Orphans and Incentives Developing Technology to Address Emerging Infections (1997) / Chapter Skim
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Appendix C
Appendix C
Pages 63-100
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From page 63... ...
The statute provides that non-profit and small-business recipients of government grants, contracts, or cooperative agreements are allowed to retain title to the inventions made with that support, provided that they comply with the act's procedural requirements and take steps to achieve practical application of the inventions. With relatively few restrictions, recipients of federal funding may commercialize such inventions themselves or license them to others for commercialization (Sherman end Englund, 1995~.
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From page 64... ...
The act was intended to prompt the pharmaceutical industry to develop drugs for use against rare diseases affecting fewer than 200,000 people in the United States. The incentives for industry include research grants, a 50-percent income-tax credit on most clinical research expenditures, assistance with FDA approval, and exclusive license to market the product for 7 years immediately
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From page 65... ...
In the 17 years prior to the act, industry sponsored 34 marketed and 24 experimental orphan drugs; in the first 7 years since it was passed, it sponsored 39 of 42 marketed orphan products (Ashbury, 1991~. In recent years, although a bill has been introduced repeatedly in the Senate to extend the orphan drug tax credit permanently, it has not been passed.
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From page 66... ...
B Metheny, ed. Senate bill would extend permanently the orphan drug tax credit.
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From page 67... ...
Outcomes As of 1993, there appeared to have been a drop in the number of vaccinerelated lawsuits and increased activity in vaccine-related research and development (IOM, 1993~. However, at the time, none of the companies that had dropped out of vaccine manufacturing in the 1970s and 1980s had returned, a situation that has since changed (Mercer Management Consulting, 19949.
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From page 68... ...
Summary of UNICEF Study: A Commercial Perspective on Vaccine Supply. New York: Mercer Management Consulting.
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From page 69... ...
. Small Business Innovation Research Program Background The Small Business Innovation Research Program (SBIR)
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From page 70... ...
The recipient may copyright and publish material developed under SBIR grants. Outcomes For the 1997 fiscal year, agencies with extramural research and development budgets over $100 million must reserve 2.5 percent thereof for SBIR grants under the terms of the program's authorization (~Washington Fax, 29 January 1997~.
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From page 71... ...
as a threeyear pilot, modeled after the Small Business Innovation Research Program (SBIR)
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From page 72... ...
The pharmaceutical industry enjoys a further benefit: When a patent claims that a human drug product has
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From page 73... ...
The Drug Price Competition and Patent Term Restoration Act of 1984 (PL 98-417) was enacted to restore part of the patent life lost during the regulatory approval process; it allows the extension of the patent term equal to the total time taken by the FDA to review the new drug application plus one-half of the clinical testing time, but not beyond 14 years of effective patent life (IOM, 1993~.
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From page 74... ...
Another issue in developing a new international intellectual property rights agreement is the filing system for applications. Most countries besides the United States award patent rights based on the earliest application filed for a certain invention.
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From page 75... ...
Since no federal product liability law exists in the United States, state statutes and common law govern liability issues. Companies therefore tend to rely on FDA regulations in asserting compliance and seeking a defense against allegations in product liability suits.
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From page 76... ...
In order to have some compensation, an IOM Committee to Review the Fialuridine (FIAU) Clinical Trials recommended the establishment of a system of no-fault compensation for research injury by government, sponsors, or some combination of both (IOM, 1995~.
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From page 77... ...
Small businesses have been unable to proceed with clinical testing of devices developed in the Small Business Innovation Research Program (SBIR) program owing to the inability to obtain product liability insurance.
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From page 78... ...
June 6, 1995. Prescription Drug User Fee Act Background Enacted in 1992, the Prescription Drug User Fee Act authorizes the FDA to charge user fees to prescription drug and biologic drug industries for review of new drug applications or product license applications.
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From page 79... ...
INDs may receive FDA approval once a new drug application (NDA) has been submitted, initiating a process that can take up to 5 years for clinical trials to be conducted before final approval is granted.
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From page 80... ...
Drug Approval Process Alternatives: Parallel Tracking Background The AIDS epidemic spawned new interest in the question of quicker availability of investigational new drugs (lNDs) and, in 1989, Anthony Fauci, Director of the National Institute of Allergy and Infectious Diseases?
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From page 81... ...
Other unresolved issues include developing procedures to identify and evaluate unexpected deaths or serious illnesses resulting from the use of the experimental drugs; deciding who would pay the costs of associated medical care, administering the drugs, and monitoring laboratory procedures; determining the level of liability of sponsoring drug companies or physicians participating in the program; establishing specific criteria for determining patient eligibility; and developing informed consent requirements for participating patients (GWU, 1990~. References Miller Jones J
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From page 82... ...
Furthermore, the approval time for SNDAs has been criticized as slow and therefore as impeding physician awareness and making it possible for thirdparty payers to deny payment for therapies, even though they may be considered standard therapy for a given indication, even absent formal clinical trials. The Prescription Drug User Fee Act of 1992 has worked to reduce the amount of time it takes the FDA to approve efficacy supplements by providing the FDA with additional resources and holding it accountable for rapid action on efficacy supplements.
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From page 83... ...
Jagger S Prescription Drugs: Implications of Drug Labeling and Off-Label Use.
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From page 84... ...
A management analysis commissioned by UNICEF concluded that "the benefits of tiered pricing extend beyond the developing world to the manufacturers and the industrialized countries," since the latter would be forced to pay higher prices if the very large, high-volume UNICEF market did not exist. The volumes involved are seen as sufficient to "move the market" (Mercer Management Consulting, 19949.
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From page 85... ...
The end result predicted at the time was a diminution in industry cash flow of from $90 million to $120 million a year, with funds available for vaccine R&D dropping as a consequence by $30 million to $40 million. The Mercer analysis noted that this shift already establishes the federal government as the driver of the vaccine business (Mercer Management Consulting, 19964; if the government became the sole purchaser of all pediatric vaccines, the little competition that exists among vaccine manufacturers in the United States would diminish even further (IOM, 19939.
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From page 86... ...
companies to compete more effectively in the global marketplace. The R&D tax credit lowers the cost of investment in qualified research activities by providing a 20 percent tax credit on incremental R&D spending.
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From page 87... ...
tax code was enacted in 1921 to stimulate the economy of Puerto Rico through provision of tax incentives for companies that set up facilities and provided jobs in the Commonwealth. The tax credit was perceived as beneficial to the island's economy, accounting for one-third of total employment, and was also credited with enhancing U.S.
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From page 88... ...
The government of Puerto Rico is pursuing congressional passage of a similar proposal in 1997. Issues Limitations on these tax credits enacted by Congress in recent years, with consequent estimated revenue gains, have been based primarily on congressional efforts to reduce the U.S.
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From page 89... ...
, and they continue to be able to generate mass awareness and attract research money, money that has both expedited research, either as start-up funds or as bridge Finding that carries researchers through lag times while awaiting approval of larger federal grants. The formation of the National Breast Cancer Coalition (NBCC)
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From page 90... ...
The first tactic of AIDS activist groups was to expand access to experimental drugs by questioning the fundamental regulatory core of the drug development process and the system of increased regulation that had been developing since the 1962 thalidomide tragedy. Although that tragedy did not involve U.S.-based companies, after 1962 Congress expanded the role of the FDA, giving it more responsibility for policy decisions and requiring it to evaluate the efficacy as well as the safety of pharmaceutical products.
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From page 91... ...
Issues There are fears that relaxation of drug access regulations, now requiring only that a drug show promising test results and no sign of major toxicity, may be excessive. Another concern is that modification of the drug importation ban makes the prospects of clinical trials much more difficult, since the potential combination of drugs makes true evaluation of efficacy impossible (Rothman end Edger, 19913.
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From page 92... ...
~ The Juvenile Diabetes Foundation (JDF) , founded in 1970 by the parents of children with diabetes, is a not-for-profit, voluntary health organization with U.S.
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From page 93... ...
New York, 1996. Juvenile Diabetes Foundation International (JDFI)
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From page 94... ...
In 1994, Seoul, South Korea, was selected to house the IVI, at present the only international research center dedicated to vaccine research and development for the developing world. The institute will not produce vaccines but will work cooperatively with international organizations, national institutions, nongovernmental organizations, industry, and health and vaccine specialists to catalyze, facilitate, and stimulate new vaccine research and introduction by other concerned organizations (IVI Home Page, 1997~.
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From page 95... ...
Background The International AIDS Vaccine Initiative (IAVI) was established in 1996, with the mission of ensuring development of safe, effective HIV vaccines for worldwide use.
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From page 96... ...
expanded safety studies of live-attenuated HIV. The organization is seeking partners to collaborate in preclinical trials with HIV-DNA vaccines and to address safety concerns and possible risks and benefits of pursuing human trials using a live-attenuated HIV vaccine.
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From page 97... ...
Those companies formed the "InterCompany Collaboration for AIDS Drug Development," with the purpose of better enabling each of them to independently evaluate new antiviral investigational drugs for potential clinical benefit, either alone or in combination with marketed or investigational drugs of other companies. By sharing relevant preclinical and clinical data (including negative data on antiviral compounds that have failed in drug development)
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From page 98... ...
The Subcommittee is developing standardized cytotoxicity and primary isolate susceptibility protocols, as well as a database of HIV antiviral resistance. The Scientific Panel also appointed a Clinical Trial Subcommittee with the primary objective of developing a consensus protocol for clinical evaluation of multi-drug combinations of antiviral agents, both investigational and marketed.
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From page 99... ...
in 1993. SIREVA is an international programming, administrative, and coordinating initiative to improve the quality, effectiveness, and cost of vaccines by working to coordinate all stages and participants involved in vaccine development and production for the people of Latin America and the Caribbean.
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From page 100... ...
References CVI Forum. Special Vaccine Industry Issue (11~.
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