The story of modern vaccines begins in 1749, when Dr. Edward Jenner observed that milkmaids exposed to cowpox later subsequently not contract

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2 The story of modern vaccines begins in 1749, when Dr. Edward Jenner observed that milkmaids exposed to cowpox later subsequently not contract smallpox. Dr. Jenner used fluids from a cow s blisters to develop a vaccine to intentionally protect individuals from smallpox.. 2

3 Our modern vaccines have built upon Jenner s observation that exposure to a weakened version of a bacteria or virus provides protection from the disease. In that way, vaccines can be thought of like a training course for the immune system. They prepare the body to fight disease without exposing it to disease symptoms. Vaccines help your immune system fight infections faster and more effectively. When you get a vaccine, it sparks your immune response, helping your body fight off and remember the germ so it can attack it if the germ ever invades again. And since vaccines are made of very small amounts of weak or dead germs, they won t make you sick. Vaccines often provide long-lasting immunity to serious diseases without the risk of serious illness and because you avert the disease and any serious consequences, vaccines are much safer than immunity achieved through natural infection. 3

4 Vaccines are among top 10 achievements in public health during the 20 th century; from contributing to higher life expectancy and decreasing disease burden, they truly have transformed our lives. Each year, childhood immunizations save 42,000 lives & prevent 20 million cases of disease in the U.S. alone. Annually in the U.S., childhood immunizations save $13.6 billion in direct medical costs, and $68.9 billion when indirect societal costs are included. Benefits for those vaccinated & unvaccinated (herd immunity). 4

5 Vaccines listed here are all FDA approved not all are given routinely Dark blue = vaccines given over LIFETIME One disease has been eradicated smallpox Polio eradication efforts are underway; measles is likely to follow 5

6 Same general development pathway as other pharma products, with extra step of ACIP and post-market review. 6

7 Because vaccines are given to healthy people, the risk/benefit ratio is very different from drugs. - Demonstrating safety is paramount - Large trials - Global 7

8 Established in Its mission is to recommend the use of FDA-licensed vaccines in the U.S. given the broader public health and epidemiologic concerns of controlling infectious diseases. The ACIP is a very powerful committee that creates the national standard of care for immunization. Historically, the CDC & HHS always adopts ACIP recommendations ACIP meets in person three times a year in Atlanta. Membership Voting members are largely medical doctors from academia and public health departments. 1 voting member is the consumer representative. Working groups, organized by disease groups (e.g. Pertussis WG, Influenza WG ) meet at least once a month to conduct extensive reviews of all relevant data and to prepare draft policy recommendations for consideration by the full ACIP in open meetings. Terms are 4 years 8

9 For each recommended vaccine, ACIP develops written guidance for administration (published in footnotes of schedule), including: age for administration dose and frequency of administration precautions and contraindications for use information on adverse events. Recommendations vary. Examples: - Routine: MMR vaccine administer first dose at age months and second dose at age 4-6 years. Schedule footnotes clearly say routine recommendation - Permissive: HPV vaccine for males age Age-based: flu vaccine recommended for all people 6 mo and older - Risk-based: pneumococcal conjugate vaccine (PCV-13) recommended for all adults age 19 yrs and older with immunocompromising conditions 9

10 The U.S. government defines the economics & potential market for vaccines because the CDC ACIP recommendation process drives demand and adoption in both the public & private sectors. ACIP determines whether a vaccine is: included in the Vaccines for Children (VFC) Program (Medicaid children) covered by private plans per the Affordable Care Act (ACA) covered by Medicare GRADE GRADE = Grading of Recommendations, Assessment, Development and Evaluation In addition to the 5 factors, the ACIP also now uses an approach called GRADE, developed by the Word Health Organization, to evaluate the data it reviews. Randomized controlled trials are weighed more heavily than observational studies for instance. Through GRADE, ACIP makes Category A recommendations (made for all persons in an age or risk group) and Category B recommendations (made for individual clinical decision making). 9

11 Because vaccines are given to healthy people, there is heightened awareness and less tolerance for adverse events / side effects. And today, many parents and healthcare practitioners have never seen diseases like polio, mumps, and measles etc, so the benefits of vaccines are often underestimated. Even following licensure and approval, multiple systems are in place to continually collect information on adverse events following administration of a vaccine. A few of the U.S. systems are listed on this slide, but global monitoring systems also exist. These systems collect and review data and any potential safety signals are flagged. If the signal is deemed significant, ACIP can revise or revoke its recommendation. Vaccine Adverse Event Reporting System = VAERS - National vaccine safety surveillance program co-sponsored by CDC & FDA - Anyone (providers, manufacturers, & vaccine recipients) may report information about adverse events that occur after administration of vaccines. - VAERS is part of an overlapping vaccine safety system in the U.S. that also includes Vaccine Safety Datalink and Phase IV studies. Companies have an important role to play in encouraging providers to report adverse events to VAERS. Sales forces are a key part of this. VSD and PRISM are two networks of providers that work to collect and analyze immunization data to continually monitor vaccine safety using electronic health data. VSD Works with with CDC s Immunization Safety Office. This includes information on vaccines: the kind of vaccine given to each patient, date of vaccination, and other vaccinations given on the same day. The VSD also uses information on medical illnesses that have been diagnosed at doctors offices, urgent care visits, emergency department visits, and hospital stays. The VSD conducts vaccine safety studies based on questions or concerns raised from the medical literature and reports to the Vaccine Adverse Event Reporting System (VAERS). PRISM FDA partnership with healthcare providers and insurance companies; using a computer-based system to analyze data to identify potential associations between vaccines and adverse events Finally, CDC s CISA project is a collaboration with 7 medical research centers that conducts clinical research studies on vaccine safety, adverse event risk factors, and identifies prevention strategies for adverse events, particularly among special populations. Boston Medical Center Cincinnati Children s Hospital Medical Center Columbia University Duke University Johns Hopkins University Kaiser Permanente Northern California Vanderbilt University CISA Current Activities Clinical Case Reviews: CISA provides a clinical case evaluation service for US healthcare providers who have vaccine safety questions about a specific patient residing in the US. CISA provides clinical expertise in various disciplines, including neurology, allergy, immunology, pediatrics, hematology, and obstetrics/gynecology. Expert Evaluation of Vaccine Safety Issues: CISA experts provide advice that has led to a broader understanding of vaccine safety issues and informs clinical or public health practices. CISA has also reviewed technical issues and contributed to Advisory Committee on Immunization Practices (ACIP) recommendations. Research: To advance knowledge of vaccine safety and inform clinical and public health practice clinical research is essential. CISA has published and continues to develop research studies that address vaccine safety priorities, such as those identified in the US National Vaccine Plan. Current priority areas for CISA research studies include influenza vaccine safety, vaccine safety in persons with autoimmune diseases, and vaccine safety in pregnant women. CISA complements other vaccine safety systems and focuses its efforts on scalable, prospective studies for US-licensed vaccines. These studies are designed to address clinical vaccine safety questions in targeted or special populations that are often excluded from pre-licensure clinical trials. CISA is well suited to study more common, non-medically attended events (e.g., fever) and to collect biological specimens after vaccination. CISA investigators also have access to special populations (e.g., persons with autoimmune diseases) and specialists who care for these patients. Public Health Response: CISA has procedures in place to assist in response to vaccine safety emergencies, such as a pandemic, and to assist state health departments during an emergency. 10

12 No pharmaceutical intervention is completely without risk but which is more likely? Lightning Strike Odds according to National Weather Service - odds of being struck in US in one year = 1 in 700,000 - Odds of being struck in lifetime = 1 in 3,000 11

13 In the 1970s and 1980s, litigation against vaccine manufacturers forces all but one manufacturer of DTP out of the market. Concerned that the country s vaccine supply was at risk, Congress enacted unique legislation governing liability called the National Vaccine Injury Compensation Program (VICP), which was established in The VICP is a no-fault alternative to the traditional tort system for resolving vaccine injury claims. It sits within the U.S. Court of Federal Claims and cases are heard by Special Masters who are appointed by the Court. VICP is run by HRSA, the Health Resources and Services Administration within HHS. The excise tax is collected by companies upon sale of vaccine to providers etc. New vaccines with pediatric or maternal indications and a routine recommendation from the ACIP must be added to the VICP program through a multi-step process that includes enactment of new tax legislation (e.g. update to flu vaccine tax language to include quadrivalent vaccines). 12

14 Looking forward, the pipeline for new vaccines includes RSV, Group B strep, Zika, and improved influenza vaccines. 13