Thank you for the opportunity to comment on the guest post by the assistant general counsel of the PhRMA Jeffry Francer raising important issues of accountability. Francer’s post is compiled from the verbatim excerpts from his testimony at the FDA Transparency meeting; his full testimony can be foundat http://www.regulations.gov/fdmspublic/ContentViewer?objectId=09000064809e7abb&disposition=attachment&contentType=pdf on p 12-14.

In support of the PhRMA’s stand that “FDA should make it obvious, to those who are interested, how scientific data leads to its approval decisions”, I would like to suggest the following steps that could enhance transparency, benefit the industry, public and investors, and improve FDA’s image.

1. Accompany any FDA decision taken contrary to its advisory panel recommendation by timely releasing (within 1 month) the full FDA expert analysis and its internal documents explaining why such decision was taken.

2. For any label change, make the supporting internal analysis by the FDA experts publicly available via Drigs@FDA page in a timely manner (within 1 month).

3. For any public health advisory, make the supporting internal analysis by the FDA experts publicly available and linked directly from the advisory page in a timely manner (ideally, immediately).

4. Make public every “complete response letter” the same day as the company-sponsor receives it. ( “Complete response letter” is an official letter from the FDA to the company-sponsor of the drug requesting additional information, trials and explaining reasons for non-approval.) The practice of keeping such letters confidential gives unfair advantage to the insiders over the outside stockholders and creates significant potential for securities fraud.

5. Notify the sponsoring companies, public and investors about the reasons for the delays on applications filed under PDUFA and the estimated new date of their review completion. This can be done via a special PDUFA-updates web page. The reasons for the delay could be kept general to preserve commercial secrets, for example, “large volume of data requires additional time”, “toxicology analysis requires additional time”, “GMP submission requires additional documentation” etc.

The FDA should do more to communicate with the public and the clinical trial researchers and doctors in practice about the status of approval for life saving drugs. My father has mucusal melanoma and the melanoma has begun to spread. The cancer has already been treated with chemotherapy & radiation at premier cancer research institutions in the US. It is possible to continue with these treatments, but a new drug waiting for approval is PDL1.

There had been rumors that the drug was expected to be released this summer, but there is no word on when it will be released & there is no information on the FDA website as to where it is in the approval process.

My Dad just celebrated his 57th birthday yesterday. He has his first grandchild on the way and my sister-in-law is due on Christmas. Dad's cancer is very aggressive and I pray everyday that he will get to enjoy time with his grandchild and be there for when the time comes that I have a child.

The FDA should be more transparent with the approval process. The FDA should be accountable for delays just as any private organization works. My family runs a construction business and they have to be accountable for when a project is delayed or costs run over budget. The government should be accountable as well for when projects are delayed and transparent with the taxpayers who fund their role in our society.

As the FDA, you should be held responsible for getting lifesaving drugs into the public and providing documentation on where a drug is in the approval pipeline. If there is a hold up, there should be a verifiable & justified reason and no reason not to be transparent with the public. Otherwise, it just seems like you are trying to obscure your inefficiencies from the taxpayers.

Also, there are other potentially lifesaving drugs that my Dad could be enrolling in their clinical trials...if he had a different kind of melanoma. Because he has mucusal melanoma, no one will accept him into their clinical trials for the very reason that he would be at risk of skewing their precious data. I've taken statistics and marketing research classes - I know how you can skew results based on your "sample." I cannot believe how these clinical researchers can deny someone even a chance to respond to their treatment. How is that ethical & how can you allow this? There should be exceptions that allow researchers to enroll "special" cases where it is well documented that this person is held outside of their sample prior to the patient starting treatment, but for the purpose of giving them a shot again at life. Why are we doing all of this research if not to save people's lives?

Please, please open PDL1 up to the public or approve for the next phase of clinical trials or whatever needs to happen for it to be an available treatment. You can buy it online for lab testing on rats. How absurd is that? I can google PDL1 and find several sites to order from for rats, but it is not available as a drug to save Dad's life.

Anyway, I highly doubt you will take any action or even post my comment, but out of desperation for my fathers life, I have to reach out and hope that somehow a process will change and someone else will not have to go through this unbelievable heartache.

What reforms have been instituted to mitigate retaliatory actions against FDA employees?

I commend the FDA scientists; particularly the whistleblowers and authors of the August 31, 2006, memorandum identified in the Project for Government Oversight February 2009 report, as Appendix A, for courageously sounding the alarm on what appears to be a very serious problem in CDRH. They paid a significant price for reform in CDRH. Are there any efforts to restore their reputation?

Furthermore, talented scientists were driven out of CDRH, including the Center's former GLP expert, for investigating criminal activity (fraud and research misconduct) in applications. Their investigations raised serious questions about the scientific integrity of the FDA medical device approval process. Dr. Marcarelli, Mr. Timothy Ulatowski, and Dr. Donna Bea Tillman know who they are.

We look forward to return of these dedicated public servants. CDRH needs all of its talented employees to ensure that the medical devices Americans depend on are safe and effective.