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This is the biggest legislative problem affecting people with porphyria that we have encountered in the last 30 years. A new bill was introduced in Congress at the end of July that will impact access to orphan medicines, H.R 2674 - The 340B Improvement Act. This legislation will expand government price controls to a wide range of locations, regardless of need, in some instances forcing medicines to be sold for as low as a penny, regardless of the costs to innovation and patient access. If the bill passes, a number of treatments for rare diseases, including porphyria, may no longer be available.

While well-intentioned, such an expansion of price controls could be devastating for those people who now take Panhematin or who may need to take it in the future. I will explain why.

This bill forces drug companies sell treatments to approximately 5000 hospitals for artificially low prices, regardless of the costs to manufacture and the cost to distribute. In some instances, orphan medicines, such as Panhematin, will be sold for as little as one penny, regardless of the losses incurred to make it available. The new expansion to the 340B program also expands the program beyond geographic needs, basically allowing affiliate programs of a central qualifying hospital to purchase price controlled products, regardless of location or need. In other words, a “340B hospital” can purchase a rare disease drug for one penny and in turn and sell it to another non-qualified hospital for a large profit. To me, this is outrageous and puts those of us who use a rare disease treatment in terrible jeopardy

Under this new bill, 340B was expanded significantly to hospitals, rural and community clinics to include the addition of inpatient and outpatient services (1500 new hospitals, clinics, etc.). In the new bill, there are NO exemptions for rare disease drugs. As you can imagine, companies like the one that manufactures Panhematin, would be hard pressed to make this complex biologic and sell it for one penny. How could they or any other company that manufactures rare disease treatments continue to make those treatments available under such conditions?

Many rare disease treatments, like Panhematin, are life-saving. Patients can have attacks and lose their lives without this medicine. You may say, "This cannot be possible. Surely our Congressmen would not put us in such jeopardy?" But, it is true; some members of Congress have already signed on to support this legislation. Below are five ways you can help:

1. Contact your Congressman! Tell them we MUST have an exclusion for rare disease drugs included in H.R. 2674, the 340B Program Improvement Act.

2. If you cannot talk to your Representative personally, ask to speak with their Legislative Aid.

3. Write a letter to your Congressman. This is one of the most powerful things you can do. Write a handwritten letter (not an email) and tell them how important the “orphan drug” exclusion is and that it must be included in the 340B Program Improvement Act.

4. Contact your local newspaper and television station about this issue.

5. Ask your friends and family members to call and write their Congressman. The more voices that speak up, the stronger the message.

The APF cannot educate members of Congress on the unintended consequences of such legislation without your help. This legislation could create a life threatening situation that involves YOU. Even if you don't take such a treatment, your children may need it in the future, but it may NOT be available.

We must advise our Congressmen that expansions of 340B, no matter how well-intentioned, can have devastating consequences for patients who need orphan medicines. To find out who your Congressman is go to

To contact me, the APF office number, toll free, is: 866.APF.3635 or 713.266.9617. My email is: We want to hear about your successes spreading this important message. Call the office and let us know what you are doing to help. I look forward to speaking with you because together we can stop this bill from passing and save our future - the lives of our children.


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Happy Winter Season!

                  Happy Winter Season!