Drug Development - Revision history

Akrokosky: /* Resources */

2018-04-08T18:24:08Z

Resources

Akrokosky at 18:14, 8 April 2018

2018-04-08T18:14:52Z

Akrokosky at 18:12, 8 April 2018

2018-04-08T18:12:55Z

Akrokosky at 18:10, 8 April 2018

2018-04-08T18:10:38Z

Akrokosky at 18:08, 8 April 2018

2018-04-08T18:08:40Z

Akrokosky: Created page with "'''Externally Led Patient Focused Drug Development Meeting - Experiences from Groups''' *''Friedreich’s Ataxia Research Alliance'' FARA has hosted an ELPFDD as a stand al..."

2018-04-08T18:04:59Z

Created page with "'''Externally Led Patient Focused Drug Development Meeting - Experiences from Groups''' *''Friedreich’s Ataxia Research Alliance'' FARA has hosted an ELPFDD as a stand al..."

New page

'''Externally Led Patient Focused Drug Development Meeting - Experiences from Groups'''

*''Friedreich’s Ataxia Research Alliance''

FARA has hosted an ELPFDD as a stand alone meeting, held a reception the night prior and a board meeting following the ELPFDD. FARA leadership felt the merit of the ELPFDD was important and sufficient to stand on its own. A great deal of time and attention was given to identify appropriate panelists and in preparing panelists for their role in this meeting. Preparation was also provided to members of the audience who wished to make statements. The meeting was described by Ron Bartek as monumental in building relationships with the FDA and in providing insights about Friedreich's ataxia from the patient's perspective. The group hired a consultant. Participation was not restricted to US residents. Anyone from anywhere was included. Many members of the community joined remotely. All responses provided via cell phone were tallied together and responses feedback to all regardless of whether the participant was attending the face-to-face meeting or joining online.

@@ Line 14: / Line 14: @@
 *''Organic Acidemia Association'' and ''XLH Network'' are currently planning a ELPFDD meeting.
-== Resources ==
+== Externally Led Patient Focused Drug Development Resource Links ==
-[http://www.example.com link title]
+[http://www.porphyriafoundation.com/content/fda-meeting-acute-porphyrias-march-1st-0 Acute Porphyrias]
-Global Genes
+[http://www.curefa.org/pdf/research/CoverPage-VOP-Report.pdf Friedreich's Ataxia (FA)]
-http://www.curefa.org/news/friedreich-s-ataxia-voice-of-the-patient-report
+[http://www.curefa.org/news/friedreich-s-ataxia-voice-of-the-patient-report Friedreich's Ataxia (FA)#2]
-Tuberous Sclerosis
+[https://globalgenes.org/fdaworkshop Global Genes]
-http://www.tsalliance.org/individuals-families/patient-focused-drug-development/
+[https://www.sweathelp.org/taking-action/patient-focused-drug-development.html Hyperhidrosis]
-CG3, National Kidney Foundation
+[https://lupuspfdd.org/ Lupus]
-https://www.kidney.org/sites/default/files/C3G-PFDD-Agenda_FINAL.pdf
+[http://www.myotonic.org/developing-therapies-brain-related-dm-symptoms-add-your-voice Myotonic Dystrophy]
-Lupus
+[https://www.kidney.org/sites/default/files/C3G-PFDD-Agenda_FINAL.pdf CG3, National Kidney Foundation]
-https://lupuspfdd.org/
+[https://www.arthritis.org/Documents/Sections/Advocate/Regulatory-Letters/AF-Comments-FDA-PDUFA-VI-Comittment-Letter.pdf Osteoarthritis]
-Hyperhidrosis
+[http://www.curesma.org/news/sma-voice-of-the-patient.html?referrer=https://www.google.com/ Spinal Muscular Atrophy]
-https://www.sweathelp.org/taking-action/patient-focused-drug-development.html
+[http://www.tsalliance.org/individuals-families/patient-focused-drug-development/ Tuberous Sclerosis ]

@@ Line 15: / Line 15: @@
 == Resources ==
-[[FDA: https://www.fda.gov/ForIndustry/UserFees/PrescriptionDrugUserFee/ucm453856.htm]]
+[https://www.fda.gov/ForIndustry/UserFees/PrescriptionDrugUserFee/ucm453856.htm FDA]
 Global Genes

← Older revision		Revision as of 18:12, 8 April 2018
Line 14:		Line 14:
	*''Organic Acidemia Association'' and ''XLH Network'' are currently planning a ELPFDD meeting.		*''Organic Acidemia Association'' and ''XLH Network'' are currently planning a ELPFDD meeting.

		+	== Resources ==
		+	[[FDA: https://www.fda.gov/ForIndustry/UserFees/PrescriptionDrugUserFee/ucm453856.htm]]
		+
		+	Global Genes
		+
		+	https://globalgenes.org/fdaworkshop
		+
		+	Amyloidosis
		+
		+	http://www.arci.org/fda-page/
		+
		+	Myotonic Dystrophy
		+
		+	http://www.myotonic.org/developing-therapies-brain-related-dm-symptoms-add-your-voice
		+
		+	Acute Porphyrias
		+
		+	http://www.porphyriafoundation.com/content/fda-meeting-acute-porphyrias-march-1st-0
		+
		+	Osteoarthritis
		+
		+	https://www.arthritis.org/Documents/Sections/Advocate/Regulatory-Letters/AF-Comments-FDA-PDUFA-VI-Comittment-Letter.pdf
		+
		+	Spinal Muscular Atrophy
		+
		+	http://www.curesma.org/news/sma-voice-of-the-patient.html?referrer=https://www.google.com/
		+
		+	Friedreich's Ataxia (FA)
		+
		+	http://www.curefa.org/pdf/research/CoverPage-VOP-Report.pdf
		+
		+	http://www.curefa.org/news/friedreich-s-ataxia-voice-of-the-patient-report
		+
		+	Tuberous Sclerosis
		+
		+	http://www.tsalliance.org/individuals-families/patient-focused-drug-development/
		+
		+	CG3, National Kidney Foundation
		+
		+	https://www.kidney.org/sites/default/files/C3G-PFDD-Agenda_FINAL.pdf
		+
		+	Lupus
		+
		+	https://lupuspfdd.org/

−	~~== Resources ==~~	+	Hyperhidrosis
		+
		+	https://www.sweathelp.org/taking-action/patient-focused-drug-development.html

← Older revision		Revision as of 18:10, 8 April 2018
Line 13:		Line 13:

	*''Organic Acidemia Association'' and ''XLH Network'' are currently planning a ELPFDD meeting.		*''Organic Acidemia Association'' and ''XLH Network'' are currently planning a ELPFDD meeting.
		+
		+
		+	== Resources ==

← Older revision		Revision as of 18:08, 8 April 2018
Line 4:		Line 4:

	FARA has hosted an ELPFDD as a stand alone meeting, held a reception the night prior and a board meeting following the ELPFDD. FARA leadership felt the merit of the ELPFDD was important and sufficient to stand on its own. A great deal of time and attention was given to identify appropriate panelists and in preparing panelists for their role in this meeting. Preparation was also provided to members of the audience who wished to make statements. The meeting was described by Ron Bartek as monumental in building relationships with the FDA and in providing insights about Friedreich's ataxia from the patient's perspective. The group hired a consultant. Participation was not restricted to US residents. Anyone from anywhere was included. Many members of the community joined remotely. All responses provided via cell phone were tallied together and responses feedback to all regardless of whether the participant was attending the face-to-face meeting or joining online.		FARA has hosted an ELPFDD as a stand alone meeting, held a reception the night prior and a board meeting following the ELPFDD. FARA leadership felt the merit of the ELPFDD was important and sufficient to stand on its own. A great deal of time and attention was given to identify appropriate panelists and in preparing panelists for their role in this meeting. Preparation was also provided to members of the audience who wished to make statements. The meeting was described by Ron Bartek as monumental in building relationships with the FDA and in providing insights about Friedreich's ataxia from the patient's perspective. The group hired a consultant. Participation was not restricted to US residents. Anyone from anywhere was included. Many members of the community joined remotely. All responses provided via cell phone were tallied together and responses feedback to all regardless of whether the participant was attending the face-to-face meeting or joining online.
		+
		+	*''Genetic Alliance''
		+
		+	Genetic Alliance led ELPFDD for 3 diseases using the Platform for Engaging Everyone Responsibly (PEER) system. Some communities use PEER for registries, some for campaigns and some for trials. The six disease groups (including three orgs representing sickle cell disease) developed videos and copy placed on GA portal. Tablets were used by volunteers in infusion clinics to get people’s preferences. These three groups were selected to be included in the first phase of the FDA Patient Focused Drug Development.
		+
		+	*''Tuberous Sclerosis Alliance ''
		+	Hosted a ELPFDD, see resources provided below.
		+
		+	*''Organic Acidemia Association'' and ''XLH Network'' are currently planning a ELPFDD meeting.