The FactorNet

Spring 20161

The FactorNetSpring 2016 / Volume 27 Issue 1

Welcome 3

Statewide Education and Fun Weekend 4

World Hemophilia Day Celebration 5

Bleeding Disorders Awareness Month 5

Camp Warren Jyrch 6

Marcus McClure Family Camp 6

Springfield Lobby Day 7

Hemophilia Walk 7

Becoming an Informed Consumer 8-9

Product Pipeline 10

Clinical Trials 10-11

Registering Online 12

HepCure 13

Gene Therapy 13

Support BDAI 13

Patient Assistance Programs 14-15

Our Thanks 16-17

Stay Connected 18

Bleeding Disorders Alliance Illinois

210 S. DesPlaines St.Chicago, IL 60661-5500

Phone: (312) 427-1495Fax: (312) 427-1602

info@bdai.orgwww.bdai.org

On The Inside:

Welcoming All Bleeding Disorders

Did you know World Hemophilia Day is on April 17th? Join us as we celebrate on Friday, April 15th!

Find all the details on page 5

BDAI invites you to join us for our annual Statewide Education and Fun Weekend!

Who: Everyone within the bleeding disorders communityWhat: A weekend of educational sessions, networking,

and community buildingWhere: The Pere Marquette Marriott in Peoria, ILWhen: Saturday, April 9th to Sunday, April 10th

This year’s theme is “Welcoming All Bleeding Disorders”, so join us as we come together as a community to explore what it is like for those living with bleeding disorders in today’s world. With new products being introduced all the time, the treatments available are better than ever, but there are still obstacles present within the community. Provide valuable input as we explore these issues and their solutions. This weekend is a great opportunity for families within the bleeding disorders community to network and learn from one another, while also having fun at the same time. We believe that community members are one of the best support systems out there for each other, and this is a chance for you to experience this first hand as you meet others in a similar situation and bond while sharing new ideas and opinions. Check out page 4 to learn more about the breakout sessions we have planned for the adults, and to find information about the teen track and day care program for the children.

The Bleeding Disorders Alliance Illinois

Spring 2016 2

BDAI Resources The Bleeding Disorders Alliance Illinois exists to improve the quality of life for persons affected byhemophilia and other inherited bleeding disorders.

Mission:

Contact InformationBleeding Disorders Alliance Illinois (312) 427-1495 www.bdai.org

National Hemophilia Foundation (800) 424-2630 www.hemophilia.org

Hemophilia Federation of America (800) 230-9797 www.hemophiliafed.org

Resource InformationBleeding Disorders Legal Hotline (800) 520-6154

Centers for Disease Control & Prevention (800) 311-3435 www.cdc.gov

Committee of Ten Thousand (COTT) (800) 488-2688 www.cott1.org

LA Kelley Communications (978) 352-7657 www.kelleycom.com

Patient Services Incorporated (PSI) (800) 366-7741 www.uneedpsi.org

World Federation of Hemophilia (514) 875-7944 www.wfh.org

Board of Directors 2016

Daniel Contreras President Marianne Kelly Vice President Dan Ciolino Treasurer Paul Lee Secretary Margo Schwartz-Newton Kelli Kovak

StaffBob Robinson Executive Director brobinson@bdai.org

Lily Schwartz Associate Director lschwartz@bdai.org

Bea Carr Financial Director bcarr@bdai.org

Adam Hii Program Coordinator ahii@bdai.org

Cory Holzhauer Communications cholzhauer@bdai.org Coordinator

Jessica Head Communications jschwartz@bdai.org Consultant

ILLINOIS

Northwestern Center for Bleeding Disorders

676 North Saint Clair, Suite 850Chicago, IL 60611(312) 695-4041

Stroger Hospital of Cook CountyAdministration Building, Room 1151

1900 West Polk StreetDepartment of Pediatric Hematology

Chicago, IL 60612(312) 864-4167

Ann & Robert H. Lurie Children’s Hospital

225 E. Chicago, Box 30 Chicago, IL 60611 (312) 227-4090

Rush Hemophilia & Thrombophilia Center Rush University Medical Center

1653 W. Congress Pkwy Chicago, IL 60612 (312) 942-3034

Bleeding & Clotting Disorders Institute9128 N. Lindbergh

Peoria, IL 61615-1422(309) 692-5337

ILLINOIS SATELLITE HTCs

Bleeding & Clotting Disorders Institute Satellite HTCs

Phone: 309-692-5337

Arthur Family Health Center 525 N. Vine Street Arthur, IL 61911

Frances Nelson Health Center819 Bloomington RoadChampaign, IL 61820

Crossing Healthcare320 E. Central Ave.Decatur, IL 62521

Medical Arts Building Visiting Physician Office

527 E. Grant StreetMacomb, IL 61455

Pediatric Specialty Clinic612 35th Ave

Moline, IL 61265

Fox River Cancer Center1200 Starfire DriveOttawa, IL 61350

Crusader’s Clinic on Broadway1100 Broadway Road

Rockford, IL 61104-1429

SIU School of Medicine Cancer Institute415 North 9th Street

PO Box 19678Springfield, IL 62794

Rush Satellite HTCs Phone: 312-942-8114 Fax: (312) 942-8975

Oak Park Medical Center610 S. Maple, Suite 3440

Oak Park, IL 60611

INDIANA

Indiana Hemophilia & Thrombosis Center

8326 Naab Road Indianapolis, IN 46260

(317) 871-0000 ext. 236

MISSOURI

The John Bouhasin Center for Children with Bleeding DisordersSaint Louis Univ. Dept of Pediatrics

Missouri/Illinois Regional Hemophilia Center 1465 South Grand Blvd.

St. Louis, MO 63104 (314) 577-5332

Saint Louis University Center for Bleeding & Thrombotic Disorder Hemophilia Treatment Center,

Adult ProgramSt. Louis University Hospital, West Pavilion Cancer Center

3635 Vista Avenue at Grand Blvd. 3rd Floor HemOnc

St. Louis, MO 63110-0250 (314) 577-6178

IOWA

Iowa Regional Hemophilia CenterDepartment of Pediatrics

2507 JCPIowa City, Iowa 52242

(319) 356-4277

WISCONSIN

University of Wisconsin Hospital and Clinics Comprehensive

Program for Bleeding Disorders 5105 University Avenue

Madison, WI 53705 (608) 890-9495

Comprehensive Center for Bleeding Disorders

The Blood Center of WisconsinP.O. Box 2178

Milwaukee, WI 53201-2178(414) 257-2424

About This PublicationThe FactorNet Newsletter is the official newsletter of

the Bleeding Disorders Alliance Illinois. It is produced free of charge to the bleeding disorders community.

The Bleeding Disorders Alliance Illinois does not endorse the products or services of its sponsors or advertisers. Any communications presented in this

newsletter is strictly information only. The decision as to what to do with the communication is the

responsibility of the individual recipient. The recipients are advised to check with their personal physicians as to their own physical status before testing any product

or using any information presented.

Rush Copley2020 Ogden Avenue, Suite 400

Fox Valley, Aurora, IL

Ashton Medical Group1800 McDonough Road, Suite 203

Hoffman Estates, IL 60192

Hematology/Oncology Associates2420 Glenwood Avenue

Joliet, IL 60435

Lurie’s Children’s Hospital Satellite HTCs312-227-4814

Northwest Community Hospital Busse Center for Specialty Medicine

880 W. Central Road, Suite 6400Arlington Heights, IL 60005

2301 Enterprise DriveWestchester, IL 60154

Outpatient Center in Lake ForestNorthwestern Lake Forest Hospital Campus

Lake Forest, IL 60045

The FactorNet

Spring 20163

Welcome! A community is a social, religious, occupational, or other group that shares common characteristics or interests and perceives itself as distinct from the larger society. Many people would not initially think that rare and isolating chronic disorders, such as bleeding disorders, would actually make them part of a collective and caring community, but it does. I guess we’re just weird that way – but I’m glad to be part of this unique group of wonderful human beings. Many of you give in so many ways – by volunteering, attending meetings, sharing your interests and stories face-to-face and on social media, and by fundraising for our programs and services. Such dynamic involvement is the reason why this community is thriving and growing, but are there new ways to be involved or things we have not thought of at BDAI? We are going to explore this throughout 2016 by conducting a needs assessment. A needs assessment is a process used by organizations to determine priorities, make organizational improvements, and allocate resources. It determines the needs, or gaps, by examining where the organization envisions itself in the future and the organization’s current state. This means something to me, but it may not necessarily speak to you. So, let me explain what we really want. We want to spend time during 2016 finding out what you – people with bleeding disorders, family members and caregivers – think being a part of the bleeding disorder community means. What are the benefits? What are the rewards? Are there any pitfalls or problems? And, most importantly, what should we be doing to keep this community strong for years to come? There will be multiple opportunities throughout the year to participate in this needs assessment as we start bringing it to our meetings and events. I ask you to start thinking now about the changes you would like to see within the community and when asked formally in the future, please share your thoughts. I promise that your voices will be heard, and through your participation you will begin to see changes within the community. The first place you will see the assessment is at the Annual Statewide Education & Fun Weekend. It is being held in Peoria this year on April 9th – 10th. We are excited about the theme – Welcoming All Bleeding Disorders - and the diverse agenda we have put together. I hope as many of you as possible are able to join us. One of the things we will be doing at this event is holding a special activity to recognize World Hemophilia Day, which is April 17. Supported by the World Federation of Hemophilia (WFH), hemophilia awareness day was first established in 1989. The 17th of April was chosen to honor Frank Schnabel the founder of WFH, whose birthday falls on the same date. Unfortunately, there are still many people in the world who receive inadequate treatment or no treatment at all for hemophilia and other bleeding disorders. According to the World Federation of Hemophilia, about 1 in every 1000 person has a bleeding disorder, many of them untreated. The aim of World Hemophilia Day is to raise awareness about hemophilia and increase the availability of treatments for this condition around the world. We will also be holding another event the following weekend in downtown Chicago where we will light the Wrigley building red for this awareness day. While reflecting on bleeding disorders worldwide, it is important to recognize the medical advancements being made in treatments for bleeding disorders, and also in Hepatitis C treatments which is another issue that affects some of our community members. In this newsletter we will be highlighting the product pipeline which lays out new treatment options, as well as HepCure which is an app aiming to raise awareness and educate users and medical professionals on the new Hepatitis C treatments available. We are kicking off fundraising for the year with the Gangster Gala on March 12th. In addition to the regular activities – silent & live auction, raffle, dinner, dancing and just a lot of fun – this 1930’s themed event will include a vault filled with prizes from our Mob’s latest heist. In advance, I would like to thank everyone who is working so hard to put together this fun night. Our goal is to raise $75,000 from this event. The dollars raised at the Gala, the Golf Outing, the Walk, and the Wine Dinner & Auction are what we use to provide the programs and services you have become accustomed to BDAI providing throughout the years. Check out the list on page 5 to see some examples of how your contributions allow us to deliver our mission each day.

(continued on page 5)

The Bleeding Disorders Alliance Illinois

Spring 2016 4

Welcoming All Bleeding Disorders There are a lot of great speakers and discussions planned for this year’s Statewide Weekend and we hope you will be a part of this community gathering. To give you an idea of what to look forward to, here are some of the break out sessions planned:

Community Input: Saturday morning will begin with a presentation on the importance of community collaboration. This year BDAI will be conducting a needs assessment to find out how to better serve you, so please give us your input! Help us to make the changes you would like to see within BDAI by voicing your thoughts and opinions.

Product Pipeline: Next, the focus will be on the new products that are available, or will be available in the near future, for the treatment of bleeding disorders. With an ever changing market, it is important to understand what options are available and how they differ from one another. Part of being an informed consumer is understanding why your medication is the best fit for you and your lifestyle.

In the afternoon, there are a variety of breakout sessions planned so you can choose the topic you find most interesting.

Relationships: There will be separate sessions for our Blood Brotherhood and Blood Sisterhood groups, both on the topic of relationships. The discussion will focus on various techniques for promoting healthy and positive communications within the relationships in your life. Having a child or spouse with a bleeding disorder can be a difficult element for a family to balance. Let us help you open the channels of communication and cut down on your stress levels.

Physical Health: Keeping in shape by both eating healthy and exercising is important for everyone, but especially for those living with a bleeding disorder. Exercise strengthens muscles and helps to prevent bleeds and joint damage. Participate in a discussion on fun and safe ways to stay active.

Stress Management: Between work, family, and busy schedules, life can be stressful. This session will help you find positive outlets for this stress and practice positive mental health. Mental health can be just as important as physical health, so take the opportunity for self assessment and to find ways of maintaining a positive attitude.

Walk Fundraising: Being a part of the BDAI community means not only attending events, but also taking an active role in the future of the organization. The Hemophilia Walk is your opportunity to get involved and support BDAI by being an active fundraiser. Join in the discussion of fundraising techniques and exploring ways of making this year’s Walk the most successful one yet.

Teen Track: Don’t worry teens, we didn’t forget about you! We have a special track for those ages 12-17 involving fun and interactive breakout sessions. We will focus on learning about fitness, transitioning to becoming young adults, and having a session about everyone’s favorite time of year, Camp! These sessions will take place at the same time as the adult track, so the teens will rejoin their families before dinner.

Day Care: For those ages 3-11 we will be offering a day care service so that your child can have fun playing with the others and you can focus on getting the most information out of your statewide experience.

Registration is now open!

Visit our website at www.bdai.com to

download the application form. The deadline is

March 21st and space is limited so get the form filled out and mailed or faxed to us ASAP!

The FactorNet

Spring 20165

World Hemophilia Day Celebration

In addition to showing how your donations support the work BDAI does, we also want to highlight some of the services offered. For example, if you do not currently have a medical ID bracelet we may be able to help you get one. If you have questions about the services available to you, please contact us. In February more than 300 people gathered to take part in the National Hemophilia Foundation’s (NHF) Washington Days program. This advocacy event gives the bleeding disorders community the opportunity to tell our stories to nationally elected officials. During last year’s event nearly 240 elected officials were visited by bleeding disorder advocates. In addition to simply educating elected officials about bleeding disorders and treatment costs, we also discussed the importance of federal funding for our hemophilia treatment centers. Illinois will be hosting a similar activity in Springfield on April 12 & 13. It will continue the same mission of educating state representatives and senators about bleeding disorders and the issues closer to home, this time on a local level. If you are interested in taking part in the Springfield Day contact us at the office. It is a very empowering experience and one that is well worth your time. This is what is hot for now. You can always stay up on BDAI news and information through our website – www.bdai.org or on Facebook at www.facebook.com/bdaillinois or twitter at www.twitter.com/bdaillinois. As always thanks for everything you do,

Bob Robinson, Executive Director

Hot Button Welcome (continued)

On Friday, April 15th BDAI will be partnering with Biogen to celebrate World Hemophilia Day in downtown Chicago. Everyone in the community is invited to join us as we participate in team building activities for the whole family. We will also be lighting the Wrigley Building red to honor those living with bleeding disorders and to help raise awareness within the city of Chicago. The building will remain lit from Friday, April 15th to Monday, April 18th, so help to spread the word of the reason behind the lighting.

Bleeding Disorders Awareness Month Did you know that March is “Bleeding Disorders Awareness Month”? Thirty years ago President Ronald Reagan declared March to be “Hemophilia Awareness Month”, and we are proud that the name is now being expanded to encompass all bleeding disorders. This month is a time to celebrate the close-knit relationships you have found within the bleeding disorders community, as well as fostering new ones. This can be done by attending BDAI events and meeting new people while learning valuable skills. Check out the back of this newsletter for dates of our upcoming events, or visit our website at www.bdai.org for more information. March is also a time to spread the word about bleeding disorders to friends, family, coworkers, and anyone else who may not know much about them. In doing so, we would love for you to invite them to the Hemophilia Walk. Most of our events throughout the year are geared towards those living with bleeding disorders and their families, however the Walk is a time for you to reach out and encourage your own family and friends to participate. Everyone is welcome to come out and show their support for the community by creating teams, making donations, volunteering, and walking with us.

$50 - Medic Alert bracelet and service for one child for one year

$125 - One day at Camp Warren Jyrch for one child

$400 - Average COBRA premium for one month to keep a family insured

$1,000 - Provides Family Camp for one family with a child living with a bleeding disorder to learn about Camp

$2,500 - Provides students with with a Bradley Krueger scholarships

The Bleeding Disorders Alliance Illinois

Spring 2016 6

Calling All Campers! CWJ Mark your calendars for Camp Warren Jyrch 2016 and turn in your application to save your spot! Camp this year will be from Sunday, August 7th to Saturday, August 13th, and will continue to be at YMCA Camp Benson in Mount Carroll, IL. Applications are now available online at www.bdai.org/camp/. Spots fill up quickly for both campers and counselors, so if you are interested in attending fill out your application as soon as possible. Also, please note that medical forms are not due until a later date, so save your space by turning in your application. For those of you who may not be familiar with Camp Warren Jyrch, it is a week-long, overnight camp for children living with bleeding disorders. Camp has become an important tradition within the bleeding disorders community, and we are proud to be celebrating our 44th session of camp here in Illinois, making us the second oldest bleeding disorders camp in the country. This well established program provides a great chance for children to assert their independence and meet other children like them as well. Campers spend the week participating in fun and safe activities while learning to care for themselves. Camp activities include a climbing wall, archery, arts and crafts, swimming, and more. There are also medical personnel on staff who assist the children with infusions, as well as

helping to teach them self infusion which is a key step towards a child’s independence.

If you have any questions about Camp Warren Jyrch, please contact Lily at 312-427-1495.

In addition to Camp Warren Jyrch, BDAI also provides a Family Camp for campers ages 6 to 10 who are new to BDAI’s camping program. This is a weekend for families to come together and experience camp together. If you are nervous about sending your child away on their own for a week, Family Camp

gives you the opportunity to see what camp is like and learn some of the activities the children will be participating in, while also getting to know some of the CWJ Staff. And, if you have a child who is nervous about going away to camp for the first time, this is an opportunity for them to meet other first time campers and participate in fun activities which we hope will inspire them to come back and be a part of the full camp experience. McClure Family Camp will take place on June 3rd-5th at Timber Pointe Outdoor Center in Hudson, IL. Applications for family camp are available on our website at www.bdai.org and if you have any questions please feel free to contact us at 312-427-1495.

Family

Camp

Camper and Staff Applications are now available!Get yours in now to make sure you can be a part of the fun!

Don’t forget, medical forms can be turned in at a later date, but applications must be received in order to hold your spot.

The FactorNet

Spring 20167

Save the date for the 2016 Hemophilia Walk! Mark your calendars - this wonderful, once a year event is on

Saturday, September 17th. I know what you’re thinking, “That’s still months and months away!”. Wrong!

Well, partially wrong. The actual Walk is not until September, but the months leading up to the Walk are the most important time. This is when you can get your team together, begin collecting donations, host

fundraising events, and really get everyone excited for Walk Day.

2016 Hemophilia Walk

Walk CouncilOur goal as an organization

is to raise $75,000, but to do that we need YOUR help. What

is your personal fundraising goal? What is your team’s fundraising goal? And, most importantly, what can we do to help you

achieve that goal? The Walk is a community event. So, to get

more community input, this year we are creating a council which will meet in the months leading up to the Walk. The goal of this council will be to make sure we are doing all that we can to help

teams reach their fundraising potential, and to ensure that the Walk is represented in the way

the community wants. If you are interested in being a

part of this council, please contact Cory at 312-427-1495

or cholzhauer@bdai.org.

Website Registration The Walk website is now open for registration. Getting a start on fundraising early will make it even easier for you to reach your fundraising goal later. Visit the website below to get started:

Not sure how to register? Here is a quick breakdown of your options:Registering a Team - This option is for those who wish to be captains at the Walk. Captains serve as the link between BDAI and their team. They pass along information, encourage fundraising, and set team goals.Joining a Team - Team members are individual fundraisers who wish to fundraise as part of a larger team. They will still collect their own funds and ask for donations to their personal fundraising page, but their personal total will also go towards the team goal.Joining as an Individual - If you wish to attend the Hemophilia Walk without being part of a team this option is for you. Registering will get you a personal fundraising page where you can collect donations and share why this event is important to you.Donors - If you wish to make a donation to the Hemophilia Walk but you will not be attending, you do not need to register for this event. Use the “Make a Donation” button to make a donation on behalf of a specific person by searching for their name, or you can make a general donation to the Walk. Donating to an individual will help them to reach their individual fundraising goal as well as their team goal.

http://walk.hemophilia.org/Chicago

Join us at our Statewide Education and Fun Weekend in Peoria, IL on April 9th and 10th where we will be having a breakout session specifically dedicated to the Walk and community fundraising.

Springfield Lobby Day Join us Tuesday, April 12th through Wednesday, April 13th as we head to Springfield, IL to meet with our state government to discuss issues currently affecting the bleeding disorders community. We will be meeting on Tuesday night to first go over our platform and the issues we will be focusing on. Then, when meeting with the elected officials on Wednesday we will be able to show a united front and present them with a clear message stating the issues we recognize and the changes we would like to see to have them resolved. This program is open to everyone in the community and is a great opportunity to learn more about advocacy. One of the issues we will focus on is the budget crisis the state is currently facing and how it will impact reimbursement of publicly funded programs. We will also draw their attention to the Affordable Care Act and how the plans it offers are bringing about higher out of pocket costs for consumers. With bleeding disorders being one of the most expensive chronic diseases to treat in the United States, this is

an issue that is affecting many people within our community. Because of the price tag associated with treatment, Fail First Policies - where insurance companies require proof that a less expensive treatment option is not working before covering the preferred treatment - are becoming more common. Join us as we bring these issues to our state government and stand up for the bleeding disorders community.

The Bleeding Disorders Alliance Illinois

Spring 2016 8

Becoming an Informed Consumer The diagnosis of a bleeding disorder can create a uniquely complicated situation for patients and their family members. Most people have little or no understanding of these disorders and the treatment options available prior to diagnosis, so many find themselves scrambling to gather information so that they can make the best decisions possible. At all stages of diagnosis and treatment, gathering information is an important aspect of coping. This process is never easy and it becomes more difficult as the patient and their family has to deal with fears and worries associated with treatment and financial pressures. Being an “informed consumer” is one of the most important ways that a patient and their family can cope. Information about bleeding disorders is more readily available every day through books, newsletters, online articles, professional journals, newspapers, television, the internet, and from person to person interactions. The availability of so much information creates another “job” – one that is formidable and for which most people have little training. How does a person who has been diagnosed with a bleeding disorder discern what information may be useful and what to disregard? And why does it matter? This “job” is to decipher what information is helpful and true. There is as much misinformation about bleeding disorders available at your fingertips as there is well-documented and sound advice. While most bleeding disorders are manageable today, they can still be life-threatening, so the decisions you make about what to believe and which treatments to follow can affect your life’s outcome, as well as your financial status and emotional and physical well-being. You probably are like most patients in that you and your loved ones have a strong desire to strive for both the quantity and quality of life. The decisions you make regarding your treatment will affect both. These decisions will often be shaped by the information you have available to you. Therefore, it becomes important to look carefully at the sources of this information and how you and your loved ones process it.What are the different sources of good information and bad information?

These two lists are the same. How can you determine which information to trust? The first step in becoming an informed consumer is to develop a sense of what constitutes quality information. Quality information is based on reasonable data and seems logical based on everything else you know. What is reasonable data? Reasonable data is information that is obtained through a process called the scientific method. This process encompasses developing new theories, asking questions, testing multiple hypotheses that support or reject these theories, and drawing conclusions – all of which eventually leading to breakthroughs in understanding. The scientific method entails continually posing new questions, and then designing and carrying out new studies in an attempt to answer those questions. A single study cannot answer all the questions, and with each study having its own strengths and weaknesses, it takes time for reasonable data to accumulate. Plus, new studies modify old information, making the accumulation of reasonable data an ever changing process. While some may question the scientific method, it is the method by which the medical and scientific communities operate and it has brought about tremendous improvements in treatment, quality of life, and survival. It is not a perfect process, but well-designed and well-researched theories have led to advancements in bleeding disorders research and treatments, bringing us to where we are today.

Good Information Bad InformationInternet Pamphlets Internet PamphletsNewspapers Videos Newspapers VideosBooks Others who are affected Books Others who are affectedTelevision Family & friends Television Family & friends

The FactorNet

Spring 20169

Becoming an Informed Consumer (continued)

Patients frequently express their frustrations that studies have not been conducted on the issues that specifically affect them for which they are searching for answers. This frustration is compounded by the fact that one cannot draw a foolproof conclusion for themselves from the findings of larger studies. While these studies may indicate what will happen to the group as a whole, they do not speak to how one person might be affected. For example, if you experience an unusual side effect from a drug, it doesn’t matter that the studies suggested that such a side effect is unlikely; you are still left dealing with the consequences. So, it is important to remember that the information from studies can be helpful and relevant, but you still need to take your personal health into consideration when deciding upon your treatment choices. Anecdotal information Despite its many limitations, the scientific method is our best means of producing reliable conclusions that can be reproduced in multiple studies over time with the same results. The information gathered through the scientific method is information that patients can regard as tested and useful. It is important to distinguish information like this, which has been tried and tested over time, from information which is based on just one person’s personal experience. Personal information like this is called anecdotal information and it is not based on the scientific method. The scientific community uses anecdotal data in the very early stages of research when they are developing theories and hypotheses, but beyond that, anecdotal information usually has little or no value. It can be full of biases and filled with inaccurate information due to the fact that it is largely untested. Be cautious when dealing with information coming from the untested experiences of one or a few persons.Does the information make sense? How can you learn to evaluate information and claims? You can start by taking a hard look at the information to determine if it makes sense. Is the theory sound? Is it plausible? Think it through carefully and always ask, “Does this sound too good to be true?” Chances are that if it sounds too good to be true, it probably is. Many products, vitamins, attitude adjustments, treatments, and so on imply results or make claims that really do not make sense. They offer solutions that are too simplistic and may be driven by profit or some other intent that has nothing to do with your well-being. For example, there are many books on the market today which are written by survivors of a whole range of diseases/disorders. These books contain anecdotal information – their stories of how they handled their disease. These books contain their diets, treatment regimes, and prescriptions for recovery. Many people write these books as a form of therapy – as their way of dealing with or finding meaning in their disease. It is fine to read these books and find comfort and reassurance in the testimony of someone who is surviving. Personal stories can provide hope, which we all need to nurture, but you should draw the line when it comes to heeding their recommendations. It is important to be able to sift through which information is valuable for you and which is not. This is especially important when reading testimonies with claims for cures. Before considering anecdotal information, be sure you read the disclaimers and then ask yourself: • Does this make sense? • Why is this person writing this account? • Did this person have an actual diagnosis confirmed by proper testing? • What are the author’s credentials? • What claims does this individual make about his or her particular treatment?Processing information The ways in which you process the information presented to you can be as important as the information itself. Two different people often draw very different conclusions from the same piece of data. Our particular biases and belief systems affect the ways in which we process or filter information. One of the filters that most patients have is their desire to get well. This desire, while life preserving, can also leave patients and family members vulnerable. It is natural to search for ways to have control over the situation, which often can feel really out of control, however be sure to evaluate how your option will affect yourself and others before taking action. (continued on page 13)

The Bleeding Disorders Alliance Illinois

Spring 2016 10

Product Pipeline With medical advancements constantly being made, treatment options are growing for those living with bleeding disorders. The term “product pipeline” refers to the clotting factor concentrates in development at the moment. There are different levels in the approval process for these products and the pipeline is intended to keep everyone informed of their progress through the stages of preclinical work, clinical trials, and regulatory approval. As of December 2015, there were 31 new therapies either in development or starting to be marketed. This includes twelve for hemophilia A and VWD, eight for hemophilia B (including gene therapy), eight bypassing therapies to treat patients with inhibitors, and three others. While some of these are still at the preclinical stage, when the research is conducted on mice and other animals, many are in, or have completed Phase III trials with patients, the final stage before an application is made to market the product commercially. Extended half-life products One of the biggest developments currently taking place is the development of extended half-life products. This could be the first major improvement for hemophilia treatment since the creation of virally safe concentrates and prophylaxis in the late 1980s. All claims are still being tested, but it is possible that the half-life of factor IX could be extended three- to five-fold, factor VIII one and a half times and recombinant factor VIIa eight-fold. Currently, prophylaxis aims to maintain at least a 1% factor level at all times. With the creation of extended half life products, should physicians and patients be content with 1% or is a higher factor level desirable to prevent bleeding?More potent products/Novel mechanisms of actionSome of the products in development are designed to be more potent and more effective in stopping bleeding or to be based on entirely novel mechanisms of action. This is critically important for patients with inhibitors, for whom current treatments are not nearly as effective as conventional treatments for patients with hemophilia A and B without inhibitors.Broader portfolios for companiesAnother upcoming change that will occur due to the growing product pipeline is the development of broader portfolios for companies so that they can market products in all three major areas—hemophilia A, hemophilia B and inhibitors—and not just one or two, as is the case today. This has the potential to increase world supply and competition. In a worldwide market growing by 8% a year, this is a good thing.

While not all of these products will make it to market, many are very promising.

Did You Know?In pharmacology, the term mechanism of action (MOA)

refers to the specific biochemical interaction through which a drug

substance produces its pharmacological effect. A

mechanism of action usually includes mention of the specific molecular targets to which the drug binds, such as an enzyme

or receptor.

Clinical Trials As we stated with the Product Pipeline, there are many new treatment options being introduced for those living with bleeding disorders, but before these treatments become available they must pass through a series of clinical trials. In fact, research and development often depends on the willingness of volunteers to take part in clinical trials. Clinical trials test new ways to prevent, diagnose, or treat various diseases and conditions. This testing shows whether a treatment is safe and effective before it is made available for widespread use. There are more than 200 clinical trials involving hemophilia and more than 50 involving von Willebrand disease (VWD) underway worldwide, according to the National Institutes of Health’s ClinicalTrials.gov Web site. Because the bleeding disorders community is so small, it will take many people to volunteer to participate in clinical trials for the entire community to gain more knowledge and develop new treatments. Those with hemophilia and other bleeding disorders should expect state-of-the-art care from their providers but that care is really only as good as the evidence that supports it, and the way we get that evidence is through clinical trials. If you think clinical trials are inconvenient, unsafe or not confidential enough, that is not always the case. Clinical trials can be simple, easy, and beneficial.

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Preclinical Study Research conducted in the lab, sometimes with animals, to test the safety and efficacy of a new drug

Phase I Trial A study conducted with a small number of humans to test whether a new drug is safe

Phase II Trial A study conducted with a small number of humans to test whether a new drug is safe and effective

Phase III Trial A study conducted with a larger number of humans to test whether a new drug is safe and effective, usually in comparison to existing drugs

Phase IV Trial A study conducted after a drug has been made commercially marketed to confirm the results of the clinical trials

Preclinical trials can offer researchers a lot of good information, but studying how a treatment or procedure works in humans is different than studying mice or certain cells in a lab. Clinical trials are needed to answer the two major questions: 1. Does the treatment work better than what is currently being used? 2. Is the treatment or procedure safe for people to use?Why Clinical Trials Are Important Clinical trials are an important step in discovering new treatments as well as new ways to detect, diagnose, and reduce the risk of disease. They also help researchers and doctors decide if the side effects of a new treatment are acceptable when weighed against the benefits. Researchers don’t know what the results of clinical trials will be, and it is this uncertainty that can make it hard to decide if you want to participate in clinical trial. In rare cases, clinical trial volunteers have been hurt by the treatment or procedure being tested, but at the same time, hundreds of thousands of people have been helped and are alive because of previous clinical trials that resulted in new, more effective treatments. While clinical trials are important, the choice to participate in one is very personal and depends on your unique situation. How Clinical Trials Are Conducted Clinical trials are usually conducted at universities, hospitals, or clinics. Most studies are available at local community hospitals unless special equipment or training is necessary. For patient safety and for reliability of the study, there is a rigorous process involved where the researcher must lay out a detailed plan of how the study will be conducted. This plan is known as a protocol, and after the protocol is created, it is reviewed by a board who considers the ethics of the study, the safety of those involved, and the treatment of participants while they are involved in the study. The participant’s responsibility is to give “informed consent” before joining a clinical trial. Informed consent requires researchers to explain the clinical trial’s protocol to the potential participants and states they understand the material presented to them and agree to participate. The researchers must also disclose any possible risks and benefits of participating in the trial.

What Are Clinical Trials? A clinical trial is a research project that looks at how well a new treatment or medical procedure works in people. Other studies that are done on cells in petri dishes or on animals are called preclinical trials. Clinical trials are started only after preclinical trials suggest that the new treatment or procedure will help people and will also be safe for people. On the right are the phases of trials a product needs to go through before it is on the market for consumer use:

Clinical Trials (continued)

Participating in a Clinical Trial With so many medical advancements being made, it is your job to be an informed consumer and assess all options when figuring out what is the best plan for you in your situation. Each clinical trial has its own benefits and risks, so if you are interested in participating, discuss the study with your doctor and decide if it is right for you. Here are some potential benefits and risks to consider before participating:

Benefits: - Access to a treatment that isn't available yet - Regular and careful attention from some of the best doctors - Treatment that may be free or low cost - Contributing to research that may save lives in the future - The feeling that you're taking an active role in your care

Risks:- The new treatment may not work for you, even if it benefits other people in the trial- The treatment may not be as effective as your existing treatment- More severe side effects than current treatments- Your insurance company may not cover all the costs- More frequent testing and doctor visits.

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Transfer of information Today we communicate almost instantly with one another by telephone, cell phones, e-mail, and the Internet. A recent poll estimated that 60 million Americans use the Internet to search for health information, comprising two thirds of those using the Internet. The internet is one of the most unregulated forms of communication, and with no one controlling what appears on the Internet, it can be a source of unreliable or partially reliable information. Anyone with a little computer knowledge, some hardware, and a desire to “publish” can communicate whatever he or she wants to people across the world. The internet can also be a great source for information and communication, but check the validity of sources you are trusting, while keeping in mind that harmful information is just as easily available as helpful information. A recent article entitled, “Can You Trust Internet Health Sites?” put it very well by stating: “There is no question that the quality of health information on the Internet is variable. It’s uneven. Some of it’s extremely good and some of it’s extremely bad. Universities and patient advocacy groups put out material as a public service. Other sites promote questionable products and offer bad advice. The only way that the consumer is going to get reliable information is to find a Web site that has a person or institution or organization that they already have confidence in.” Just remember, as an informed consumer knowledge is the strongest tool you have, so hone it by ensuring sources are reliable before taking information as fact.

Being an Informed Consumer (continued) Register for BDAI Events Online

That’s it! You are now registered for a BDAI event! We here at BDAI are striving to make this process as easy as possible for you because it is very important for us to have an accurate account of who will be attending events. When people show up who have not RSVP’d, we run into the issue of lack of seating and resources. Also, when people RSVP and do not attend, it often results in a waste of time and money that could be used for future events. We understand life gets busy and plans change, but we ask that you be considerate and RSVP to events you wish to attend, and if plans change please call and let us know. Thank you for your understanding and we hope to see you at an event soon!

Registering to attend BDAI events is now easier than ever! Simply visit our website at www.bdai.org to

view upcoming events and to RSVP. Here are some step by step instructions:

Step 1:Go to

www.bdai.org and click on the

“Upcoming Events” button

Step 2:Find the event

you wish to attend and click the “RSVP Here”

button

Step 3:Now that you are on the EventBrite

site, click the “Register” button

Step 4:Fill in the

information for all those who will be attending the

event, including all children

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Support BDAI

Did you know you can easily donate to BDAI online? You can! Visit our website at www.bdai.org and click on the Donate button in the top right corner of the screen. You can also sign up to receive email updates about upcoming BDAI events. Just click on the “Subscribe” button to the right of the “Donate” button to stay informed about what’s happening in the community.

Support comes in all shapes and sizes, so even if you cannot make a donation we would still love for you to be an active participant in the BDAI community. You can do this by attending events, volunteering, and participating in our Community Blog. Another great way to stay involved and show your support is through fundraising. The Walk is our largest fundraiser of the year, and that is because of community members like yourself who are willing to fundraise year round and gain support for this event. Here are some ideas of how you can start today!

- Organize a yard sale

- Have a bake sale

- Collect loose change in a donation jar

- Ask for donations to your walk team in place of birthday gifts

- Offer to shovel a neighbors driveway for a donation

And anything else you can think of - creativity is key!

The Centers for Disease Control and Prevention estimate that more than half of all people living with hepatitis C in the U.S. are unaware of their infection and only 9% have been successfully treated and cured, despite the availability of well tolerated curative treatments for the past several years. As part of the plan to resolve this issue, the HepCure initiative was created. It aims to expand the number of health care providers treating HCV and improve provider and patient knowledge. HepCure is a free, innovative provider and patient education toolkit, which uses web-based and smartphone enabled resources, navigation, consultation, and support to enhance the quality of care for persons living with HCV. The HepCure patient is available for download on patients’ smartphones through Google Play and in the Apple App Store. The patient app assists patients in finding out what treatment options are available to them based on medical recommendations; assessing their own readiness to start treatment and accessing resources to improve readiness; providing medication reminders and tracking adherence and appointments; recording symptoms while on treatment; communicating with their health care provider regarding treatment adherence, symptoms and personal lab data; and accessing patient resources such as fact sheets.

HepCure Gene Therapy A team of researchers from the University of North Carolina and The Children’s Hospital of Philadelphia reported successful application of gene therapy in dogs with Factor VII deficiency. This is a great advancement that could signify eventual clinical trials for humans. Gene therapy is an experimental technique that uses genes inserted into a patient’s cells instead of using drugs or surgery to treat or prevent disease. This could be the next step in the treatment of hemophilia, eventually replacing the need for factor and infusions. The FVII deficient dogs tolerated the gene therapy infusions well. In other related studies on dogs with hemophilia B, similarly positive findings have translated well to the people with hemophilia B. The next step will be to conduct clinical gene therapy trials in humans who have been diagnosed with FVII deficiency. Medical advancements are being made daily and the future of the bleeding disorders community is bright!

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Patient Assistance Programs

As you’ve been reading on previous pages, we are living in an exciting time for medicine. New treatments are coming about to improve the lives of those living with bleeding disorders, but the remaining question is the affordability of these treatments. Whether you are staying on your current treatment or looking for something new, it is helpful to understand the Patient Assistance Programs offered by your factor provider. Some of these offer assistance with co-pays, free trials of a new medication, and more. Life with a bleeding disorder provides its own set of challenges, so there are programs in place to help ease the financial burden. Check out the chart below, which is also maintained* on BDAI’s website at www.bdai.org, to learn more. As always, BDAI is sharing this as information only, so please consult your physician with any questions regarding personal health decisions.

Company Program Limit Total

Insurance Requirements Eligibility Contact

InformationAdditional

InfoBaxalta Baxalta’s

Hemophilia CoPay Program

$12,000 Must have comercial insurance

Must use eligible Baxalta hemophilia products

888-229-8379

www.hematology support.com

Can be used for medication related copayment/coinsurance costs

Free Trial Program

4 to 8 doses

Not for those already using a Baxalta medication

www.hematology support.com

For new patients thinking about switching to a new product

Bayer Bayer Access Solutions

$12,000 Must have a private health insurance plan

For those with Hemophilia A

800-288-8374

www.kogenatefs.com

No income requirements; may be eligible for $0 Co-Pay Program

Biogen Co-Pay Program

$12,000 Must have private insurance

Avaliable to those using ALPROLIX or ELOCTATE

855-693-5628

Free Trial Plus Program

30 days free

For those who are uninsured or underinsured - Those with government healthcare coverage are not eligible

For those who are newly prescribed a Biogen product

ALPROLIX 855-692-5776ELOCTATE855-693-5628

May also be eligible to receive free for up to one year until healthcare coverage begins

Factor Access Program

For those experiencing a gap in their insurance coverage

May be eligible between jobs, changing insurers, or if you have reached your insurance cap or annual dollar limit

855-692-5776 Those who are eligible will receive free factor

CSL Behring

MyAccess Cost-Share Program

$12,000 For those with private insurance, federal or state programs not eligible

For those using a CSL Behring eligible product for treating von Willebrands or Hemophilia A

800-676-4266 No financial or age restrictions apply

CSL Behring Assurance

one month supply

For those experiencing a lapse in private insurance

Must be currently using an eligible CSL product

866-415-2164 Earn points towards a free month supply of factor

Patient Assistance Program

For those uninsured or underinsured

Must currently be using an eligible CSL product

844-727-2752

www.cslbehringus.com/patientser-vices/

Can help when product is not covered by insurance

*Chart on website is updated quarterly

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Company Program Limit Total

Insurance Requirements Eligibility Contact

InformationAdditional

InfoEmergent Biosolutions

IXINITY Savings Program

$12,000 Must have commercial insurance

For those with a valid prescription for IXINITY

855-494-6489

www.ixinity.com/save-on-ixinity

No monthly limits or income requirements & can be used retroactively for up to 12 months

Grifols Co-Pay Program

$20,000 Must have commercial insurance

Must have a prescription for ALPHANATE or AlphaNine

ALPHANATE877-255-4829AlphaNine877-264-1405

No income requirements and presciption is covered up to the patient’s annual out-of-pocket maxiumum

Free Trial Program

3 doses, up to 12,000 IU

For patients who have not previously had a prescription for ALPHANATE or AlphaNine SD

888-474-3657www.alphanate.com

www.alphaninesd.com

Novo Nordisk

NovoSecure

$12,000 Private insurance required for some benefits

Must be using a Novo Nordisk product for an approved diagnosis

844-668-6732www.mynovos-ecure.com

Program provides financial, insurance, and educational assistance as well as one-on-one access to a Novo Secure Specialist

Octapharma Co-Pay Assistance Program

$6,000-12,000

Must have commercial insurance

Must currently use a factor product from Octapharma

800-554-4440

www.wilateusa.com

May be applied to copayments, deductibles, and co-insurance costs

Wilate Bridge Program

5,000 IUs

For patients not currently using Wilate

877-794-5283

www.wilateusa.com

Those who are eligible will receive a free trial

Pfizer Pfizer Rx-Pathways

$10,000 For privately insured, uninsured, or those lacking coverage

Patients can check their eligibility online to see what services are available

877-744-5675

www.pfizerrxpath-ways.com

Provides copay assistance for privately insured and may provide free medicine for patients without insurance or lacking coverage

Pfizer Factor Savings Card

$12,000 Must have a prescription for Pfizer products

888-240-9040

www.hemophiliavil-lage.com

This card can be used to help cover copay, deductible, and coinsurance costs

Pfizer Trial Prescription Program

one month supply up to 20,000 IU

For those with commerical insurance

For newly prescribed patients

888-327-7787

www.hemophiliavil-lage.com

Those who are eligible will have factor delivered at no cost

Patient Assistance Programs (continued)

Currently, hemophilia therapy is among the most expensive in the world with a total annual costs per patient ranging from $60,000 to as much as $1,000,000 for some patients. This is why carefully selecting insurance coverage is so important. Patient assistance programs can help fill in

the gaps if you are between coverage and can help to lower out-of-pocket expenses.

Did You Know?

The Bleeding Disorders Alliance Illinois

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The Bleeding Disorders Alliance Illinois

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A Special Thanks to Our Sponsors:

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Check out our website to find out about upcoming events at: www.bdai.org

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or share your knowledge with the community. With your participation we can build a stronger community for those with

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Check out our Blog at: www.bdaiblog.blogspot.com

Parents

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Save the Date For These Upcoming Events

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For more information, log onto www.BDAI.org or call 312-427-1495.

Plus, now you can register online for many of these events by visiting www.bdai.org/events.

March Bleeding Disorders Awareness Month

March 19th Women’s Group Event on Self Care - Chicago, IL

March 24th Educational Event on Financial Assistance

April 9th-10th Statewide Education and Fun Weekend - Peoria, IL

April 13th Springfield Advocacy Days - Springfield, IL

April 15th World Hemophilia Day Event - Chicago, IL

April 17th World Hemophilia Day

April 21st Educational Event