Vaccine Policy Issues

CRS Report for Congress
Vaccine Policy Issues
Updated May 19, 2005
Susan Thaul
Specialist in Social Legislation
Domestic Social Policy Division

Congressional Research Service ˜ The Library of Congress

Vaccine Policy Issues
Summary
This report’s focus is on vaccination, one of the most cost-effective methods
available to prevent infectious diseases. Whether a vaccine’s target is naturally
occurring or present because of hostile intent, the issues policy makers must deal
with include vaccine development, production, availability, safety, effectiveness, and
access. Vaccines are biologics: their basic components begin as living material.
They introduce bacteria or dead or weakened viruses into a person or animal to
stimulate an immune reaction that the body will remember if assaulted by the same
pathogen in the future.
There is no central federal authority for vaccine policy. In the Department of
Health and Human Services (HHS), the National Vaccine Program Office (NVPO)
coordinates vaccine-related activities, and the Food and Drug Administration (FDA)
is responsible for the regulation of vaccines and other biologics. Also involved in
vaccine activities are other components of HHS (e.g., the National Institutes of
Health, the Centers for Disease Control and Prevention, and the Health Resources
and Services Administration), the Departments of Defense, Veterans Affairs, and
Homeland Security, and the U.S. Agency for International Development.
Concerned about bioterrorist attacks in the United States, the 107th Congress
passed several vaccine-related measures and the 108th Congress continued with
legislative and oversight activities regarding the development and purchase of
vaccines against possible bioterrorist attacks and dealing with the sudden shortage
of influenza vaccine at the outset of the 2004-2005 flu season.
Obstacles to vaccine availability — such as production costs, concern for
liability expenses, weak markets, and difficulties in predicting need — often have
economic roots. As mechanisms to enhance availability, Congress may consider
financial incentives, public-private partnerships, improved coordination, and
alternatives to safety and effectiveness documentation.
A pillar of U.S. policy on drugs and vaccines is the protection of the individuals
who use them. FDA does not license a product for sale in the United States until it
is satisfied that the vaccine is safe and effective. Scientists, clinicians, Members of
Congress, and the public must make decisions of vaccine safety despite uncertainties
and varying perceptions of risk. To ameliorate the difficulties, Congress could
address post-licensure adverse-event surveillance, education and risk communication,
studies in pharmacoepidemiology and pharmacoeconomics, and improving available
mechanisms to compensate individuals injured by vaccinations.
Successful development and production of safe and effective vaccines does not
ensure that everyone who needs a vaccine gets it. Congress may take up the
coordination of government childhood immunization programs and financing levels
and strategies for vaccine-related care. Noting concern for health needs of
developing countries, some Members seek to increase access to existing vaccines and
to spur development of affordable vaccines for global health threats. This report will
be updated as warranted.

Contents
Background ......................................................1
What Is A Vaccine?............................................1
Procedures for the Approval and Regulation of Biololgics..............2
Stakeholders ..................................................3
Laws Approved by the 107th Congress.............................4
Legislative Activity in the 108th Congress..........................5
Continuing Legislative Issues........................................6
Availability .......................................................6
Problems ....................................................7
Production Costs..........................................7
Production Failures........................................7
Liability .................................................7
Market ..................................................7
Planning .................................................8
Possible Legislative Solutions....................................9
Financial Incentives........................................9
Liability ................................................10
Partnerships .............................................10
Improved Coordination....................................10
Ensuring Safety and Effectiveness May Delay Availability........11
Safety and Effectiveness...........................................14
Problems ...................................................15
Side-Effects .............................................15
Insufficient Knowledge and Inadequate Risk Communication......15
Assessment of Competing Products...........................16
Production of Generic Biologics.............................16
Possible Legislative Solutions...................................16
Improving Post-Licensure Adverse-Event Surveillance...........16
Education and Risk Communication..........................17
Studies in Pharmacoepidemiology and Pharmacoeconomics.......17
Compensation ...........................................18
Access .........................................................18
Problems ...................................................19
Possible Legislative Solutions...................................19
Coordination of Government Financing Programs...............19
Payment for Vaccination and Follow-Up Care..................20
Global Health............................................20

Vaccine Policy Issues
Vaccines are almost staple topics in daily U.S. media. Television news viewers
have known about a flu vaccine shortage and that heated differences of opinion
highlight discussions of possible risks involved in both routine childhood
immunizations and vaccinations that the U.S. military requires of its personnel.
Some may even have heard that scientists recently announced some success with a
malaria vaccine.
This report’s focus is on vaccination, one of the most cost-effective methods
available to prevent infectious diseases. Whether a vaccine’s target is naturally
occurring or present because of hostile intent, the issues to address include vaccine
development, production, availability, safety, effectiveness, and access.
Background
What Is A Vaccine?
Vaccines are biologics — their basic components begin as living material —
that introduce “weakened or killed disease-causing bacteria, viruses, their
components”¹ (such as proteins, recombinant proteins, or polysaccharides) or toxoids
into a person or animal to stimulate an immune reaction that the body will remember
if exposed to the same pathogen in the future. Most vaccines are given by injection.
Although many people use the words vaccination, immunization, and
inoculation interchangeably, the terms are not technically synonymous. Vaccination
is “the physical act of administering any vaccine ...” and immunization is a “more
inclusive term denoting the process of inducing or providing immunity artificially by
administering an immunobiologic.”² Inoculation also involves introducing a
microorganism but not necessarily intentionally; the term vaccination was coined to
mean intentional inoculation with the vaccinia virus that causes cowpox to provoke
an immune response to protect against the smallpox virus.

¹Food and Drug Administration (FDA), “Vaccines Provide Effective Protection and FDA
Makes Sure They are Safe,” Just the Facts, FDA Publication No. FS 02-6, Feb. 2002, at
[http://www.fda.gov/opacom/factsheets/justthefacts/19vaccine.html], visited Nov. 22, 2004.
²Centers for Disease Control and Prevention, General Recommendations on Immunization:
Recommendations of the Advisory Committee on Immunization Practices, Morbidity and
Mortality Weekly Report (MMWR), 1994;43 (No. RR-1).

The U.S. Food and Drug Administration (FDA) licenses 46 vaccines, covering
20 diseases, now available for public use in the United States.³ Dozens more are in
active development; research teams worldwide are working to develop vaccines
against tuberculosis, malaria, HIV/AIDS, Alzheimer’s disease, some cancers, and
other diseases of which most Americans have never heard. The National
Immunization Program, part of the U.S. Centers for Disease Control and Prevention
(CDC) and its Advisory Committee on Immunization Practices (ACIP), issues
recommended immunization schedules for children, adolescents, and adults in the
United States.⁴
Procedures for the Approval and Regulation of Biololgics
FDA procedures to approve a biologic for marketing in the United States follow
the same basic format as the procedures for new drug approvals.⁵ Animal testing is
extensive, including the safety assessment of the viruses grown in animal or human
cells. After satisfactory animal safety testing, clinical trials in humans begin.
Phase I clinical trials, which include a small number of human volunteers, test
for safety. The sponsor continues with Phase II and Phase III trials to gather evidence
of the biologic’s effectiveness in larger groups of individuals, while continuing to
monitor safety data. If the product remains feasible, the sponsor, based on data
collected in the clinical trials, submits two license applications to FDA: a product
license for the vaccine and an establishment license for the manufacturing plant.
FDA scientists review the clinical data, along with the proposed labeling and
manufacturing protocols; inspect the manufacturing facility to assess whether it can
produce a consistent product; and performs test on the vaccine and its components.
Advisory committees, made up of experts from outside of FDA, also review the data,
are available for consultation, and make recommendations to FDA regarding
approval.
Because contamination is a greater threat with vaccines than with drugs because
they are made from living organizations, FDA maintains an active presence even
after a vaccine is approved. It requires extensive testing of vaccines and all

³A smallpox vaccine is available only through DOD or CDC programs. Vaccines Licensed
for Immunization and Distributed in the U.S., information last updated Sept. 21, 2004, at
[http://www.fda.gov/cber/vaccine/licvacc.htm], visited Oct. 16, 2004.
⁴For 2004, ACIP recommendations, which vary by age and medical conditions of potential
recipients, include vaccines for diphtheria, Haemophilus influenzae type b, hepatitis A,
hepatitis B, influenza, measles, meningococcal disease, mumps, pertussis, pneumococcal
disease, polio, rubella, tetanus, and varicella (chicken pox) ([http://www.cdc.gov/nip],
visited Oct. 25, 2004).
⁵The FDA website provides links related to the FDA approval process for biologics; for
example: Isadora B. Stehlin, How FDA Works to Ensure Vaccine Safety, FDA Consumer,
Dec. 1995, at [http://www.fda.gov/fdac/features/095_vacc.html]; and Michelle Meadows,
Understanding Vaccine Safety: Immunization Remains Our Best Defense Against Deadly
Disease, FDA Consumer, July-Aug. 2001, at
[ ht t p: / / www.f da.gov/ f dac/ f eat ur es/ 2001/ 401_vacc.ht ml ] .

ingredients (e.g., diluents, preservatives, or adjuvants) for characteristics including
identity, purity, and potency. FDA continues to assess the production process, too,
including samples of each lot and data regarding purity and potency.
Finally, because even large clinical trials can identify only common potential
adverse effects, FDA maintains postmarketing surveillance programs and sometimes
works with a manufacturer in Phase IV studies of long term safety and effectiveness.
Stakeholders
Many groups have a stake in vaccine-related issues, including the government
entities responsible for research and development, licensing, post-licensing
surveillance of adverse reactions, provision of health care, protection of the
population, interstate and international trade, intellectual property protections, and
homeland security.
There is no central authority for vaccine policy within the federal government.
In the Department of Health and Human Services (HHS), the National Vaccine
Program Office (NVPO) coordinates vaccine-related activities and the FDA is
responsible for the regulation of human vaccines and other biologics.⁶ The National
Institutes of Health (NIH) conducts intramural vaccine research and development and
funds research in universities, for example. CDC, charged with protecting the health
and safety of the population, houses the National Immunization Program (NIP) and
its ACIP, which work to coordinate nationwide activities, including the Vaccines for
Children (VFC) program and the state immunization grants program.⁷ CDC also

⁶In the United States, FDA — mostly within its Center for Biologics Evaluation and
Research (CBER) — bears the responsibility for vaccine regulation, primarily under the
authorities granted the Secretary of HHS in the Federal Food, Drug and Cosmetic Act and
the Public Health Service Act. To receive a license from FDA to market a vaccine, the
sponsor (often the manufacturer) must demonstrate to the satisfaction of FDA that the
product is safe and effective for human use. Data to support those claims come, primarily,
from clinical trials. Once a product is approved, the sponsor must comply with detailed
Good Manufacturing Practices (GMPs) and regulations concerning the surveillance of
adverse reactions among individuals receiving the vaccine. FDA policies regarding vaccine
approval are similar to FDA policies for prescription drugs. See CRS reports focusing on
drugs: CRS Report RL30989, The U.S. Drug Approval Process: A Primer, by Blanchard
Randall IV; CRS Report RL30913, Pharmaceutical Research and Development: A
Description and Analysis of the Process, by Richard E. Rowberg; and CRS Report
RS20033, Food and Drug Administration: Selected Funding and Policy Issues, by Donna
U. Vogt.
⁷Following a congressional directive in P.L. 99-660, in 1986 HHS established a National
Vaccine Program within the Public Health Service’s Office of the Assistant Secretary for
Health to coordinate vaccine research, development, safety and efficacy testing, and
production and procurement across federal agencies. Transferred organizationally in 1994
to CDC and then back to HHS, the National Vaccine Program Office manages the Inter-
Agency Vaccine Group and the National Vaccine Advisory Committee, and works toward
achieving the National Vaccine Plan [published in 1994], which involves “pursuing the
prevention of infectious diseases through immunizations” ([http://www.hhs.gov/nvpo],
(continued...)

maintains the Strategic National Stockpile (SNS), which includes some vaccines
against bioterror agents. The National Vaccine Injury Compensation Program
(VICP), which is jointly administered by the Health Resources and Services
Administration (HRSA), where it is located, and the U.S. Court of Federal Claims
and the U.S. Department of Justice, “provides compensation for injuries judged to
have been caused by certain vaccines.”⁸ Also administered from HRSA is the
Smallpox Vaccine Injury Compensation Program, set up in 2003.⁹
Vaccine responsibilities lie outside of HHS as well. The Department of Defense
(DOD) maintains research and development programs for vaccines against both
naturally occurring infectious diseases and bioweapons. DOD administers routine
and deployment-related vaccines to military personnel and some civilian employees
and contractors. As a primary health care provider, DOD also administers vaccines
as necessary to its retirees and current personnel and their families. The Department
of Veterans Affairs administers vaccines to U.S. veterans who seek care in its
facilities. The U.S. Agency for International Development (USAID) supports routine
immunization programs in developing countries and works to reduce the impact of
vaccine-preventable disease worldwide. State and local governments conduct
vaccine activities within their public health role, such as conducting vaccine clinics,
maintaining immunization registries, and establishing immunization requirements for
school attendance. Veterinary biologics are regulated by the U.S. Department of
Agriculture, Animal and Plant Health Inspection Service, under authority of the
Virus, Serum and Toxin Act. These products must meet similar standards of safety,
efficacy, purity and potency as do human products.
Interested parties outside government include individuals and private entities,
both for-profit and not-for-profit, such as current and potential vaccine recipients and
their families, employers offering health care benefits, insurers, traditional vaccine
manufacturers, biotechnology firms, trade associations such as the Pharmaceutical
Research and Manufacturers of America, academic biomedical researchers,
economists, trial lawyers, health care professionals and institutions, and patient and
disease-specific advocacy groups.
Laws Approved by the 107th Congress
Concerned about bioterrorist attacks in the United States, the 107th Congress
approved several bills that included vaccine-related issues:
The USA PATRIOT Act (P.L. 107-56) includes a sense-of-Congress statement
expressing the need to provide funding for bioterrorism preparedness and response
(Section 1013); the National Defense Authorization Act for Fiscal Year 2002 (P.L.

⁷ (...continued)
visited Nov. 10, 2004).
⁸The National Vaccine Injury Compensation Program home page is at
[http://www.hrsa.gov/osp/vicp], visited Nov. 4, 2004.
⁹The Smallpox Vaccine Injury Compensation Program home page is at
[http://www.hrsa.gov/smallpoxinjury], visited Nov. 4, 2004.

107-107) directs the Secretary of Defense to accelerate research, development, and
production of items such as vaccines for defense against biological agents used as
weapons, and includes authorization to build a government-owned contractor-
operated vaccine production facility (Section 1044). The Public Health Security and
Bioterrorism Preparedness and Response Act of 2002 (P.L. 107-188) directs the HHS
Secretary to undertake specific activities with regard to national stockpiles of drugs
and vaccines and the accelerated approval of high-priority countermeasures (Sections

121-126).

The Homeland Security Act (HSA) of 2002 (P.L. 107-296) protects
manufacturers and health care workers who administer the smallpox vaccine from
tort liability and restricts that liability assumed by the United States to negligence of
those parties (Section 304). Other sections of the HSA of 2002 that related to
vaccines have since been repealed.^10,11
Legislative Activity in the 108th Congress
The 108th Congress passed some vaccine-related legislation. The Smallpox
Emergency Personnel Protection Act of 2003 (P.L. 108-20) created a mechanism to
compensate individuals who, in response to a Secretarial request for smallpox
vaccine preparedness, are injured by the vaccinia virus used in smallpox vaccines.
Vaccinees and their contacts are eligible for medical care expense reimbursement,
lost income benefit, and death benefits, administered through the HHS Health
Resources and Services Administration.¹² Other enacted laws that include references
to vaccine coverage or funding are the Consolidated Appropriations Resolution, 2003
(108-7); the United States Leadership Against HIV/AIDS, Tuberculosis, and Malaria
Act of 2003 (P.L. 108-25); the National Defense Authorization Act for Fiscal Year

2004 (P.L. 108-136); the Medicare Prescription Drug, Improvement, and

¹⁰These CRS reports, most produced to support discussion of homeland security, address
vaccine-related issues: CRS Report RL31960, Smallpox Vaccine Injury Compensation, by
Susan Thaul; CRS Report RL31864, High-Threat Biological Agents: Characteristics,
Effects, and Policy Implications, by Dana A. Shea; CRS Report RS21414, Mandatory
Vaccinations: Precedent and Current Laws, by Angie A. Welborn; CRS Report RL31694,
Smallpox Vaccine Stockpile and Vaccination Policy, by Judith Johnson; CRS Report
RL31649, Homeland Security Act of 2002: Tort Liability Provisions, by Henry Cohen; CRS
Report RL31354, Possible Impacts of Major Counter Terrorism Security Actions on
Research, Development, and Higher Education, by Genevieve J. Knezo; and CRS Report
RL31263, Public Health Security and Bioterrorism Preparedness and Response Act (P.L.

107-188): Provisions and Changes to Preexisting Law, by C. Stephen Redhead, Donna U.

Vogt, and Mary E. Tiemann.
¹¹H.J.Res. 2 (P.L. 108-7, Division L, Section 102) repealed Sections 1714-1716 of the HSA
of 2002, which would have covered adverse effects attributed to thimerosal, a mercury-
containing vaccine preservative, within the National Vaccine Injury Compensation Program.
The Project BioShield Act of 2004 (P.L. 108-276, Section 3) returned the Strategic National
Stockpile (of drugs and vaccines) to the Dept. of Health and Human Services, undoing
Sections 502 and 503 of the HSA of 2002, which had transferred management responsibility
to the Dept. of Homeland Security.
¹²For more detail, see CRS Report RL31960, by Thaul.

Modernization Act (P.L. 108-173); and the Consolidated Appropriations Resolution,

2004 (P.L. 108-199).

Dozens of bills relating to vaccine research, purchase, and coverage were
introduced in the 108th Congress. The Improved Vaccine Affordability and
Availability Act (S. 754), introduced by Senate Majority Leader Frist, received the
most attention, but scheduled markups of the bill were postponed several times in
spring 2003. The bill would “amend the Public Health Service Act to improve
immunization rates by increasing the distribution of vaccines and improving and
clarifying the vaccine injury compensation program....” In fall 2004, in response to
the sudden shortage of influenza vaccine, additional bills were introduced and several
hearings were held.¹³
Continuing Legislative Issues
In its final three sections, this report organizes the range of legislative issues
pertaining to vaccines that Members of Congress may consider into three groups:
^! availability,
^!safety and effectiveness, and
^! access.
Cost — of research, development, production, regulation and oversight, for example
— underlies each of these concerns.
Availability
Thirty-seven American companies made vaccines in 1967; in October 2004,
there were nine.¹⁴ Why? Reasons given are mostly economic. The road to a shot in
the arm can take decades of research and development and, according to industry
estimates, about $800 million per licensed vaccine,¹⁵ requiring great financial
reserves to sustain a company through the research and development and clinical trial
process. Once a vaccine is licensed, its continued production remains complex, as
the 2004-2005 flu vaccine shortage in the United States illustrates.

¹³CRS Report RL32655, Influenza Vaccine Shortage and Implications, by Sarah A. Lister
and Erin D. Williams, includes a comprehensive listing of legislation.
¹⁴Rino Rappuoli, Henry I. Miller, Stanley Falkow, The Intangible Value of Vaccination,
Science, v. 297, Aug. 9, 2002, p. 937; and FDA, Vaccines Licensed for Immunization and
Distributed in the US, information updated Sept. 2004, at
[http://www.fda.go v/ cber/vaccine/licvacc.htm] .
¹⁵Pharmaceutical Research and Manufacturers of America (PhRMA), PhRMA Industry
Profile 2002. Researchers at the Tufts Center for the Study of Drug Development added in
the cost of post-approval studies and revised the estimate to $897 million (News Release,
May 13, 2003, at [http://csdd.tufts.edu/NewsEvents/RecentNews.asp?newsid=29], visited
Nov. 23, 2004).

Problems
Production Costs. Because vaccines are biologics, even routine manufacture
involves care, expertise, and expense much beyond that required for pharmaceuticals.
To produce a drug, the manufacturer essentially repeats a chemical formula.
Vaccines require dedicated production facilities that include physical and chemical
barriers to protect workers from pathogen exposure and finely regulated temperature
and ventilation to keep the biologics viable while stored. Also, because the product
is injected, the purity standard has to be much higher than for a pill. Although FDA
inspects both drug- and vaccine-production facilities, it inspects every lot of vaccine
produced and only a sample of drug production lots.¹⁶
For example, manufacturers ceased production of licensed vaccines against
plague because of manufacturing difficulties, and against adenovirus infection
because of contract cost issues. To restart production, these or other manufacturers
must submit new license applications to FDA, conform to current good
manufacturing practice standards, and demonstrate the safety and effectiveness of the
new vaccines — all before they can make the vaccine available to the public.
Production Failures. In October 2004, when the U.S. manufacturer Chiron
announced that the British drug regulatory authorities had shut its Liverpool plant,
the United States was instantly plunged into a flu vaccine shortage. Chiron was
slated to supply approximately half of the vaccine for U.S. use in the 2004-2005 flu
season. The British agency — and then with FDA concurrence — found some
bacterial contamination and manufacturing practices that could not assure the safety¹⁷
of the rest of the vaccine production.
Liability. Why go to the trouble for a product that does not promise the sales
volume common to pharmaceuticals — particularly when manufacturers may be
liable if vaccines cause injury? The huge claims for compensation that followed the
swine flu immunization program in the mid-1970s have made the vaccine industry
wary. Manufacturer executives recall, for example, discussions of unindemnified
liability in their decisions to decline plans to produce anthrax or Lyme disease
vaccines.
Market. For some diseases, scientists know how to produce protective
vaccines but manufacturers have chosen not to pursue the time-consuming and
expensive steps necessary to obtain FDA approval. For some, there is insufficient
market size in the United States to warrant the effort. An example is the tick-borne
encephalitis vaccine that DOD administered to troops in Bosnia during the 1990s.
Although licensed in Europe where the disease-carrying ticks are more widespread,
the vaccine is not FDA-licensed. To get FDA approval requires U.S. clinical trials

¹⁶FDA, Vaccine Frequently Asked Questions (FAQs), Center for Biologics Evaluation and
Research, at [http://www.fda.gov/cber/faq/vacfaq.htm], visited Nov. 10, 2004.
¹⁷See CRS Report RL32655, Influenza Vaccine Shortage and Implications, by Sarah A.
Lister, for an extended discussion of the issues.

for safety. Neither the interested manufacturer nor DOD has been willing to bear the
expense of those trials.¹⁸
Malaria, tuberculosis, and HIV/AIDS kill 7 million and sicken 400 million
worldwide each year.¹⁹ Yet, aside from HIV, manufacturers have little incentive to
develop vaccines because U.S. incidence is low, involving mostly travelers or
immigrants and their contacts, and preventive and treatment medications are readily
available. In countries where vaccines could make a big difference, few resources
are available to support development or purchase. Interestingly, private benefactors
have been stepping in to support public-good-focused research. The Bill and
Melinda Gates Foundation, for example, gave $150 million to the Malaria Vaccine
Initiative, which announced in October 2004 initial findings that a malaria vaccine
it is developing with industry was somewhat effective in young children.²⁰
Planning. Finally, difficulties with planning can render even licensed vaccines
temporarily unavailable. In 2002, such problems created shortages of licensed^21,22,23
vaccines for eight of the 11 vaccine-preventable childhood diseases.
Aside from production set-backs, influenza vaccine poses a perennial challenge
involving choice of vaccine components and amount to manufacture. Each year,
public health authorities and manufacturers analyze worldwide surveillance
information to determine which flu virus strains are likely to put humans at risk in
the coming year. Sometimes, the viral strain that most threatens the U.S. public is

¹⁸U.S. Army Medical Materiel Development Activity Information Paper, Tick-Borne
Encephalitis Virus Vaccine, dated Sept. 30, 2004, at
[http://www.usammda.army.mil/info335.pdf], visited Oct. 25, 2004.
¹⁹Joint United Nations Programme on HIV/AIDS, Global summary of the AIDS epidemic,
Dec. 2004, at [http://www.unaids.org/wad2004/EPI_1204_pdf_en/Chapter0-1_intro_en.pdf];
World Health Organization (WHO), Tuberculosis, fact sheet no. 104, revised March 2004,
at [http://www.who.int/mediacentre/factsheets/fs104/en/print.html]; and WHO, What is
Malaria, Roll Back Malaria information sheet, at
[http://www.rbm.who.int/cmc_upload/0/000/015/372/RBMInfosheet_1.htm], all visited
Nov. 23, 2004.
²⁰Pedro L. Alonso, Jahit Sacarlal, John J. Aponte, Amanda Leach, et al., Efficacy of the
RTS,S/AS02A vaccine against Plasmodium falciparum infection and disease in young
African children: randomised controlled trial, The Lancet, v. 364, issue 9443, pp. 1411-

1420, Oct. 16, 2004.

²¹Robin J. Strongin, U.S. Childhood Vaccine Availability: Legal, Regulatory, and Economic
Complexities, National Health Policy Forum Issue Brief, no. 785, Nov. 15, 2002, at
[http://www.nhpf.org/ pubs/pubs.htm#2002].
²²Questions and Answers on 2002 Vaccine Shortages, National Immunization Program, at
[http://www.cdc.gov/nip/news/shortages/faqs_shortages_3-7-02.htm#s1], and
Current Vaccine Delays and Shortages, NIP, at
[http://www.cdc.gov/nip/news/shortages/default.htm#Which], both visited Nov. 23, 2004.
²³As of Oct. 2004, the NIP reports a shortage of only influenza vaccine among all CDC-
recommended vaccines for children and adolescents. Current Vaccine Delays and
Shortages, NIP, at [http://www.cdc.gov/nip/news/shortages/default.htm#Which], visited Oct.

25, 2004.

not among those chosen for that year’s vaccine, the situation in 2003 when vaccine
developers could not formulate a Fujian component in time for the season’s
distribution.²⁴
Because influenza changes slightly each year, healthy adults have partial
immunity to new strains; each year, the virus typically makes healthy people sick, but
not too sick. Several times a century, however, the virus changes enough that there
is no partial immunity. This situation can lead to rapid worldwide spread of the
virus, called an influenza pandemic, with severe illness and death, even in healthy
people, and possible serious disruptions in services and social order. Some have
expressed concern that the problems evident in the national response to the 2004-
2005 flu vaccine shortage serve as a relevant drill — and warning — for pandemic
preparedness.²⁵
Also a topic of international surveillance and planning is avian influenza (“bird
flu”) — an influenza strain affecting poultry in Asia — in anticipation of its possible
joining with a viral strain that infects humans. Rather than a unique circumstance,
this international investigation is part of the intensive, ongoing process of animal and
human (clinical and laboratory) surveillance, testing, and action that is public health
practice. For example, CDC laboratories collaborate with the World Health
Organization in testing and describing avian viruses to help in developing a potential
vacci ne.²⁶
Possible Legislative Solutions
Congress may consider at least four kinds of measures to enhance vaccine
availability: financial incentives, public-private partnerships, improved coordination,
and alternatives to safety and effectiveness documentation.
Financial Incentives. Most people in the United States now view protections
against bioterrorism or biowarfare agents, such as vaccines, as in the public interest.
Many also so categorize all vaccines, whether intended against naturally occurring
or bioterror-related infectious diseases. The bottom line in persuading companies to
produce vaccines is largely one of opportunity cost: whether they can afford to put
aside other potentially lucrative projects to do so.
Over the years, Members of Congress have proposed changes to the Internal
Revenue Code involving tax credits for certain vaccine research and distribution
activities to effectively lower the cost to manufacturers. Another way to provide a
financial incentive is to assure that a primary purchaser is available to generate
demand for the vaccine. When the government is the primary purchaser, such as for

²⁴See CRS Report RL32655, by Lister; and Questions and Answers: The 2003-2004 Flu
Season, CDC, at [http://www.cdc.gov/flu/about/qa/0304season.htm], visited Nov. 22, 2004.
²⁵See CRS Report RL32655, by Lister.
²⁶CDC, Avian Influenza, at [http://www.cdc.gov/flu/avian/] and World Health Organization,
Avian influenza, at [http://www.who.int/csr/disease/avian_influenza/en/], both visited Nov.

22, 2004.

anthrax or smallpox vaccines, it can develop contracts with manufacturers that
address volume, liability protection, and long-term plans, for example, that make
production practical.
In his 2003 State of the Union speech, President George W. Bush proposed a
federal initiative, Project BioShield, to encourage private industry to develop medical
countermeasures to bioterrorism threats. The Project BioShield Act of 2004 was
signed into law in July 2004 (P.L. 108-276).²⁷
Liability. Because some manufacturers have named liability concerns among
reasons to forgo vaccine production, legislative proposals have addressed
indemnification, liability insurance, and injury compensation plans. Protection of
potential manufacturers, seen as a necessary incentive to participation, was included
in the Homeland Security Act of 2002 and the Smallpox Emergency Personnel²⁸
Protection Act of 2003. The American Jobs Creation Act of 2004 added the
influenza vaccine to those that the National Vaccine Injury Compensation Program
covers.
Partnerships. Some Members of Congress have expressed interest in
industry consortia or public-private partnerships to accelerate vaccine research and
development and manufacture. This may involve considering issues of intellectual
property protection among collaborators and anti-trust law accommodations to allow
private manufacturers to make joint decisions. Such partnerships have been
recommended as ways to spread financial risk, thus making vaccines available for
diseases that are prevalent among small or impoverished groups.
Improved Coordination. Better coordination among federal regulators,
private manufacturers, government scientists, and purchasers could avoid many
supply shortages, such as occurred with childhood vaccines in 2002. Coordination
could also shorten the time between initial research and product licensure. Policy
analysts look for mechanisms to streamline FDA administrative — but not human
safety and effectiveness assurance — procedures. Some, including the Institute of
Medicine (IOM), have suggested establishing a National Vaccine Authority.²⁹ Two
other IOM committees, in DOD-sponsored reports addressing naturally occurring
infectious diseases³⁰ and biowarfare agents,³¹ recommended better coordination

²⁷For a fuller discussion of the legislative activity, see CRS Report RS21507, Project
BioShield, by Frank Gottron, and CRS Report RL32549, Project BioShield: Legislative
History and Side-by-Side Comparison of H.R. 2122, S. 15, and S. 1504, by Frank Gottron
and Eric Fischer.
²⁸P.L.108-357, signed by the President on Oct. 22, 2004.
²⁹Council of the Institute of Medicine, Statement on Vaccine Development, Nov. 5, 2001,
at [http://www.iom.edu/report.asp?id=5487], visited Nov. 23, 2004.
³⁰IOM, Protecting Our Forces: Improving Vaccine Acquisition and Availability in the U.S.
Military, Stanley M. Lemon, Susan Thaul, Salem Fisseha, Heather C. O’Maonaigh, editors,
Washington, D.C., National Academies Press, 2002.
³¹IOM, Giving Full Measure to Countermeasures: Addressing the DOD Program to Develop
(continued...)

within the DOD vaccine acquisition programs and between DOD and other entities,
particularly HHS. These groups anticipate that a coordinated decision and budget
authority could present a coherent front to private manufacturers and within
government. After the 2002 reports, DOD assigned vaccine acquisition
responsibilities to a higher administrative level³² and interagency agreements and
working groups have been formed. Cutting down on frustrations arising from getting
different answers or timetables from different offices could make vaccine work more
appealing to those in research and development and manufacture.
While almost any change in research and development, production, monitoring,
and sales would involve FDA, some questions focus on FDA itself. These include
the extent to which its budget limits the scope and timeliness of its activities. Some
analysts suggest that FDA standards are too high to be reasonably feasible.
Sometimes all the necessary pieces of potential solutions exist, but without an
organizing force. For years, various representatives of HHS, DOD, and private
manufacturers had met over contracts to develop and produce vaccines to protect, for
example, against smallpox and anthrax. The September and October 2001 attacks
on the U.S. population quickly dissolved the sticking points. Political will — and its
ability to get all the right people and their checkbooks in the room at the same time
— fueled action to acquire smallpox vaccine, both doses of previously made and
licensed product and contracts to develop new products. In the years since then, HHS
has announced contracts to develop new vaccines against smallpox, plague, and
tularemia;³³ and, with Project BioShield funding, a second-generation anthrax
vaccine will be stored in the Strategic National Stockpile.³⁴
Ensuring Safety and Effectiveness May Delay Availability. It takes a
long time from the start of clinical trials to FDA licensure.³⁵ Many observers

³¹(...continued)
Medical Countermeasures Against Biological Warfare Agents, Lois M. Joellenbeck, Jane
S. Durch, and Leslie Z. Benet, editors, Washington, D.C., National Academies Press, 2004.
³²First Joint Program Executive Office for Chemical and Biological Defense Formed, U.S.
DOD News Release [http://www.defenselink.mil/news/Apr2003/b04252003_bt277-03.html]
dated April 25, 2003, visited Nov. 23, 2004; and DOD briefing (by COL David Danley and
others) to CRS staff, Apr. 30, 2003.
³³HHS, HHS Awards $232 Million in Biodefense Contracts for Vaccine Development, News
Release, Oct. 7, 2004, at [http://www.hhs.gov/news/press/2004pres/20041007a.html].
³⁴HHS, HHS Continues to Strengthen Umbrella of Protection from Bioterrorism, News
Release, Sept. 10, 2004, at [http://www.hhs.gov/news/press/2004pres/20040910.html].
³⁵PhRMA cites research estimating 10 to 15 years from idea to FDA approval (J.A. DiMasi
et al., The Price of Innovation: New Estimates of Drug Development Costs, Journal of
Health Economics, v. 22, pp. 151–185, 2003, cited in Pharmaceutical Industry Profile 2004,
Washington, D.C., PhRMA, 2004, at
[http://www.phrma.org/publications/publications/2004-03-31.937.pdf]). FDA performance
standards call for review of a new drug or biologics application within 10 months of
submission (6 months for priority review), goals it met over 95% of the time in 2002 (FDA,
FY 2003 Performance Report to Congress, at
(continued...)

consider much of that time as necessary to ensure that vaccines produced and sold
are safe and effective. FDA has formal mechanisms to expedite the review process:
fast-track drug development, priority review, and accelerated approval. Its “Animal
Rule” and provisions under the Project BioShield Act of 2004 also support shorter
time from idea to approved public use.³⁶ The Congress could assess the extent to
which the laws that direct HHS and DOD to facilitate or accelerate research and
development and approval of bioterrorism countermeasures as well as needed
vaccines for serious conditions³⁷ are succeeding and consider legislative ways to help
those processes along.
Fast-Track Mechanism. The Food and Drug Administration Modernization
Act of 1997 (FDAMA, P.L. 105-115) described a “Fast Track” mechanism whereby
the manufacturer and FDA discuss development plans and strategies before the
formal submission of a Biologics License Application (BLA, for a vaccine
application, for example) or a New Drug Application (NDA, for a drug). The early
interaction can help clarify goals and work through obstacles that would delay
approval decisions if they became evident only at BLA submission.³⁸
Accelerated Approval. For the treatment of a serious or life-threatening
illness, FDA regulations allow “accelerated approval” of a biologic product that
provides a “meaningful therapeutic benefit ... over existing treatments.” The
approval is based on clinical trials that, rather than using standard outcome measures
such as survival or illness, use “a surrogate endpoint that is reasonably likely ... to
predict clinical benefit.” FDA usually requires postmarketing studies of biologics
approved this way.³⁹
Animal Rule. Scientists use clinical trials and field trials to test a vaccine’s
effectiveness. Clinical trials designed to test vaccines for most infectious diseases
usually involve random assignment of individuals to groups — members of one
group are given the new vaccine and members of the other (the control group) are

³⁵(...continued)
[ h t t p : / / www.f d a.go v/ oc/ pduf a/ r e por t 2003/ or gnpas2003.ht ml ] ) .
³⁶Cynthia L. Kelley, Senior Advisor for Counterterrorism Medical Countermeasures, FDA
Center for Biologics Evaluation and Research, “Fast-Tracking Biodefense Vaccines and
Therapeutics: An Urgent Challenge We Must Meet,” presentation prepared for the Third
Annual Research, Technologies and Applications in Biodefense, Washington, D.C., Aug.

18, 2004, at [http://www.fda.gov/cber/summaries/biodef081804ck.pdf]; and William E.

Egan, Acting Director, FDA Office of Vaccines Research and Review, “Routes to Expedite
Vaccine Approvals,” presentation prepared for the World Vaccine Congress, Lyon, France,
Oct. 2004, at [http://www.fda.gov/cber/summaries/worldvacc101104we.pdf].
³⁷The FDA Modernization Act of 1997 (FDAMA, P.L. 105-115); the National Defense
Authorization Act for Fiscal Year 2002 (P.L. 107-107); and the Public Health Security and
Bioterrorism Preparedness and Response Act of 2002 (P.L. 107-188).
³⁸FDA, Guidance for Industry, Fast Track Drug Development Programs — Designation,
Development, and Application Review, at [http://www.fda.gov/cber/gdlns/fsttrk.pdf], visited
Nov. 9, 2004.
³⁹21 CFR 601 subpart E, Accelerated Approval of Biological Products for Serious or Life-
Threatening Illnesses.

given a placebo or an existing licensed vaccine — to assess whether the
investigational vaccine would effectively prevent disease. When the disease under
study has no known treatment and has severe outcomes (including death), it is
unethical to challenge human research subjects, because if the vaccine is not
effective, the subjects are irreparably harmed. Field trials, in which scientists observe
naturally occurring disease among vaccinated and unvaccinated groups, are not
feasible for diseases that occur sporadically or would occur only in the context of
bioterror or biowarfare action. For these reasons, researchers and regulators may
sometimes need alternative models for demonstrating vaccine effectiveness.
In 2002, FDA published its long-debated final Animal Efficacy Rule. The
regulations apply only “when adequate and well-controlled clinical studies in humans
cannot be ethically conducted and field efficacy studies are not feasible.” They allow
submission of data from animal studies of effectiveness as evidence to support
licensure applications of new drug and biological products that target “serious or life-
threatening conditions” in humans.⁴⁰ It could apply, for example, to inhalational
anthrax, for which there is no surrogate of immunity and no natural endemic disease.
Although no vaccine has yet been approved under this rule, FDA used the rule for the
first time in February 2003 to approve a drug.⁴¹ Congress may follow the
implementation of this rule and discuss alternative actions in its oversight of safety,
effectiveness, and availability goals.
Priority Review. Unlike Fast Track, Accelerated Approval, or Animal Rule
activities, the Priority Review process begins only when a manufacturer officially
submits a BLA (or an NDA). Priority Review, therefore, does not alter the steps
taken in a drug’s development or testing for safety and effectiveness. It does,
however, for products believed to address unmet needs, shorten the anticipated
amount of time until approval decision from 10 months to 6 months.⁴²
Medical Countermeasures to Bioterrorism Attack. Although FDA, NIH,
and DOD had been working on vaccines against potential bioterrorism or biowarfare
agents, the events of September and October 2001 infused the efforts with resources
and political capital. Product development was supported with increased interaction
among and within government agencies and with manufacturers, and consideration
of fast track, accelerated approval, and animal efficacy mechanisms to bring products
to review. The Project BioShield Act of 2004 allows the HHS Secretary (with
directions regarding potential risk and benefit and required recordkeeping) to
authorize the emergency-use of products that do not have FDA approval in the event
of potential serious or life-threatening effects of a biological agent for which there

⁴⁰21 CFR, Parts 314 and 601; FDA, New Drug and Biological Drug Products: Evidence
Needed to Demonstrate Effectiveness of New Drugs When Human Efficacy Studies Are Not
Ethical or Feasible, Final rule, Federal Register, vol. 67, no. 105, pp. 37988-37998, May 31,

2002, at [http://www.gpoaccess.gov/fr/advanced.html], visited Nov. 23, 2004.

⁴¹FDA Approves Pyridostigmine Bromide as a Pretreatment Against Nerve Gas. FDA News,
Feb. 5, 2003, at [http://www.fda.gov/bbs/topics/NEWS/2003/NEW00870.html], visited Nov.

23, 2004.

⁴²FDA, Oncology Tools: Fast Track, Priority Review and Accelerated Approval, CDER, at
[http://www.accessdata.fda.gov/scripts/cder/onctools/Accel.cfm], visited Nov. 8, 2004.

is no approved product. Other provisions of the Act address spending authority for
medical countermeasure (such as vaccines) development and purchase.
Safety and Effectiveness
A pillar of U.S. policy on drugs and vaccines is the protection of the individuals
who use them. Vaccines cannot be marketed within the United States without a
license from FDA; and FDA does not license a product until it is satisfied that the
vaccine is safe and effective and that the manufacturing process can produce it.
Congress may be called upon again to discuss issues of both safety and effectiveness.
This could mean assessing how safe is safe. It will also mean defining effectiveness:
absolute or simply better than nothing or better than the available alternative? It can
also mean assessing effectiveness in terms of value-for-cost.
Safety is assessed by the nature and frequency of adverse reactions attributable
to vaccine use. A vaccine need not be side-effect free for FDA licensure; the licensed
smallpox vaccine carries an estimate, based on data from its routine use 30 years ago,
of one or two deaths per million recipients. Similarly, effectiveness does not mean
that a vaccine must protect permanently or completely. Researchers attempt to assess
efficacy, generally, with expensive and lengthy clinical trials that compare infection
or illness rates in two groups, both exposed to the disease-causing agent, but with
only one provided with the hypothesized vaccine protection. Effectiveness describes
how the product works in a real-world situation. For drugs, effectiveness is often
lower than efficacy because of interactions with other medications or health
conditions of the patient, sufficient dose or duration of use not prescribed by the
physician or followed by the patient, or use for a off-label condition that had not been
tested. Because vaccines are administered by the clinician, some of these conditions
are not relevant. Effectiveness may, however, still be diminished by the health of the
vaccinee and whether circumstances permit all shots in a required series to be given
according to schedule.
FDA and CDC monitor safety, in part, with their Vaccine Adverse Event Report
System (VAERS), which assembles reports from parents, clinicians, and
manufacturers of problems that may be related to vaccination. Another FDA
program, MedWatch, informs the public with clinical information about safety issues
involving vaccines and other medical products. These so-called passive surveillance
systems rely on consumers and physicians to both recognize adverse events as
possibly vaccine-related and to follow through with reporting their concern to FDA.
Such reports are valuable aids to researchers looking for potential risks. The picture
painted by the data, however, is incomplete.⁴³

⁴³The FDA webpage that gives an overview of VAERS describes strengths and limitations
of the reporting system ([http://www.fda.gov/cber/vaers/what.htm]).

Problems
Side-Effects. Some scientists, parents, and consumer advocates raise
concerns that U.S. vaccine policy, with its recommended 20 shots to infants by age
two,⁴⁴ endangers the children it aims to protect. They cite hypotheses that the
vaccines or preservatives or packaging might cause autism and other
neurodevelopmental disorders. One focus has been on thimerosal, a mercury-
containing preservative used in some vaccines. In this case, even though the science
is not definitive,⁴⁵ manufacturers have chosen to reformulate many vaccines so
thimerosal is not used.
An example of the government’s interagency system to protect vaccine
recipients involves the rotavirus vaccine that the CDC Advisory Committee on
Immunization Practices had added to the list of recommended infant vaccinations in
1998. During the year of mass use, VAERS flagged reports of bowel obstruction
soon after rotavirus vaccination; CDC recommended suspending those vaccinations
until it could study the apparent association. In October 1999, after scientific review
of the data, the ACIP withdrew its earlier recommendation that the rotavirus vaccine
be given to infants.⁴⁶
Insufficient Knowledge and Inadequate Risk Communication. Often,
decisions of vaccine safety revolve around perceptions of risk, methodologic limits
of risk assessment, and communication of what is known about risks. Scientists,
clinicians, Members of Congress, and public policy analysts continue to face choices
on risk — hypothetical and real — that do not offer clear alternatives. These involve
uncertainties, both scientific and political, and, therefore, will reflect personal and
communal values. Smallpox vaccination policy, for example, must weigh risks and
benefits whose balance may differ when considered from the perspective of the
nation or the perspective of the individual.
Knowledge, therefore, is not an issue only for public policy. Some parents
refuse pertussis (whooping cough) and measles vaccines for their children out of
concern about vaccine safety. In some of these cases, and in polio vaccine refusals

⁴⁴CDC, Parents Guide to Childhood Immunization, NIP, at
[http://www.cdc.gov/nip/publications/Parents-Guide/pg-app1-sched.pdf], visited Oct. 25,

2004.

⁴⁵The Immunization Safety Review Committee of the Institute of Medicine reported in 2001
that it “was unable to conclude, however, from the existing evidence whether thimerosal
does or does not cause neurodevelopmental disorders” (IOM, Immunization Safety Review:
Thimerosal-Containing Vaccines and Neurodevelopmental Disorders, Kathleen Stratton,
Alicia Gable, Marie C. McCormick, editors, Washington, D.C., National Academies Press,
2001). Three years later, having reviewed additional scientific studies, the IOM authoring
committee concluded that the evidence “favors rejection” of the idea that either the measles-
mumps-rubella vaccine or thimerosal-containing vaccines cause autism (IOM, Immunization
Safety Review: Vaccines and Autism, Washington, D.C., National Academies Press, 2004).
⁴⁶ACIP’s rotavirus vaccine fact sheet is at
[http://www.cdc.gov/nip/publications/fs/Rotavirus.htm], visited Nov. 15, 2003.

(reportedly based on misinformation about side effects) in developing countries
seeking to eliminate disease,⁴⁷ avoidable and potentially horrible diseases still occur.
How can anyone decide whether getting immunized is worth the risk?
Implementation of government decisions concerning anthrax vaccination was
hindered by concern about similar questions of uncertainty. Some members of the
U.S. armed forces balked at mandatory anthrax vaccination, raising questions of
safety. In October 2004, DOD stopped its anthrax vaccination program following a
U.S. District Court for the District of Columbia injunction. The Court based its
action on its finding that FDA had not solicited public comment on its finding that
the vaccine was safe and effective for protection against inhalational anthrax.⁴⁸
Assessment of Competing Products. Comparisons of effectiveness
among all available products and between a new product and others already on the
market are possible. One could compare multiple single vaccines with various
combined (polyvalent) products, or currently licensed smallpox vaccine with both the
diluted form being tested and products now in the development pipeline. Industry
and university researchers have worked on some analyses of safety, effectiveness, and
cost. Is there enough detail and rigor in these comparative studies? How can
legislators assess the merits of the debate over side effects — and a proper remedy
for injury? Or ensure sound research about competing products? Or that the public
is better informed?
Production of Generic Biologics. Vaccines have not been prime
candidates for generic production in part because of the technologic difficulties in
measuring whether two products are equivalent. In fact, the Hatch-Waxman Act
(The Drug Price Competition and Patent Term Restoration Act of 1984, P.L. 98-417)
amendments to the FFDCA that deal with Abbreviated New Drug Applications were
not matched with a provision for abbreviated applications for biologics licenses
under the Public Health Service Act. Now, manufacturers and the public are looking
to newer biotechnology procedures as a possible route to lower medical costs.⁴⁹
Possible Legislative Solutions
Improving Post-Licensure Adverse-Event Surveillance. Congressional
approval of the National Childhood Vaccine Injury Act of 1986 (P.L. 99-660) set into

⁴⁷CDC, Progress Toward Global Eradication of Poliomyelitis, Jan. 2003-April 2004,
Morbidity and Mortality Weekly, vol. 53, no. 24, June 25, 2004, pp. 532-535, at
[http://www.cdc.gov/mmwr/preview/mmwrhtml/mm5324a5.htm]; and Amy Waldman,
Distrust Reopens the Door for Polio in India, The New York Times, Jan. 19, 2003.
⁴⁸AVIP, Anthrax Vaccine Immunization Program Pause, Oct. 27, 2004, at
[http://www.anthrax.osd.mil/resource/policies/policies.asp], visited Dec. 13, 2004.
⁴⁹Lester M. Crawford, Acting Commissioner of Food and Drugs, HHS, “The Law of
Biologic Medicine,” statement before the Committee on the Judiciary, U.S. Senate, June 23,
2004, at [http://www.fda.gov/ola/2004/fob0623.html]; and Arman H. Nadershahi and Joseph
M. Reisman, Generic Biotech Products: Provisions in Patent Drug Development Law,
BioProcess International, Oct. 2003, pp. 26-31, at
[http://www.knobbe.com/Pages/art10.htm], visited Nov. 2, 2004.

motion the VAERS activities in FDA and CDC for monitoring vaccine adverse
events. Congress may choose to strengthen surveillance programs such as VAERS,
address data coordination, and communicate surveillance analyses in ways that build
trust among concerned parents and patient advocacy groups.
When it comes to smallpox, Congress has acknowledged the need for the
consistent documentation of vaccine administration and side effects along with
coordination of the many related federal, state, and local activities.⁵⁰ As the smallpox
immunization program proceeds, Congress may want to review the completeness of
the surveillance program and the usefulness of its output.
Education and Risk Communication. Websites for HHS offices,
including CDC, the National Immunization Program, the Advisory Committee on
Immunization Practices, and the National Vaccine Program Office, and others have
hundreds of links to consumer-oriented health information, addressing reasons to
immunize, common misconceptions, safety, and even “evaluating immunization⁵¹
information on the Internet.” CDC and state health departments also try to ensure
that all health care providers are handing out the federally requested Vaccine⁵²
Information Statements each time a vaccine is administered.
Despite such Internet efforts and others, government education programs are not
reaching all who should be immunized or who have reservations. Congress might
consider additional approaches to public communication of risk and medical choices.
Studies in Pharmacoepidemiology and Pharmacoeconomics. Some
legislators, in considering cost, would broaden the analysis to include value-for-cost,
arguing that if the government is going to consider paying for drugs, it may as well
pay for the most effective ones. Researchers are adapting the study design and
statistical methods of epidemiology and economics, along with those fields’
traditional way of framing research questions, to questions regarding drugs and
vaccines.
Because declarations of comparative effectiveness could affect manufacturers’
market share and individuals’ access to drugs and vaccines, the success of that
activity could depend, in part, on all stakeholders’ trusting in the unbiased and expert
credentials of the entity analyzing and presenting the comparisons. Some have
suggested that the government, a public-private group, or a “quasi-governmental”
institution perform this function.⁵³ Others may feel such analysis is too fraught with
uncertainty for government decisions and is best left to individual physicians.

⁵⁰For example, in P.L. 107-188, Title XXVIII, Section 2801.
⁵¹The National Immunization Program website [http://www.cdc.gov/nip] has a link to the
National Network for Immunization Information, Parents: Evaluating Information on the
Web, at [http://www.immunizationinfo.org/parents/evaluatingWeb.cfm], visited Nov. 23,

2004.

⁵²CDC, Vaccine Information Statements, at [http://www.cdc.gov/nip/publications/vis/].
⁵³Gail R. Wilensky, How to Curb Spending on Drugs, The Washington Post, editorial, Feb.

15, 2004.

In its Medicare legislation, the 108th Congress directed the HHS Agency for
Healthcare Research and Quality (AHRQ) to “conduct and support research” dealing
with “the outcomes, comparative clinical effectiveness, and appropriateness of health
care items and services (including prescription drugs)....”⁵⁴ How to fund these
activities — and to what extent — remains a topic of debate.
Compensation. For those times when safety efforts have been unsuccessful,
earlier Congresses have addressed compensation. Since its creation by Congress in⁵⁵
1986, the National Vaccine Injury Compensation Program (VICP) has made many
awards, primarily to families of children, following injuries deemed to have been
associated with ACIP-recommended vaccinations. For several years, sentiment has
been growing in Congress that modifications to the program are needed to make it
more fair and efficient. Senator Bill Frist proposed changes to VICP in his Improved
Vaccine Affordability and Availability Act (S. 754, introduced April 1, 2003);⁵⁶
committee mark-up of the bill has been postponed indefinitely.
As the nation began to vaccinate certain military personnel and civilian health
care workers against smallpox, some Members of Congress discussed proposals that
would create non-tort mechanisms for smallpox vaccine injury compensation. On
April 30, 2003, President Bush signed the Smallpox Emergency Personnel Protection
Act of 2003 (P.L. 108-20), which the House and Senate had adapted from the
Administration’s proposal. It includes provisions to pay for smallpox vaccine injury-
related medical care, lost employment income under specified circumstances, and
death benefits. HRSA published the required injury table in August 2003⁵⁷ and the
administrative guidelines for the Smallpox Vaccine Injury Program in December
2003.⁵⁸ Because the perceived urgency for smallpox vaccination diminished
coincidentally with the compensation program’s passage, it remains theoretical to
what extent the program would have succeeded in recruiting volunteers.
Access
Successful development and production of a safe and effective vaccine does not
ensure that everyone who needs a vaccine gets it. People have to (1) know about it
and believe it will benefit them; (2) live near a health care provider willing to
administer it; and (3) be able to afford the cost of vaccination and follow-up care, if
necessary.

⁵⁴Section 1013, P.L. 108-173.
⁵⁵The National Vaccine Injury Compensation Program fact sheet is at
[http://www.hrsa.gov/osp/vicp/fact_sheet.htm], visited Nov. 15, 2004.
⁵⁶Mary Agnes Carey, Parties Seek Bicameral Compromise On Child Vaccine Compensation
Bill, CQ Weekly, May 17, 2003, p. 1192, at [http://www.cq.com], visited Nov. 10, 2004.
⁵⁷Smallpox Vaccine Injury Compensation Program: Smallpox (Vaccinia) Vaccine Injury
Table, Interim Final Rule, Health Resources and Services Administration, Federal Register,
vol. 68, no. 166, Aug. 2003.
⁵⁸Smallpox Vaccine Injury Compensation Program: Administrative Implementation; Interim
Final Rule, HRSA, Federal Register, vol. 68, no. 241, Dec. 16, 2003.

Problems
Vaccines fare better than prescription drugs in health benefits coverage in the
United States. In 2002, 75% of U.S. children between 19 and 35 months old had
completed the recommended series of vaccinations, not yet reaching the HHS
Healthy People 2000 and 2010 objectives of 80%. Recommended adult
immunization rates are even farther from HHS goals: 66% of non-institutionalized
adults at least 65 years of age reported receiving a flu vaccine within the past year,
far less than the HHS goal of 90% by 2010.⁵⁹ Also, there are regional and economic
disparities in access to immunization services. Reasons given for these problems
include insufficient coordination of varying eligibility rules among private insurers
and government vaccine programs; incomplete documentation of immunizations
achieved; and inadequate financing.⁶⁰
As federal and state agencies coordinated options and plans for smallpox
vaccination, weaknesses in the U.S. public health infrastructure became apparent.
These include the need for improvements in technology, training, hospital and
laboratory capacity, and communication among participants.⁶¹ In many developing
countries, inadequate public health infrastructure overshadows any question of
access. Congress has gone beyond a domestic focus and showed legislative interest
in the lack of access to vaccines in less developed areas around the world.
Possible Legislative Solutions
Congress has the opportunity to touch on access to safe and effective vaccines
in its consideration of prescription drug benefit bills and broader issues involving
global health.
Coordination of Government Financing Programs. Individual states
and assorted federal programs work toward increasing childhood immunization rates.
To improve immunization rates among U.S. children as well as the financial
efficiency of the efforts, legislative discussions could address difficulties of
coordination among the publicly funded vaccine programs, such as Medicaid, the
State Children’s Health Insurance Program, and Vaccines for Children.
Adult immunization insurance coverage and government financing is less
complete. Although Medicare covers the major, recommended vaccines for adults,
such as influenza and pneumococcal infections, many younger adults have no

⁵⁹These and other data collected and analyzed by CDC are available at, for example,
[http://www.healthypeople.gov/document/html/objectives/14-24.htm] and
[http://www.cdc.gov/nchs/data/hus/tables/2003/03hus051.pdf], both visited Nov. 10, 2004.
⁶⁰IOM, Calling the Shots: Immunization Finance Policies and Practices, Washington, D.C.,
National Academy Press, 2000; and IOM, Financing Vaccines in the 21st Century: Assuring
Access and Availability, Washington, D.C., National Academies Press, 2003.
⁶¹CRS Report RL31719, An Overview of the U.S. Public Health System in the Context of
Bioterrorism, by Holly Harvey and Sarah Lister.

coverage for these routinely recommended vaccines. Congress may consider funding
levels and financing strategies for vaccine-related care in the United States.
Payment for Vaccination and Follow-Up Care. In the context of medical
countermeasures to bioterrorism attacks, such as the smallpox vaccine, Congress may
consider questions about the payment for the vaccine administration and, then, for
follow-up and treatment, if necessary, of vaccine-related illness. As other vaccine
countermeasures are developed, Congress may need to consider who would have⁶²
access to that continuum of medical care.
Global Health. Concerns about access to vaccines are not limited to
biodefense and domestic use. Many government agencies and private groups work
toward international health objectives, such as eradicating polio. Some Members
noted concern for public health needs of developing countries worldwide and the
need to assist those countries in fights against infectious diseases. Their concern
stemmed from both humanitarian impulses and a growing awareness of the links
between poor health and economic and political instability. Their work resulted in
P.L. 108-25, the United States Leadership Against HIV/AIDS, Tuberculosis, and
Malaria Act of 2003. Legislators may want to increase access to existing vaccines,
spur the development of affordable new vaccines for which the technology already
exists — an issue of both availability and access, and consider increased long-term
investment in vaccine development for these diseases

⁶²See also: National Vaccine Advisory Committee, Strengthening the Supply of Routinely
Recommended Vaccines in the United States: Recommendations from the National Vaccine
Advisory Committee, Journal of the American Medical Association, vol. 290, no. 23, Dec.

17, 2003, pp. 3122-3128; R. Giffin et al., Childhood Vaccine Finance And Safety Issues,

Health Affairs, vol. 23, no. 5, Sep./Oct. 2004, pp. 98-111; and F.A. Sloan et al., The
Fragility of the U.S. Vaccine Supply, New England Journal of Medicine, vol. 351, Dec. 2,

2004, pp. 2443-2447.