JACI Highlights - July 2010

Respiratory virus infections are common in patients with hypogammaglobulinemia

Patients with primary hypogammaglobulinemia suffer from recurrent bacterial infections. It is generally thought that they are not more prone to respiratory viral infections than immunocompetent individuals. Kainulainen et al conducted a prospective, 12-month longitudinal study designed to capture respiratory viral infections that occurred in 12 adult patients with primary hypogammaglobulinemia. Nasal swab samples and induced sputum samples were provided by the subjects every 3 months, irrespective of the symptoms, and at the onset of acute respiratory infection. Viruses were identified through use of PCR tests for 15 respiratory viruses. The patients had a median of 5 episodes of respiratory infection, this being significantly more than in the spouses who served as controls. Rhinovirus was the most common virus, and it was positive in one third of the infections. Interestingly, some patients had recurrent and persistent rhinovirus infections. Rhinovirus was found both as a sole pathogen and together with bacteria. The most long-acting persistence of the same rhinovirus, confirmed with sequencing, was 4 months in a patient with XLA; this compared with shedding of 1-2 weeks in immunocompetent adults. The observations raise interesting questions about the role of humoral immunity in defence of respiratory viral infections.

“Recurrent and persistent respiratory virus infections in patients with primary hypogammaglobulinemia” Remarks by Kainulainen et al. (JACI July 2010 / Volume 126, No. 1)

The Journal of Allergy and Clinical Immunology (JACI) is the official scientific journal of the AAAAI, and is the most-cited journal in the field of allergy and clinical immunology.

Contact Your Senators!

Urgent! Ask Your Senators to Support Kerry/Wyden et. al IVIG Amendment!

Take Action!

Your help is needed! This could be our last chance this year!

With Majority Leader Harry Reid’s introduction of the Senate’s merged health care reform bill, the Senate debate on health care reform has begun.

Senators Kerry (D-MA), Wyden (D-OR) and others have filed an IVIG access amendment to the bill. The amendment is based on S. 701, the Medicare Patient IVIG Access Act, but scaled back. It establishes a three year demonstration project that would authorize payment for the items and services necessary to infuse IVIG in the home setting.

IDF supports this amendment as the only viable means to strengthen the Medicare Part B home infusion benefit for the next few years. Current law does not allow coverage of nursing services in the home and therefore Medicare patients with primary immunodeficiency diseases (PIDD) do not have access to IVIG home infusion. Currently, Medicare will pay for the plasma product, but not pay for the items and services for a home infusion, making the benefit ineffective. Passage of this amendment can make the difference between Medicare patients with PIDD receiving their treatment or going without.

Also, the Kerry/Wyden et al. IVIG Amendment requires additional studies on IVIG access and reimbursement, including an update of the February 2007 government report entitled “Analysis of Supply, Distribution, Demand and Access Issues Associated with Immune Globulin Intravenous (IGIV).”

The end of the health care reform process is near. Please contact your Senators to urge them to help address IVIG access in health reform. Use IDF’s Action Alert to ask your Senators to support the Kerry/Wyden et al. IVIG Access Floor Amendment.

This is what Dr. Hans Ochs is working on at Seattle Children’s Hospital.

He is one of the top Dr’s who works on our behalf.

Seattle Children’s researcher Dr. Ochs is looking at the connection between immune deficiencies and genes.

Primary immune deficiency diseases (PIDD), an umbrella term referring to more than 130 genetic defects involving the immune system, affect as many as 500,000 Americans and 10 million people worldwide.

People with PIDD are unable to fight off bacteria, viruses, parasites, fungi and malignant cells, which can lead to frequent infections that are difficult to fight, and to an increased incidence of cancer.

PIDD is not contagious; it is hereditary — parents can pass it to their children. According to the Centers for Disease Control and Prevention (CDC), many primary immunodeficiencies are the result of a single gene defect.

The diseases can strike males and females of all ages, with the more severe immunodeficiency diseases usually detected most frequently in children.

Early diagnosis and treatment of PIDD is essential to prevent the infections from causing permanent damage, especially to the lungs. The condition can go undetected because the symptoms appear as “ordinary” infections of the sinuses, ears or lungs, or as gastrointestinal problems or inflammation of the joints.

As a result, families and physicians are often unaware that the troubling conditions they see in the child are actually caused by an underlying defect of the immune system.

Signs, Symptoms and Treatment of Primary Immune Deficiency Diseases

How do physicians know if a child has a primary immune deficiency disease? A pattern of recurring illnesses may be explained by an underlying PIDD.

According to the Jeffrey Modell Foundation, some warning signs of PIDD that physicians should look for include:

• Eight or more new ear infections within one year
• Two or more serious sinus infections within one year
• Two or more months on antibiotics with little effect
• Two or more pneumonias within one year
• An infant’s failure to gain weight or grow normally
• Recurrent deep-skin or organ abscesses
• Persistent thrush in mouth or elsewhere on the skin after age 1
• Need for intravenous antibiotics to clear infections
• Two or more deep-seated infections
• A family history of PIDD

Dr. Hans Ochs, professor of pediatrics at Seattle Children’s and the University of Washington, emphasizes that it’s important to know the family’s medical history.

“If a pregnant woman had a male sibling or other close male relatives who died prematurely of an infection or has other children with immune deficiencies, I’d recommend that the family be evaluated for a genetic form of PIDD and, if the disorder is X-linked, the woman undergo prenatal testing.

“That way we can take appropriate measures right after the baby is born. Also, some forms of PIDD diseases are associated with a tendency to bleed and the obstetrician may recommend a Caesarean section to prevent damage to the baby.”

Infections in a patient with PIDD can be chronic and severe and re-occur frequently. These infections tend to require prolonged therapy. Patients also may respond poorly to a conventional course of antibiotics.

The treatment for immunodeficient patients depends on the severity of the infections and other health problems and the specific genetic defect that is involved.

Most patients require aggressive treatment with antibiotics or antiviral agents. Some also benefit from regular (every two to four weeks) antibody replacement therapy (often referred to as IVIG therapy), which works by replacing the antibodies that the body cannot make on its own.

Finally, some patients require bone marrow transplant or other alternative treatments. In the future, gene therapy may also be a viable treatment option.

Progress Seen in PIDD Research

About 50 years ago, doctors developed a way to prevent infections by replacing antibodies via infusion of gamma globulin, a component of blood serum that is high in disease-fighting ability.

Gamma globulin from many donors is pooled and, in recent years, is administered intravenously or subcutaneously, giving the patient the antibodies he or she needs. However, this is a control measure, not a cure; PIDD patients may need infusions their entire lives.

In fact, about half of the patients the PIDD clinic sees are adults. “Many of these individuals would have died in childhood if it weren’t for antibiotics, IVIG or other treatments for infection,” said Dr. Ochs.

Seattle Children’s is one of only a few hospitals in the country researching and treating patients with PIDD and is a leader in developing new diagnostic techniques to combat PIDD.

By the early 1990s, Children’s researchers, such as Dr. Ochs and Dr. David Rawlings, head of Children’s Immunology division and associate professor of pediatrics and immunology at the University of Washington, were making the connection between immune deficiencies and genes.

New treatment possibilities

This opened new possibilities for treatment. Recent work at Children’s and elsewhere, coupled with improved methods for gene sequencing and the human genome project, has led to rapid acceleration in identification of the genetic basis of many previously unknown immune disorders.

In recent years, a team of Children’s researchers led by Drs. Ochs and Rawlings have helped open new treatment possibilities, such as gene therapy.

One important area of research focuses on removing stem cells from a patient, infecting the cells with a normal copy of the defective gene, and returning the stem cells to the patient.

“Most primary immune diseases begin in childhood, which means Children’s sees a lot of these kids,” said Dr. Rawlings. “As a result, we have developed an expertise no one else in the region has and we have become the referral center for children and adults with immune problems.

“We have genetic testing capabilities no one else has. We serve as an international center for prenatal and neonatal testing, and for testing of adults to identify carriers of immune deficiency disorders.”

Dr. Ochs is also the primary investigator for a $12 million grant administered by the U.S. Immune Deficiency Network (USIDnet), an international consortium of 50 researchers and specialists in the field.

The consortium studies PIDD, educates young investigators about the disease and administers, reviews and provides grants. It also funds registries to track the long-term health of patients and provides an anonymous repository that makes DNA or cell lines generated from selected patients available in order to study molecular defects of these diseases.

The Immune Deficiency Foundation (IDF) and the Jeffrey Modell Foundation, two organizations founded by parents of children affected by PIDD, both support research in Dr. Ochs’ laboratory in Children’s Division of Immunology.

Additionally, the Jeffrey Modell Foundation recently made available matching funds for an endowment for a fellowship in primary immunodeficiency disease.

This is from the CSL Behring Advocacy Newsletter:

Health Care Reform - Will Plasma and Recombinant Therapies be Protected?

CSL Behring Public Affairs

CSL Behring is committed to educating decision-makers and impacting public policies that affect patient access to care. The company has an active public affairs group in the United States including Dennis Jackman, Sr. Vice President for Public Affairs; Patrick Collins, Director, Public Affairs; Kim Isenberg, Manager State Government Affairs and Ryan Faden, Manager State Government Affairs. All four have extensive public policy backgrounds and work closely with affected stakeholders and political thought leaders to affect change. Please contact them with any comments you may have regarding public policy issues.

As part of his platform in running for the presidency, Barack Obama pledged to reform the health care system in the United States. Now that he is in office, President Obama is strongly advocating for such reform as his highest domestic priority. With a supportive Democratic Congress, the chances for reform are very possible.

What does health care reform mean in terms of accessing life-saving plasma and recombinant therapies? Moreover, how will such reform impact the patient populations who rely on these therapies?

Several issues currently being debated as part of health care reform are of specific interest to the plasma and recombinant user communities. They include the following:

Increasing the Medicaid Drug Rebate
All manufacturers of drugs and biologics, including those who produce plasma and recombinant therapies, pay a rebate to the federal government in order to have state Medicaid programs cover their therapies. This rebate is currently 15.1% of the Average Manufacturers Price for the individual therapy. The President proposed increasing this percentage to 22.1% to raise revenue in large part to extend health care coverage to the uninsured. Such a proposal will not only raise the rebate to be paid in order to have Medicaid cover such therapies, but it will also increase the discount institutions within the 340B Public Health Service (PHS) pricing program acquire therapies. These entities, including a majority of hemophilia treatment centers, purchase product at the PHS mandated discount rate – which is identical to the Medicaid rebate.

This proposal would raise revenue but there are some unintended consequences that could ensue. They include a potential negative impact on research and development into new product formulations and rare disease therapies. This is due to the onerous financial burden that might be placed on manufacturers of new therapies for small patient populations of which a high percentage are enrolled in Medicaid or obtain their therapy from PHS programs.

President Obama signing
legislation into law

CSL Behring is participating in a collaborative effort with patient advocacy organizations and industry to ensure that consideration for a rebate increase for orphan populations – populations with fewer than 200,000 people with the disease — takes into account the impact of small patient populations that rely on plasma and recombinant therapies.

Comparative Effectiveness Research
As part of the stimulus package approved by the Congress, the Comparative Effectiveness Research Institute was created. President Obama and the Democrats in Congress are pushing comparative effectiveness as a way to save money for larger health care reform efforts while obtaining clinical data on how different treatments work for the individual.

Comparative effectiveness research would analyze different brands of therapy within a class of therapies to determine which brands work best. This may seem ideal for large chemical compound drugs, but for biologics such as plasma and recombinant therapies, we have seen that a “one size fits all” approach does not work. The concern with comparative effectiveness is that such clinical findings will be used to create restrictive formularies and preferred drug lists, as has occurred in the United Kingdom and France. This could prove devastating to patients in the United States if implemented for biologics such as plasma and recombinant therapies, since the possibility exists that decision making would be taken out of the hands of physicians. This is very dangerous for rare diseases where biologic therapies do not work identically for everyone and where physician/patient access to the most appropriate therapies is needed.

Accelerating Access to Generic Biologics
President Obama and the Congress also propose the creation of an accelerated pathway to generic biologics (also called follow-on biologics) as a way to save on health care costs. At present, there is no approval process for generic biologics. A pathway for generic biologics will need to ensure protections for patent rights of branded therapies for a reasonable period of time, in order to ensure an adequate return on the investments by creating innovative therapies, while also adhering to clinical trial requirements to ensure product safety.

There are two proposals currently active within Congress. One version of the legislation would create a viable generic biologics pathway similar to that for drugs, with a data exclusivity period of 12 years. All safety measures associated with the clinical trial process would remain in place. This provision would protect patients through appropriate safety measures and innovation by allowing manufacturers the opportunity to recoup their investment after years of developing a therapy for a small patient population. Without such protections, there is a real risk that biologics companies will no longer innovate and pursue new therapies.

The other proposal, which would allow only five years of exclusivity and is similar to the Obama Administration proposal, calls for seven years exclusivity. This proposal does not require as many safety protections for generics as what currently exist within a branded therapy clinical trial process. As a result, this proposal would make it far easier to create generic biologics for sale in the United States — albeit with reduced safety assurance.

Removal of Lifetime Caps on Private Health Plans
Many private insurance plans have in place a lifetime ceiling on costs for the individual, with some caps as low as $1 million or less. For those with a rare disease, such as those reliant on plasma and recombinant therapies, an individual can reach their cap quickly. This could result in a person having their therapy rejected by their existing health plan.

Both the Obama Administration and the Democrats in Congress have proposed completely removing lifetime insurance caps on private health insurance plans. This is a very positive step toward improving coverage for those with rare conditions reliant on plasma and recombinant therapies.

Patient organizations reliant on plasma and recombinant therapies have re-enlisted the Plasma User Coalition to ensure that access to therapy is not adversely impacted. The removal of such caps addresses one of the Coalition’s general principles.

Plasma User Coalition General Principles

1. Recognition that one size does not fit all in terms of treatment

2. Individuals with rare diseases must have access to: appropriate specialized treatment, centers of excellence; full range of treatments determined by the physician; timely treatment and affordable coverage.

3. Removal of lifetime insurance caps and unreasonable out of pocket expenses.

Over the course of the summer, the Congress will attempt to reach agreement on health care reform. It is imperative that those who rely on plasma and recombinant therapies make their voices heard so their representatives will ensure continued access to these therapies.

IDF Needs Your HELP to Fix IVIG Access and Reimbursement
Contact Your Senators and Representative!

After the 4th of July recess, Senate and House Committees will begin to consider the much talked about health care reform legislation, including changes to the Medicare program. And both the House of Representatives and the United States Senate will continue to debate health care reform during the month of July. It is imperative that the language in HR 2002 and S. 701, the Medicare Patient IVIG Access Act be included in any health care reform legislation passed by this Congress. Your voice at this critical time is important for both chambers of Congress to hear!!

The members of the Senate and the House need to know that people care about IVIG and they need to have IVIG access problems addressed in the health care reform bills.

Please act now and ask your families and friends to contact their Senators and Representative by participating in IDF’s Action Alert. You make the difference!

Contact Your Representative

In 2005, Congress changed the way Medicare pays for IVIG and, as a result, many doctors no longer provide the treatment - making it harder for patients to get IVIG.  Also, current law does not pay for the nursing and IV items needed for IVIG therapy administered in the home. These serious problems affect the entire community as an increasing number of private pay insurers are following Medicare’s lead.

To fix this problem, Representatives Israel (NY), Brady (TX) and, Schwartz (PA) introduced HR 2002, the Medicare Patient IVIG Access Act, and Representatives Matsui (CA) and Tanner (TN) introduced HR 1765. Both of these bills are critical to patients who rely on IVIG therapy for their health, as they correct the current problems and respond to government reports that confirmed these problems.

Please contact your Representative’s office and urge them to include HR 2002 and HR 1765 in any health care reform legislation passed by this Congress. Call the US Capitol Switchboard at (202) 224-3121 and request to be transferred to your Representative’s office. Please also CLICK HERE TO SEND THE ACTION ALERT LETTER to your Representative!

Contact Your Senators

In 2005, Congress changed the way Medicare pays for IVIG and, as a result, many doctors no longer provide the treatment - making it harder for patients to get IVIG.  Also, current law does not pay for the nursing and IV items needed for IVIG therapy administered in the home. These serious problems affect the entire community as an increasing number of private pay insurers are following Medicare’s lead.

To fix this problem, Senators Kerry (MA), Alexander (TN), Wyden (OR), Whitehouse (RI) and Brownback (KS) introduced the Medicare Patient IVIG Access Act, S 701.

Please contact both of your Senators, and urge them to include S. 701 in any health care reform legislation passed by this Congress. Call the US Capitol Switchboard at (202) 224-3121 and request to be transferred to your Senator’s office. Please also CLICK HERE TO SEND THE ACTION ALERT LETTER to both of your Senators!

IDF Action Alert - Contact Your Representative!

IDF needs your HELP to Fix IVIG Access and Reimbursement

Take Action!

Contact Your Representative!

Both the House of Representatives and the United States Senate are currently debating and will continue to debate, during the month of July, health care reform legislation.  It is imperative that the language in HR 2002 and S. 701, the Medicare Patient IVIG Access Act be included in any health care reform legislation passed by this Congress.  Your voice at this critical time is important for both chambers of Congress to hear!!

In 2005, Congress changed the way Medicare pays for IVIG and, as a result, many doctors no longer provide the treatment - making it harder for patients to get IVIG.  Also, current law does not pay for the nursing and IV items needed for IVIG therapy administered in the home. These serious problems affect the entire community as an increasing number of private pay insurers are following Medicare’s lead.

To fix this problem, Representatives Israel (NY), Brady (TX) and, Schwartz (PA) introduced HR 2002, the Medicare Patient IVIG Access Act, and Representatives Matsui (CA) and Tanner (TN) introduced by HR 1765. Both of these bills are critical to patients who rely on IVIG therapy for their health, as they correct the current problems and respond to government reports that confirmed these problems.

Please contact your Representative’s office and urge them to include HR 2002 and HR 1765 in any health care reform legislation passed by this Congress.  Call the US Capitol Switchboard at (202) 224-3121 and request to be transferred to your Representative’s office.  Please also send a letter!

This is another reminder from the IDF to take action on this bill ASAP.   Please take a couple of minutes and do this to ensure that we, as PIDD patients, have access to our much needed IgG therapy.

Action Alert! - We Need Your Help!
New Senate Legislation to fix Access and Reimbursement

Many members of primary immunodeficiency community experience delays in and denial of treatment due to insufficient Medicare reimbursement. A report from the Office of the Inspector General showed Medicare reimbursement for IVIG is lower than the cost many providers pay for the product. As a result, a number of physicians and hospitals cannot afford to administer IVIG treatment to Medicare patients. This serious problem affects the entire community as an increasing number of private pay insurers are following Medicare’s lead to determine reimbursement rates for IVIG.

To fix this problem, legislation was introduced March 25, 2009 into the United States Senate. Senators John Kerry (MA), Lamar Alexander (TN), Sheldon Whitehouse (RI), Ron Wyden (OR) and Sam Brownback (KS), introduced the Senate Bill S. 701, The Medicare Patient IVIG Access Act. An identical House bill is expected to be introduced by the end of April 2009. This legislation, offers a solution to the current IVIG access crisis by establishing appropriate reimbursement in all sites of care for our patients. It also changes current policy regarding Medicare coverage of home infusion to include the cost of items and services related to the administration of IVIG in the home for primary immunodeficient patients.

IDF is working in coalition with other patient organizations and physician organizations including the American Academy of Allergy Asthma and Immunology (AAAAI) and the Clinical Immunology Society (CIS). With the strong leadership of the policymakers who have signed onto support this legislation, IDF hopes to improve access to treatment and the quality of life of the countless patients who struggle with negative health outcomes, increased intervals of care, change in site of infusion, difficulty finding providers, and denial of treatment.

Click here to view the Medicare Patient IVIG Access Act page

What you can do to help

Go to the IDF Action Alert and contact your Senators to ask them to sign on as cosponsors to S. 701, The Medicare IVIG Patient Access Act. With your help, we can move forward to restore proper access to this life- saving treatment for patients with PIDD. Your legislators need to know why IVIG therapy is so important, so please utilize the text box to add the story of how IVIG treatment impacts your life, focusing on any access problems.

Be the voice of the PIDD patient community!

Meet with your legislators at home

April 6-17 marks the Spring District Work Period. During this time, your Senators and Representatives will be working at home in their district offices. This presents the ideal opportunity to speak with your legislators about the importance of this legislation. As a constituent, it is important that you share your concerns with them. IDF encourages you to visit your legislators during the district work period and ask your Senators to sign on to cosponsor S. 701, The Medicare Patient IVIG Access Act. Ask your Representative to be an original cosponsor of the House companion bill, which will soon be introduced.

To locate contact information for the local offices for your Senators and Representative, simply visit www.senate.gov and www.house.gov.

Take Action!

New Senate Legislation to Fix Access and Reimbursement for IVIG in the New 111th Congress

Take Action!

Contact Your Senators

Many members of the primary immunodeficiency community experience delays in and denial of treatment due to insufficient Medicare reimbursement. A report from the Office of the Inspector General showed Medicare reimbursement for IVIG is lower than the cost many providers pay for the product. As a result, a number of physicians and hospitals cannot afford to administer IVIG treatment to Medicare patients. This serious problem affects the entire community as an increasing number of private pay insurers are following Medicare’s lead to determine reimbursement rates for IVIG.
To fix this problem, legislation was introduced March 25, 2009 into the United States Senate. Senators John Kerry (MA), Lamar Alexander (TN), Sheldon Whitehouse (RI), Ron Wyden (OR) and Sam Brownback (KS), introduced the Senate Bill S. 701, The Medicare Patient IVIG Access Act. An identical House bill is expected to be introduced by the end of April 2009. This legislation, offers a solution to the current IVIG access crisis by establishing appropriate reimbursement in all sites of care for our patients. It also changes current policy regarding Medicare coverage of home infusion to include the cost of items and services related to the administration of IVIG in the home for primary immunodeficient patients.
IDF is working in coalition with other patient organizations and physician organizations including the American Academy of Allergy Asthma and Immunology (AAAAI) and the Clinical Immunology Society (CIS). With the strong leadership of the policymakers who have signed onto support this legislation, IDF hopes to improve access to treatment and the quality of life of the countless patients who struggle with negative health outcomes, increased intervals of care, change in site of infusion, difficulty finding providers, and denial of treatment.

What you can do to help

Contact your Senators to ask them to sign on as cosponsors to S. 701, The Medicare IVIG Patient Access Act. With your help, we can move forward to restore proper access to this life- saving treatment for patients with PIDD. Your legislators need to know why IVIG therapy is so important, so please utilize the text box below to add the story of how IVIG treatment impacts your life, focusing on any access problems.

Be the voice of the PIDD patient community!

Think about how often you’ve tried to explain PIDD to someone new.   Think about the reaction(s) you may have gotten.  Many thought you might be crazy, many thought you might have ANOTHER disease, and many just stared at you like a deer caught in the headlights.

This month is the time we attempt to and often successfully educate others about our PIDD.   The Immune Deficiency Foundation has offered some great suggestions in their recent newsletter.   I’m going to paste the info and the links here so that you can TAKE ACTION and help SPREAD THE WORD.

April is PIDD Awareness Month
Time to Spread the Word!

This April, celebrate Primary Immunodeficiency Disease Awareness Month by educating others.  Now is the time to take action in your community and below is a list of some of the many ways you can help spread awareness. Let us know of your activities and success with promoting awareness, and remember that your efforts might help save a life!

Get the Word Out

Set up a display or post materials at hospitals, health fairs, libraries, plasma centers or other community gathering spots.

Awareness Month is the perfect time for you to become involved and help others understand more about primary immunodeficiency diseases. The “Is it Just and Infection?” poster is the perfect poster for your display, and is available by contacting IDF at 800.296.4433, or by clicking here.

If you want more posters or you need additional handout information about primary immunodeficiency diseases and IDF services, simply contact IDF at 800.296.4433 or e-mail idf@primaryimmune.org. All materials are free of charge.

Contact Your Local Media

Reporters pay attention to stories about real people, so share your own personal experience with primary immunodeficiency disease. Use Primary Immunodeficiency Disease Awareness Month as an opportunity to educate the public.
Click here to download the IDF Media Kit; which has ideas, and sample materials to send to your local media: 

THINK ZEBRA!

In medical school, many doctors learn the saying, “when you hear hoof beats, think horses, not zebras.” Most physicians are taught to focus on the likeliest possibilities when making a diagnosis, not the unusual ones.  However, sometimes physicians need to look for a zebra. Primary immunodeficiency patients are the zebras of the medical world. So IDF says THINK ZEBRA! Let’s teach the world about “ZEBRAS” while raising funds to help promote awareness of primary immunodeficiency diseases.

Click here to visit www.primaryimmune.org/zebra and learn more about IDF’s THINK ZEBRA! campaign:
Blue Jeans for Healthy Genes
Blue Jeans for Healthy Genes Day is a special event when people are asked to make a donation to the IDF for the chance to wear jeans (to work, school, etc.) and join the fight against primary immunodeficiency diseases. An alternative way to participate is by hosting the Hanging Up Jeans program, which creates awareness and raises funds by offering small cut-out pairs of paper jeans for a donation. The jeans are then signed by their sponsor and put on display.

Host a Blue Jeans for Healthy Genes Day or the Hanging Up Jeans program at workplaces, companies, schools and organizations and seize the opportunity to educate those around you and raise funds for further research and patient programs. Blue Jeans for Healthy Genes kits and more information are available here.
Take Action!
Primary Immunodeficiency Disease Awareness Month is the ideal time to undertake some of these ideas or try a few of your own. IDF staff is available to answer questions, help with plans and provide the materials you need.  For more information, contact Adam Freestone at 443.632.2546 or afreestone@primaryimmune.org.

Standards of Care Introduced in Minnesota

A growing number of people living with primary immune deficiencies, alpha1-antitrypsin deficiency, hemophilia and von Willebrand disease are experiencing problems accessing their medically appropriate therapies and services. This is due in large part to cost-containment measures by state Medicaid and other public programs and private insurers. These patients and their families have learned that politicsis not a spectator sport. They have made advocacy a primary focus. The patient organizations have joined forces with providers and industry to tackle the complex legislative and regulatory policy issues that affect quality of care and provide access to their lifesaving therapies so they can lead productive lives. Standards of Care legislation offers protection to people living with these rare genetic disorders.

CSL Behring Public Affairs has made it a priority to join with patient advocacy organizations across the United States to pursue state-based Standards of Care legislation ensuring access to all plasma-derived therapies and recombinant analogs, including necessary ancillary services. The drive for Standards of Care began with the passage of model legislation in the state of New Jersey in 2005. Pennsylvania and Minnesota followed with bills introduced in 2007. In 2008, the number of states pursuing Standards of Care grew to include California. In 2009, more states intend to continue this trend. Alabama, Missouri and Texas are in discussions with legislative leaders; California and Indiana are exploring regulatory implementation; and Pennsylvania, Massachusetts, Connecticut and Minnesota are introducing legislation.

Elected and appointed officials, lobbyists and historians often warn that changing public policy takes commitment, hard work, patience, persistence, perseverance, diligence and even luck. Pursuing Standards of Care legislation is no exception.

Of course, momentum can help as well. In Minnesota last spring, Standards of Care initiatives were infused with new energy when a coalition of patient advocates from across the state attended a legislative day in St. Paul, Minnesota. Minnesotans who rely on plasma protein therapies, their families and representatives from industry joined forces to educate lawmakers about rare chronic diseases. They shared their personal stories with legislators and educated them on the importance of access. Every participant played a vital role in advancing Standards of Care.

As legislators listened to their stories, the participants were empowered to continue to reach out and build awareness. Some of the participants even visited their legislators and contacted candidates during the summer months.

Patient advocates had become a resource to their elected officials on issues of health care, rare genetic diseases and access. The Senate and House authors were pleased to introduce newly drafted Standards of Care in 2009 because of the persistence and hard work of patient advocates from the Immune Deficiency Foundation and the Alpha1 Association.

Working together, patients, families, providers and representatives from industry can influence the policy issues affecting quality of care.

Learn more about Standards of Care legislation by reading the “Key Issues Dialogue: Standards of Care Legislation.”

This dialogue features Minnesota resident, Kathy Antilla,Director of volunteer programs at the Immune Deficiency Foundation, who leads advocacy for this legislation in her state. Also featured are Ann Rogers, Executive Director of the Delaware Valley Chapter of the National Hemophilia Foundation (NHF), Val Bias, Executive Director of NHF and Jerry Powell, M.D., Director of the hemophilia treatment center at the University of California, Davis Medical Center.

Volunteers from the Minnesota immune deficient, alpha1 and bleeding disorders communities pose with the Minnesota bill sponsors, Senator Kathy Sheran and Representative Kim Norton at the 2008 legislative day in support of Minnesota Standards of Care