2004N-0181 - Critical Path Initiative; Establishment of Docket
FDA Comment Number : EC1
Submitter : Dr. Albert Musella Date & Time: 06/10/2004 05:06:27
Organization : Musella Foundation For Brain Tumor Research & Info
Consumer Group
Category :
Issue Areas/Comments
Comment Issues
4. For each problem identified, if a solution would facilitate the development of drugs, biologics, and/or devices for a particular disease or categories of disease, please indicate which diseases would be affected?
I am talking about the treatment of malignant brain tumors.
2. Please rank each hurdle identified in Question 1, above, in priority order according to which hurdles create the most severe product development problems.
IF this payment problem were solved, it would make all of the other hurdles insignificant. Taking away the profit motive is the easiest way to shut down research.
1. Hurdle Identification. Please describe the product development issue, the nature of the evaluation tool that is out-of-date or absent, how this problem hinders product development, and how a solution would improve the product development process.
In my opinion, the major hurdle is the way new treatments are paid for by insurance companies, specifically Medicare. Many private insurance companies follow Medicare's lead.
This greatly affects the profit potential of a new treatment, especially for diseases that are not the most popular, which discourages research.
I run a brain tumor foundation, and have 2 specific examples:
1. Gliadel wafer. This is a biodegradeable wafer implanted into the brain at the time of a brain tumor surgery and it slowly releases chemotherapy. This went through years of extensive and expensive testing and was rewarded with FDA Approval. It was the first of it's kind treatment approved for brain tumors, and should have opened up the floodgates of research to make better versions of it. Unfortunately, Medicare refuses to pay for the cost of them. They claim it is coverted, but lump it into the DRG for brain tumor surgery. The hospitals lose over $12,000 every time they are used, and many hospitals now refuse to use them.
2. Gliasite radiation therapy system. This is an implantable balloon and catheter system which is implanted at the time of the brain tumor surgery and later filled with a radioactive liquid, then removed. They have the same problem. The cost of the baloon and the radioactive material is NOT reimbursed to the hospital, so the hospital loses a lot of money when it is used, which discourages their usage.
There is an open public comment period about these 2 issues now with CMS. Visit http://virtualtrials.com/activism.cfm for details.
My solution would be to create a requirement that once FDA approval is attained, Medicare has to pay for the treatment, in a manner that is fair to the hospitals and doctors, and not create undue pressure to not use the treatment. One way would be for medicare to reimburse seperately for this type of drug / treatment in addition to the normal payment for the surgery.
7. For each problem identified, what role should FDA play and what role should be played by others?
One other roadblock has been that Medicare is now trying to use the FDA's rulings to deny medications based on off-label use. The FDA approves medicines for specific indications, and it is relatively hard and expensive to get approval for additional indications. A solution would be that once a treatment is approved for any disease, getting approval for additional indications should have it's own seperate fast track: set a requirement of a
certain number of patients with each new proposed indication and a minimum accepted result. If a trial is done with at least the minimum number of patients and achieves at least the minimum result required, approval is granted.
Another related issue is the approval of new ways to deliver drugs. Gliadel wafer is the perfect example. It was approved using a low dose of BCNU as the chemotherapy that it releases. Early tests show a high dose of BCNU works better, but it won't be available for years, because large, formal trials have to be done, and without hospitals paying for the original form, that isn't going to happen. Once the basic new delivery system is approved with one drug, any other approved drugs should be allowed, perhaps with a small trial to show that it isn't dangerous.
3. For each problem identified, please indicate the type of drug, biologic, or device to which the hurdle applies.
See above
5. Nature of the Solution. For each problem identified, please describe the evaluation tool that would solve the problem and the work necessary to create and implement the tool/solution. For example, would a solution come from scientific research to
The solution is simple. The process of going from drug/device discovery / invention, through trials and to FDA approval is a risky, long and expensive road. It deserves to have a reward waiting at the end. Without a chance at a reward, the research resources will be directed elsewhere to products that have the potential to make money.
6. For each solution identified, please indicate which could be accomplished quickly, in less than 24 months, and which require a long-term approach?
This can happen quickly. The CMS has an open public comment period. Have the Director of the FDA and the President of the USA make a phone call to the head of CMS and tell them to find a way to fix the problem immediately for Gliadel and Gliasite, which would open up brain tumor research immediately.. then long term have CMS work on a solution that would ensure ALL FDA approved treatments get covered.
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