FDA Notice on PDUFA

Attached is a notice from the FDA on Prescription Drug User Fee Act. It seeks to set up public meetings for 20 different disease areas. The candidates are:

Pulmonary arterial hypertension. 
• Heart failure.
• Primary glomerular diseases.
• Narcolepsy.
• Huntington’s Disease. 
• Depression.
• Autism.
• Peripheral neuropathy. 
• Fibromyalgia.
• Obesity.
• Nocturia.
• Chronic fatigue syndrome.
• Irritable bowel syndrome.
• Inflammatory bowel disease.
• Alopecia areata.
• Diabetic ulcers.
• Female sexual dysfunction.
• Interstitial cystitis/painful bladder syndrome.
• Fracture healing.
• Diabetic foot infections.
• Hepatitis C.
• HIV.
• Patients who have experienced an organ transplant.
• Sickle cell disease.
• Chronic graft versus host disease.
• Amyloidosis.
• Aplastic anemia.
• Melanoma.
• Lung cancer.
• Cancer and young patients.
• Cancer treatment in pregnancy.
• Cancer and sexual dysfunction.
• Cancer and depression.
• Clotting disorders (e.g., hemophilia A (factor VIII deficiency) and von Willebrand disease).
• Thrombotic disorders (e.g., antithrombin deficiency and protein C deficiency).
• Primary humoral immune deficiencies (e.g., common variable immune deficiency).
• Neurologic disorders treated with immune globulins (e.g., chronic inflammatory demyelinating polyneuropathy).
• Hereditary angioedema.
• Alpha-1 antitrypsin deficiency. 

The FDA notice reads as follows:

As part of the performance commitments of the fifth authorization of the (PDUFA V), FDA has published a notice in the Federal Register related to Patient-Focused Drug Development, an initiative that provides for a more systematic approach to obtaining the patient perspective on certain disease areas in PDUFA V.  

Over the next five years, FDA will conduct public meetings for 20 different disease areas. The meetings will focus on the patient perspective regarding two key considerations in FDA’s regulatory decision-making: the severity of the disease and the currently available treatment options. In determining the disease areas that will be addressed in these meetings, FDA has published a preliminary list of nominated diseases in a Federal Registry notice. The public is invited to comment at a public meeting on October 25, 2012, where FDA will provide an overview of Patient-Focused Drug Development. Registration to attend this meeting must be received by October 18, 2012. For more information about this meeting, including the list of nominated diseases, see the Federal Register notice (http://www.gpo.gov/fdsys/pkg/FR-2012-09-24/pdf/2012-23454.pdf). The public is also invited to submit written comments into the public docket FDA-2012-N-0967 (http://www.regulations.gov/#!searchResults;rpp=25;po=0;s=pdufa). Written comments are due by November 1, 2012.  

In addition to the October 25th meeting to “kick off” planning for the 20 disease-related meetings under the PDUFA V performance commitment, FDA will also conduct periodic consultation meetings with patient stakeholders regarding process issues related to the agency’s implementation of Patient-Focused Drug Development. The consultation meetings will address important considerations and challenges in establishing and conducting a process that will be useful to both the patient community and FDA. The first of these consultation meetings will take place on October 10, 2012. Notification of intention to participate in this series of meetings must be submitted by October 3, 2012. For more information about these meetings, see the Federal Register notice  (http://www.gpo.gov/fdsys/pkg/FR-2012-09-24/pdf/2012-23453.pdf).

We look forward to your participation in this process. If you have any questions, please feel free to contact the Office of Special Health Issues at301-796-8460.

Should Diabetes be on the Agenda of Upcoming FDA Public Meetings?

Please share your voice. Ask the FDA to include diabetes in their public meeting process. 

The FDA is asking for public comments on twenty topics in meetings on patient-focused drug development. The FDA says the idea is a systematic way of getting patient views on the severity and treatments for a set of disease areas. They have a preliminary list of nominated diseases. Diabetes per say and the specific types of diabetes are not on the list. Two specific diabetes complications are; diabetic ulcers and diabetic foot infections. 

Twenty diseases and diabetes isn't on the list. That does not seem balanced. I think the DOC should get diabetes on the FDA's list. 

I would like to see this PDUFA effort consider diabetes in a manner similar to how they propose treating cancer. If you do too please comment to the FDA.

The proposed cancer areas are a more divers perspectives. For example the draft offers to consider; melanoma, lung cancer, cancer and young patients, cancer treatment in pregnancy, cancer and sexual dysfunction, cancer and depression. So how about T1D, T2D, diabetes and young patients, Gestational and other diabetes and pregnancy, diabetes and sexual dysfunction, diabetes and depression.

If the FDA is holding meeting on twenty diseases one topic should be diabetes. The FDA is open to diseases not on the proposed preliminary list. They ask when offering an additional disease for condition for consideration the following criteria be used:

• Disease areas that are chronic, symptomatic, or affect functioning and activities of daily living;
• Disease areas that reflect a range of severity;
• Disease areas for which aspects of the disease are not formally captured in clinical trials;
• Disease areas that have a severe impact on identifiable subpopulations (such as children or the elderly);
• Disease areas that represent a broad range in terms of size of the affected population; or
• Disease areas for which there are currently no therapies or very few therapies, or the available therapies do not directly affect how a patient feels, functions, or survives.

There is a public meeting. More information is available in the Federal Register: 

http://www.gpo.gov/fdsys/pkg/FR-2012-09-24/pdf/2012-23454.pdf?source=govdelivery

Comments can also be sent before Nov 1 2012.
http://www.regulations.gov/#!submitComment;D=FDA-2012-N-0967-0001

My comments are:

I thank the FDA for this opportunity to share views as a patient on the development of medications. I am a parent of two type 1 teens and a pre type 2 patient myself. We are just few of the over 25 million Americans that the CDC estimates live with diabetes.  I urge the FDA to consider a wide perspective on diabetes in this PDUFA meeting process. Specifically I hope the FDA will dedicate one of the twenty planned disease topics to diabetes. That should include at all forms of diabetes and their related complications. Specifically diabetes should be viewed by its different types, Type 1, Type 2 LADA and gestational. 

Type 1 and LADA are chronic autoimmune diseases that have a significant impact on patients’ lives. All forms of diabetes require daily behavioral changes. Bond et. al. write in the May/June 2010 Diabetes Educator, “Despite advances in treatment regiments now available for care, the self management activities of diabetes remain complex, with treatment recommendations difficult to incorporate into existing lifestyles. The daily management of diabetes can be overwhelming.”¹ 

The severity of changes to live with diabetes vary. For some, like myself, healthy activity and diet may manage blood sugar. For other, including my teens, significant effort is required through out every day to replace insulin destroyed in the autoimmune form of the disease. For all there are significant life changes.

Innovative medications offer the promise of better outcomes. Glucose responsive insulins (GRI) may help minimize potentially fatal hypoglycemic risk of using insulin. The FDA should be preparing the expertise needed to efficiently evaluate this innovation. By doing so the agency can expeditiously consider GRI applications.

The FDA can help foster evaluation of better treatments for the very young T1D patients. Little children respond to very small amounts of insulin. Their developing communication skills make it difficult for them to share with parents and care givers when they are experiencing hypoglycemia. Many children and adolescents are not aware of theses symptoms. 

Mental Health issues are more common with people with diabetes.^2,3,4  Van Bastelaar et al site estimations that as many as “20% of diabetes patients suffer from clinical depression.”

Diabetes is a significant public health issues. As such, I hope the FDA will consider a board evaluation of the issues diabetes patients face in the PDUFA meeting process. 

¹ Bond, G. E., R. L. Burr, F. M. Wolf, and K. Feldt. "The Effects of a Web-Based Intervention on Psychosocial Well-Being Among Adults Aged 60 and Older With Diabetes: A Randomized Trial." The Diabetes Educator 36.3 (2010): 446-56. Print. 

² Van Bastelaar, Kim, Pim Cuijpers, François Pouwer, Heleen Riper, and Frank J. Snoek. "Development and Reach of a Web-based Cognitive Behavioural Therapy Programme to Reduce Symptoms of Depression and Diabetes-specific Distress." Patient Education and Counseling 84.1 (2010): 49-55. Print. 

³ Anderson RJ, Freedland KE, Clouse RE, Lustman PJ. The prevalence of comorbid depression in adults with diabetes: a meta-analysis. Diabetes Care 2001;24:1069–78.

⁴ Mosaku K, Kolawole B, Mume C, Ikem R. Depression, anxiety and quality of life among diabetic patients: a comparative study. J Natl Med Assoc 2008;100:73– 8

I am for, "Patient Participation in Medical Product Discussions"

FDA has a very short term docket (30 days closes on Dec 4) about Patient Participation in Medical Product Discussions. Right smack in the middle of Diabetes month Thanksgiving. Not ideal comment time. 

So DOC, we need your help. 

Stephen Shaul started the ball rolling to get some comments in. The goal of comments is to be complimentary of the 2014 actions FDA has taken with the diabetes community and ask to keep that going, and maybe step that up with a PDUFA meeting.

If you can cut paste a comment great, if you can riff on the talking points making you comments similar but unique, even better. Everything you need is here: http://www.stripsafely.com/comment-on-patient-participation-with-fda/

Personally I would like to see most o the comments in the docket be on diabetes issues. Let's dominate the docket. 

Time is short. 

Thanks for stepping up.

FDA / Sponsor a Patient Meeting on Diabetes

The Food and Drug Administration has begun to hold a series of patient meetings to gain a better understanding of specific diseases. Over the next five years, the agency plans to conduct at least 20 such meetings on conditions ranging from Chronic Fatigue Syndrome to Narcolepsy to Irritable Bowel Syndrome.

But not diabetes.

That is unacceptable.

Four “slots” remain open (view the current list), and we want the FDA to add diabetes to its meeting docket for 2013.

According to the American Diabetes Association, nearly 26 million Americans have either type 1 or type 2 diabetes, and an additional 79 million have prediabetes. Those numbers are also increasing each year – the CDC estimates that if current trends continue, one in three US adults will have diabetes by 2050.

No one disputes that diabetes is one of America’s – and the world’s – most serious health epidemics. In 2012, more than one in five total US healthcare dollars was spent on diabetes, totaling $245 billion, and the government covered 62% of those costs, meaning everyone is paying for this epidemic. Despite growing needs, research dollars have also leveled off.

New therapies need to be investigated, tested, and brought to market, and as part of that process, the FDA must understand the daily challenges that patients face.

We need to tell our story to the FDA: about the need for accurate strips, more physiologic insulin, a broader range of drugs, and the Artificial Pancreas.

Our voices need to be heard.

Please sign this petition to urge the FDA to sponsor a patient meeting focused on diabetes – and the sooner, the better.

Thank you very much.

You can sign the petition here:

Diatribe.org/petition

YDMV on PDUFA

FTNW: MannKind Resumbits Afrezza

Inhaled Insulin Resubmitted to FDA.

MannKind Corporation (NASDAQ:MNKD) announced that it has submitted, and the U.S. Food and Drug Administration (FDA) has accepted, MannKind's resubmission of its New Drug Application (NDA) for AFREZZA™ (insulin human [rDNA origin]) and classified it as a Class 2 resubmission. With the Class 2 designation, the FDA set a corresponding Prescription Drug User Fee Act (PDUFA) action date of December 29, 2010.
http://www.medicalnewstoday.com/articles/195474.php

It is said this insulin peaks at 14 to 14 minutes. That is interesting.

As a side note, I find it faster to read Scott S's Tweets than my news reader for news like this. Thanks Scott.