The Impact of Obamacare on Rare Disease Patients

DATE: April 7, 2010
Participants:
Ed: PSN Moderator
Diane Dorman: Lobbyist for Rare Disease Patients

Ed Moderator: Hi Diane. Welcome
Diane Dorman: Glad to be here.

Ed Moderator: let's go ahead and get started...We're here with Diane Dorman...we appreciate you taking time from your schedule which I imagine is quite busy these days.
Diane Dorman: glad to be here, very busy but fulfilling
Ed Moderator: Diane, lets start off with a bit of an introduction. Please tell us about the kind of work you do in Washington DC.
Diane Dorman: I am a lobbyist (bad word) and I represent the 30 million Americans with the 7000 known rare diseases. I also serve as a liaison to industry and federal agencies
Ed Moderator: How long in this role?#
Diane Dorman: 10 yrs
Ed Moderator: So in your role, you find out what is needed in the rare disease community and you go Capitol Hill to lobby for funds and focus?
Diane Dorman: Indeed. Most organizations are focused on their disease. I deal with larger, more global issues.
Ed Moderator: Discuss some of those specific issues.
Diane Dorman: Social Security compassionate allowances, issues related to the development of orphan products at the FDA, encourage increased research at the NIH, incentives for industry to encourage the development of orphan drugs and other therapies including humanitarian use devices
Ed Moderator: Could you define or explain humanitarian use devices?
Diane Dorman: These are medical devices that are used less than 4000 times per year. Very specialized for very small populations.
Ed Moderator: interesting...could you give an example of one?#
Diane Dorman: Of course. Dr. Robert Campbell developed the titanium rib for those children born with no, or limited ribs or extreme scoliosis. The device can be adjusted as the child grows. Took 14 years to find a manufacturer.
Ed Moderator: Amazing.
Diane Dorman: Truly is and the results are miraculous.
Ed Moderator: So talk a bit about the positive changes, accomplishments over the past 10 years in terms of awareness and goals achieved in the rare disease community goals that were help realized by such lobbying of Congress.
Diane Dorman: My goodness. Well, NORD has developed a very close relationship with the NIH and the FDA.
We worked collaboratively with SSA on the compassionate allowances initiative. In 2001 NORD worked with Hatch and Kennedy to introduce the Rare Diseases Act, which codified the Office of Rare Diseases at the NIH and authorized increased funding for the orphan products research grants program (still working on that)
Ed Moderator: Wonderful. And I know that you played a role in these initiatives.
Diane Dorman: Just a bit
Ed Moderator: It must be gratifying when such provisions get passed.
Diane Dorman: It truly is. Every day when I meet people affected by rare diseases, I realize why I do what I do. Helping those who sometimes cannot help themselves.
Ed Moderator: Well, our focus tonight is on the new healthcare reform bill. Now I know how huge it is and I'm sure not even some of the legislators themselves know all what's in it. but I'd like to do a short Q and A with some general questions--some of them asked to me from patients or caregivers in my group who want to know the impact of the new reform on them specifically as rare disease patients.
Diane Dorman: I want to add that my father died of a very rare brain disease. He was only the 97th case known world wide. We watched him die for 4 years. That's where I get my passion, my heart.
Ed Moderator: That is sweet. You know, I think that is such a trigger for people who become advocates. I know a few people who started an org because of a personal loss. I did so, because my Mom died from PAN.
Diane Dorman: Sorry to hear that. We all have personal stories. Overall, I think the legislation has a very positive impact for those with rare diseases. Eliminating pre-existing conditions, lifting annual and lifetime limits, etc.
Ed Moderator: okay, yes. So tell us a bit more in depth about the pre-existing conditions. Is this in effect now, or when does it kick in? In essence, the problem has been that insurance companies have turned down rare disease patients or won't cover their expenses, correct?
Diane Dorman: That is correct. The bill bars insurers from denying coverage to children who have preexisting conditions. For others that will come in 2014, I believe. Still trying to digest over 2000 pages
Ed Moderator: I know. Me too. And I've sent around the excellent materials you supplied. thanks
Diane Dorman: Hope the information was helpful. The devil will be in regulations and implementation. HHS is now trying to deal with some of those issues now.
Ed Moderator: In a sense, the bigger job now is to explain to the populace what's in those 2000 pages. So, to clarify, if right now there is a parent with a child who has a rare disease that child can now be covered by an existing insurance company, or if they switch to a new one..
Diane Dorman: That is correct. Specifically how to set up high risk pool until full implementation in 2014 and 2018
Ed Moderator: I think it's important that people do understand that a bit of this bill is not in place. I've heard of some patients expecting immediate changes.
Diane Dorman: I want to stress that there is still much to be accomplished. Although both bills are now law, it does not mean that things will not be fixed, changed, improved in the future.
Ed Moderator: Understood. And it's such a political landmine in so many ways. It's not going to be a fix-all and it won't be a fix-all next week.
Diane Dorman:
Unfortunately, that is what some heard - that everything would happen immediately. The government doesn't work that quickly. This is a massive undertaking that will take years to accomplish.
Landmine? You said it only too well. There are those who want to overturn the law based on constitution issues. States are suing. This is only the beginning.
Ed Moderator: What are some of the main issues? for rare disease patients when it comes to medical coverage and trying to get insurance, or being covered for the high cost of a rare illness., what have been traditional challenges for them?
Diane Dorman: The constitutional issues are probably going to be addressed. Although, I'm not sure if the supreme court will want to take it on. Can the government force people to buy insurance or be penalized. Big questions! I will be happy to send you our letters to the House, Senate and White House which outline the issues of most importance to NORD. Although there will be continuing issues, we looked at the greater good for all people. There will always be
Ed Moderator: Yes, please do, thanks. Challenge has always been....will this treatment be covered? Or, I suppose, some insurance companies can drop a high risk patient. Is that right?
Diane Dorman: There are opportunities to improve. The system is definitely not perfect. It is my understanding that insurers will no longer be able to deny coverage to high-risk patients.
Ed Moderator: I have gotten quite a few letters asking about Medicare or Medicaid. There are quite a number of patients getting these benefits and they are concerned that there will be cuts to these programs. What do you know about this issue?
Diane Dorman: I am still trying to understand the impact on Medicare and Medicaid, which is so important to many people. I can send you some information that I have received from colleagues outlining the changes in both programs.
Ed Moderator: again, thank you. that would be helpful. Well, I think the concerns are legitimate, but as you seem to be saying, it's too early yet to tell what impact it will have.
Diane Dorman: I really don't believe there will be cuts in either programs. There will be improvements, streamlining, elimination of fraud abuse, awards to docs who do the right thing. Yes, it is still too early to tell. As I said the devil will be in the details - how federal agencies interpret the language. So the work has just begun.
Ed Moderator: Okay, so the message right now is that cuts probably won't happen in Medicare or Medicaid, so for now at least patients shouldn't panic.
Diane Dorman: Patients should definitely not panic. If NORD were to identify a situation where we felt patients would be adversely affected, I can assure you we would voice our concerns ASAP.
Ed Moderator: Someone sent in a question...will I still be able to see my
rheumatologist I've been seeing or will I have a new doctor assigned to me? What say you?
Diane Dorman: As Obama has said, if you are happy with what you have, you can keep it. If that is not the case, I will need to know about it. Can't act unless we know if those circumstances.
Ed Moderator: And it's okay to say...I don't know. too early to tell. that's honest! :-) So essentially it is, if you are happy with your insurance now you can keep it
Diane Dorman: Honest, indeed. There are many positive changes required of the insurers. That is a start. This is just the beginning. If you are happy with your doctor there isn't a reason to fear losing him or her due to the new reform.
Diane Dorman: No.
Ed Moderator: How closely did NORD, NIH work with the congress and senate in the formation of this bill. Did you have direct talks or input to the bill? I mean, in terms of sharing the needs of the rare disease community
Diane Dorman: Should anything like that occur in the future, I have contacts within the White House and I can be quite adamant! NORD had direct conversations with members of Congress over the past 18 months. As federal agencies the NIH, VA, FDA and others can not take a position on any legislation I will add however that we did confer with certain people within those agencies to discuss their concerns.
Ed Moderator: Tell us about the talks you had. How were you received? Do you feel you were taken seriously? Do you see any direct reform in the new bill as being from NORD's input?
Diane Dorman: NORD played a key role in ensuring that that would not occur. Comparative effectiveness research - they will be required to call on an ad hoc committee when they consider a procedure for a rare disease or an orphan product. I might add that we worked hard to eliminate the 2-year waiting period for Medicare benefits, but the cost was too prohibitive.
Ed Moderator: when you say, "that would not occur", you're referring to....?
Diane Dorman: Originally, there was no mention of annual limits at all. Congress only focused on lifetime limits. They also did not recognize that grandfathering existing plans would do absolutely nothing to help patients. Does that make sense?
Ed Moderator: Sure. Someone who needs treatment for PAN now can't wait for three years for the law to go into effect.
Diane Dorman: For lifetime caps yes. Annual limits don't go into effect until 2014
Ed Moderator: One area of concern is how the new reform will affect research. In what ways will these new laws promote the research and treatment of new diseases and conversely, how could reform hinder or slow up research into new meds or treatments. I'm sure it will impact both ways. I meant rare diseases, not new.
Diane Dorman: On page 1922 (I think) There is still much to figure out . I don't think it will stop developing new therapies. Without new therapies, they don't make money. It will develop new pathways for RD research and OD development. Good things will be happening
Ed Moderator: I read where some pharam companies are saying they may not have the funding to put into research because of the new tax laws. Can you explain if and how biomedical companies are being taxed? Want to make sure I have my facts right.
Diane Dorman: I have to say that industry has been saying that any restrictions will eliminate research - they've been saying that for more years than I can remember
Ed Moderator: you've heard this before then..:-)
Diane Dorman: Since 1984 with the passage of the Hatch-Waxman act.
Ed Moderator: So, do you believe more funding will be directed into helping
companies develop new therapies for more rare disease patients?#
Diane Dorman: I believe so. Already large pharma is looking at the RD space - Pfizer, GSK and many others. Orphan drugs have been personalized medicine and they are beginning to look at that space
Ed Moderator: maybe with extra funding coming from revenue in this reform, companies will be more inclined to focus on needed. but less profitable drugs or therapies, does that make sense?
Diane Dorman: That is our hope - along with regulatory changes within the NIH and FDA
Ed Moderator: I mean somewhere down the line a decision is made...is this therapy going to create enough profit for the monies that went into developing it
Diane Dorman: That is why NORD is looking at incentives to encourage the development of new therapies
Ed Moderator: Well, that's our takeaway from this talk. NORD is the game, in the mix letting the lawmakers know that we have a population of rare disease patients
Diane Dorman: I promise you, we are looking at new and innovative ways to encourage the development of orphan products. I will send you the summary of our Summit last May (remind me) tomorrow. It should clarify our mission
Ed Moderator: How in the world do you tackle reading something like this health bill. I mean I'm afraid it might break one of legs on my desk if I put it down too hard at 2000 pages. My only concern is that not even all the lawmakers know exactly what is in it
Diane Dorman: I'm still trying to digest it all. To be quite honest, I'm quite sure many of our lawmakers don't have a clue. Scary, isn't it?
Ed Moderator: When I heard someone on Capitol Hill say...and I don't know who it was... said...well, we'll have to wait until it's passed to find out what's in it..I felt a chill
Diane Dorman: You are quite correct. Like I said, the devil will be in the details
Ed Moderator: Ahhh, yes. The good old fine print. Well THANK YOUfor this chat, even if it was the two of us
Diane Dorman: It has been a pleasure. Thanks for having me.
Ed Moderator: trust me...ohhhhh, trust me...the lack of attendance doesn't mean a lack of concern or question about what's coming down the road
Diane Dorman: If people have additional questions, I may not have the answers, but I can always try to find them
Ed Moderator: We are really honored and thankful to have a voice on Capitol Hill. You speak for millions. Thank you Diane. We'll have another chat as this all unfolds.. You take care and have a great evening.
Diane Dorman: A pleasure. Good night. Look forward to future chats


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