CF Vertex research and government shutdown

[Joe2007]

Does anyone know how the government shutdown will affect CF research or Vertex trials specifically? Given the orphan drug status and fed funds component of that program, could research trials be delayed or set back? Is the FDA less capable of working together with firms like Vertex during times where their staff is being impacted by the shut down? -Concerned CFer, double F508

 

[bcl0328]

they will still continue to do studies since it will take 6 months to gather their data. the government will be back up long before then.

 

[Joe2007]

Let's hope.

 

[Gammaw]

What component of CF research is backed by federal funds? I'm not aware of any....
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[Joe2007]

NIH, through federal funds received through the department of health and human services, provided grants of federal funds for 1) $721,362 U of NC at Chapel Hill, The Role of Anaerobic Bacterial Infection in Cystic Fibrosis. 2) $354,210 delta F508 CFTR Trafficking Regulation by 4-Phenylbutyrate Childrens Hospital of Philadelphia 3) $362,588 CFTR Biogenesis and Function in Epithelia, University of Alabama at Birmingham. .........I could keep going.....

This is my basis for inquiring whether the government shutdown and its mass shuttering of medical research may impair CF research.

Also, the FDA approval process is not merely a "do your study and give us a ring when you are ready to submit for approval"-type process. During an investigational process the FDA makes comments, gives advice on things like the adequacy of the technical data, reviews progress reports from the sponsor. The FDA recently put a hold on the Vertex Hepatitis study during the investigation that the FDA oversees due to liver complications.

Therefore, adequate funding of the FDA during the phase III investigational period is a concern prior to the NDA submission.

Thus the basis of my question about the government shutdown's impact on FDA workload in collaboration with companies such as Vertex which are going through clinical trials.

If anyone can put my concerns to rest that would be great. Thank you!

 

[Gammaw]

The NIH Clinical Center is open and continues to function during the partial government shutdown. Patients currently enrolled in a protocol at the Clinical Center are able to continue their participation.
However, at this time, the Clinical Center is not able to admit NEW patients -- unless deemed medically necessary by the Clinical Center Director -- if they are not already enrolled in a protocol. Please consult with your research team.


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[Gammaw]

See the above post. However, I'm unaware of any clinical trials involving cystic fibrosis underway at the NIH. I would not imagine clinical trials or research already underway outside the NIH would be immediately impacted. I dont believe Vertex receives any NIH funding. I suppose it might have problems if the shutdown lasts months or years . . . Since it might be difficult to get approval for new drugs or clinical trial progression if no one is working . ...
Having said that, I dont think anyone can know the answer at this point . . Especially since, at the risk of tweaking someone's political cheek, I suppose our President might find it useful to continue indefinitely to refuse to negotiate living within our national income limits so he can blame it on the opposing political party and attempt to gain an advantage for his party in the upcoming elections. . . He might keep it up until then! But fear not, after another election, he will have less motivation to continue the divisiveness. Ok. I will back off now.
If there is a particular program that gives you concern, I bet you could call them and outright ask if there is any impact. I believe Vertex in particular is very accessible and responsive.
I would also love to know where you are plugging into the data for federal funding. Thanks!

 

[Aboveallislove]

FDA can also continue to review and prove drugs, as it is funded by user fees. Google it and it eains. It can't accept new fees and it might have some slow down...it is within FDA to decide how fast they move versus how much shut down affects and well could be political reasons for the FDA handling discretion to act.

 

[Joe2007]

TO: GAMMAW
So you are saying that you do not think that current CF trials currently underway would be affected. But you posted a quote that specifically says that the only new enrollees for existing trials would be patients for whom it is imminently "medically necessary". Anyone who has ever done a CF study knows that there are multiple phases of enrollment periods and that the CF patients/enrollees must be stable and meet other criteria to qualify. So why would this NIH announcement lead you to conclude that ongoing studies, many still enrolling, would not be impacted?

The CF Foundation is also involved very closely with NIH through the CFF/NIH Unfunded Grant program. One of the main missions of this collaboration is to continuously and vigorously encourage NIH to assume support of meritorious CF related projects and to ensure that the momentum of CF research is not slowed.

So I just do feel as relaxed as you seem to be.

Furthermore, I intended that this thread remain specifically about the threats to lifesaving CF clinical trials for all of us who are hoping so greatly that CFers have a brighter future. But since you introduced some politics into the thread, I feel compelled to respond.

The shutdown does not have anything to do with the budget. It is 100% about tying a provision to delay and dismantle the ACA in exchange for a continuing resolution to keep the government open under current funding amounts. This is ironic because the ACA is funded by mandatory funding not appropriations of a passed budget. It is further ironic because the CBO, a well respected independent body of economists, statisticians and finance experts, have forecasted that the ACA actually shrinks the budget deficit.

The president did not refuse to negotiate the budget. Budget negotiations are done between the House and Senate. Usually, in our democratic country, the senate and house form conference committees for this purpose. Since April the Senate has attempted to have these committees appointed so that budget negotiations could begin. On all 19 occasions Republicans blocked the appointment of these committees. Meanwhile, the Republicans made sure to vote 40 times to repeal the ACA.

The facts make it pretty clear that the extreme faction of the Republican party had only one agenda, undermine the ACA. Their eleventh hour attempt at budget negotiations was only used as a false pretense to shroud their true motive. I am sorry but I respectfully must disagree with you.

It is not one particular CF study I am concerned about. It is the entire lack of regard for all those with chronic illnesses who count on the hard tireless work of medical researchers, who with federal gov funding, strive to make our futures brighter.

 

[Gammaw]

Hi Joe 2007. I don't know what happened to my responsive post from a couple of days ago, but I will try again. My intent was not to offend you. I should have left politics to private conversations! Your initial post stated you were particularly concerned with Vertex. As far as I can discern, Vertex is not running any trial at the NIH Clinical Center, the research Facility for the NIH. The NIH bulletin from their site, which I copied and inserted as a post, only indicated that trials at the NIH Center might not be able to recruit new participants until the shutdown ended. This does not seem to apply to third party researchers - such as Vertex. I am also not aware of any trials funded by the NIH directly involving cystic fibrosis. Having said that, all research benefits all research. You never know what will provide a breakthrough. But it really WOULD be nice if the Feds could help fund some of the research so vital to CF. Perhaps they will get on the bandwagon soon since there have been breakthrough discoveries and technologies that have such a wide implication for genetic diseases in general.

As for the deficit and it's relationship to the health care insurance issues that are so critical to us all - let me just say that in my house, a deficit is when the amount of money I need to pay on our debts exceeds the amount of money coming in. When that happens, we have two choices - decrease our debt or increase our income. Either way, if we manage to do either one, the deficit will certainly decrease, since my SHORTFALL (read "deficit") between income and expenses has decreased. But if I choose to increase my income (I.e. read "increase taxes") without reducing my debt, despite decreasing the shortfall (deficit) my debt load is actually higher. The Feds have chosen to increase income, rather than decrease debt. I don't much like the idea that the average American family of four has to see a $7400 a year increase in health care expenses to increase the Feds income which mathematically decreases the deficit. On top of that, if my spouse goes out and tries to incur another large debt, I can either put my foot down and say no, since we don't have the income to make the payments, or I can increase my tolerance for unmanageable debt - i.e. increase our debt ceiling - and not say anything, just smile sweetly. There is not an untenable connection. No one, not any one no matter how high up, should refuse to negotiate any of the possible debt solutions on the table. That's my take on it. And it's okay that we disagree, isn't it?

 

[Joe2007]

I appreciate your response. I do not know where you got your $7400 figure from given that ACA related taxes only effected households with adjusted gross income of $250k a yr or more and given that health insurance premiums are growing at a slower rate than in the last 50 yrs. i"ll try to figure out how to private message you so we can continue our dialogue. Thanks.