowski, "The Cost of Biopharmaceutical R&D: Is Biotech Different?," Managerial and Decision Economics, 2007; J. DiMasi et al., “The Price of Innovation: New Estimates of Drug Development Costs,” Journal of Health Economics, 2003.
6. Increasing Complexity of Clinical Trials During the last decade clinical trial designs andprocedures have become much more complex, demanding more staff time and effort, and discouraging patient-enrollment and retention Definitions: Procedures: include lab & blood work, routine exams, x-rays & imaging, questionnaire & subjective assessments, invasive procedures, heart assessment, etc. Protocol: the clinical-trial design plan Enrollment rate: the percentage of volunteers meeting the increasing number of protocol eligibility criteria (percentage screened who were then enrolled) Retention rates: the percentage of volunteers enrolled who then completed the study; declining retention rates mean that firms must enroll more patients initially and/or recruit more patients during the trial. Source: Tufts Center for the Study of Drug Development, “Growing Protocol Design Complexity Stresses Investigators, Volunteers,” Impact Report, 2008.
7. Probability of Success for Investigational Drugs Is Small Approximately 20% of self-originated new drugs that enter clinical testing will receive U.S. marketing approval.1 Clinical Approval Success Rates by Therapeutic Class1 Source: 1Tufts Center for the Study of Drug Development, “New drugs entering clinical testing in top 10 firms jumped 52% in 2003-05,” Impact Report, 2006.
8. Cumulative Approvals for Medicines: 1990–2008 Many examples exist of major therapeutic gains achieved by the industry in recent years… anecdotal and statistical evidence suggests that therapid increases that have been observed in drug-related R&D spending have been accompanied bymajor therapeutic gains in available drug treatments.2 —Congressional Budget Office Number of New Drug Approvals Note: New medicines include New Drug Applications and Biologics License Applications Sources: 1Food and Drug Administration, www.fda.gov; PhRMA, “New Drug Approvals,” Reports 1991-2008; “New Molecular Entities Approved in 2008,” The Pink Sheet, January 2009; 2CBO, Research and Development in the Pharmaceutical Industry, 2006.
9. Even After Approval, Few Medicines Are a Commercial Success Lifetime Sales Compared to Average R&D Costs After-Tax Present Value of Sales (Millions of 2000 Dollars) New Rx Drugs Introduced Between 1990 and 1994, Grouped by Tenths, by Lifetime Sales Note: Drug development costs represent after-tax out-of-pocket costs in 2000 dollars for drugs introduced from 1990–94. The same analysis found that the total cost of developing a new drug was $1.3 billion in 2006. Average R&D Costs include the cost of the approved medicines as well as those that fail to reach approval. Sources: J. Vernon et al., “Drug Development Costs when Financial Risk is Measured Using the Fama-French Three Factor Model,” Unpublished Working Paper, 2008; J. DiMasi and H. Grabowski, “The Cost of Biopharmaceutical R&D: Is Biotech Different?,” Managerial and Decision Economics, 2007.
10. Importance of Post-Approval R&D 2003 Research into new uses for approved biologics is an important source of medical advances 1998 For use in combination with Rebetol to treat pediatric hepatitis C.2 As of 2007, “47%of BLAs for recombinant DNA products and monoclonal antibodies regulated by CDER ha[d] at least one additional FDA-approved indication after the initial approval…. One-third of all additional indications of biotechnology-derived protein drugs studied were approved by the FDA more than seven years after approval of the initial indication. —Boston Consulting Group 1997 For use in combination with Rebetol capsules for chronic viral hepatitis C in patients with compensated liver disease who have relapsed following alfa Interferon therapy. To treat hepatitis B in pediatric patients.2 For use in combination with Rebetold capsules for chronic viral hepatitis C in patients with compensated liver disease previously untreated with alfa Interferon therapy. 1995 For treatment of clinically aggressive follicular non-Hodgkin’s lymphoma in conjunction with antracycline-containing combination therapy. 1993 For use adjuvant to surgical treatment for malignant melanoma for those patients free of disease but at high risk for systemic recurrence within 56 days of surgery. 1992 For use in condylomata acuminata with podophyllin. 1991 For treatment of hepatitis B in patients 18 years or older with compen-sated liver disease. 1Orphan Indication 2Pediatric Indication 1988 To treat patients with chronic hepatitis non-A, non-B/C with compensated liver diseases and history of blood or blood product exposure and/or who are HCV anti-body positive. For intralesional treatment of select cases of condylomata acuminata involving external surfaces of genital and perianal areas. For treatment of select patients with AIDS-related Kaposi’s sarcoma¹ 1986 Example: Intron-A (interferon-alfa-2b) FDA Approval of Original Indication: To treat hairy cell leukemia Sources: Boston Consulting Group, “Continued Development of Approved Biological Drugs,” White Paper, 2007; PhRMA, “Post-Approval Research on Biotech Medicines Leads to Key Medical Advances,” PhRMA Report, 2007.