Expediting Drug Development : FDA's New Regenerative Medicine Advanced Therapy Designation
In March 2017, the US FDA introduced the new Regenerative Medicine Advanced Therapy (RMAT) designation thus recognizing the enormous potential of these medicines and the need for efficient regulatory tools to accelerate their development and their commercial availability. The development of regenerative medicines is very challenging because of their complex and unique nature, especially to the rather unexperienced small- and medium-sized developing enterprises. With the new RMAT designation, FDA aims at providing intensive support to companies developing cell- and tissue-based therapies, tissue-engineering products, and combination treatments. This may also include cell-based products where the genome has been edited by emerging technologies such as CRISPR-Cas9. This article presents the newly launched "Regenerative Medicine Advanced Therapy" (RMAT) designation, outlines existing FDA regulatory tools aiming at expediting approval, and discusses the overall value of these programs. Additionally, recommendations are provided for companies developing these very specific and complex therapies on how and when to consider these tools for an integrated development and regulatory strategy.
| Medienart: |
E-Artikel |
|---|
| Erscheinungsjahr: |
2019 |
|---|---|
| Erschienen: |
2019 |
| Enthalten in: |
Zur Gesamtaufnahme - volume:53 |
|---|---|
| Enthalten in: |
Therapeutic innovation & regulatory science - 53(2019), 3 vom: 23. Mai, Seite 364-373 |
| Sprache: |
Englisch |
|---|
| Beteiligte Personen: |
Vaggelas, Annegret [VerfasserIn] |
|---|
| Links: |
|---|
| Themen: |
CAR T cell |
|---|
| Anmerkungen: |
Date Completed 27.01.2020 Date Revised 27.01.2020 published: Print-Electronic Citation Status MEDLINE |
|---|
| doi: |
10.1177/2168479018779373 |
|---|
| funding: |
|
|---|---|
| Förderinstitution / Projekttitel: |
|
| PPN (Katalog-ID): |
NLM285395939 |
|---|
| LEADER | 01000naa a22002652 4500 | ||
|---|---|---|---|
| 001 | NLM285395939 | ||
| 003 | DE-627 | ||
| 005 | 20231225045344.0 | ||
| 007 | cr uuu---uuuuu | ||
| 008 | 231225s2019 xx |||||o 00| ||eng c | ||
| 024 | 7 | |a 10.1177/2168479018779373 |2 doi | |
| 028 | 5 | 2 | |a pubmed24n0951.xml |
| 035 | |a (DE-627)NLM285395939 | ||
| 035 | |a (NLM)29895180 | ||
| 040 | |a DE-627 |b ger |c DE-627 |e rakwb | ||
| 041 | |a eng | ||
| 100 | 1 | |a Vaggelas, Annegret |e verfasserin |4 aut | |
| 245 | 1 | 0 | |a Expediting Drug Development |b FDA's New Regenerative Medicine Advanced Therapy Designation |
| 264 | 1 | |c 2019 | |
| 336 | |a Text |b txt |2 rdacontent | ||
| 337 | |a ƒaComputermedien |b c |2 rdamedia | ||
| 338 | |a ƒa Online-Ressource |b cr |2 rdacarrier | ||
| 500 | |a Date Completed 27.01.2020 | ||
| 500 | |a Date Revised 27.01.2020 | ||
| 500 | |a published: Print-Electronic | ||
| 500 | |a Citation Status MEDLINE | ||
| 520 | |a In March 2017, the US FDA introduced the new Regenerative Medicine Advanced Therapy (RMAT) designation thus recognizing the enormous potential of these medicines and the need for efficient regulatory tools to accelerate their development and their commercial availability. The development of regenerative medicines is very challenging because of their complex and unique nature, especially to the rather unexperienced small- and medium-sized developing enterprises. With the new RMAT designation, FDA aims at providing intensive support to companies developing cell- and tissue-based therapies, tissue-engineering products, and combination treatments. This may also include cell-based products where the genome has been edited by emerging technologies such as CRISPR-Cas9. This article presents the newly launched "Regenerative Medicine Advanced Therapy" (RMAT) designation, outlines existing FDA regulatory tools aiming at expediting approval, and discusses the overall value of these programs. Additionally, recommendations are provided for companies developing these very specific and complex therapies on how and when to consider these tools for an integrated development and regulatory strategy | ||
| 650 | 4 | |a Journal Article | |
| 650 | 4 | |a CAR T cell | |
| 650 | 4 | |a RMAT | |
| 650 | 4 | |a cell therapies | |
| 650 | 4 | |a expedited programs | |
| 650 | 4 | |a gene therapies | |
| 650 | 4 | |a genome editing | |
| 700 | 1 | |a Seimetz, Diane |e verfasserin |4 aut | |
| 773 | 0 | 8 | |i Enthalten in |t Therapeutic innovation & regulatory science |d 2013 |g 53(2019), 3 vom: 23. Mai, Seite 364-373 |w (DE-627)NLM243752342 |x 2168-4804 |7 nnns |
| 773 | 1 | 8 | |g volume:53 |g year:2019 |g number:3 |g day:23 |g month:05 |g pages:364-373 |
| 856 | 4 | 0 | |u http://dx.doi.org/10.1177/2168479018779373 |3 Volltext |
| 912 | |a GBV_USEFLAG_A | ||
| 912 | |a GBV_NLM | ||
| 951 | |a AR | ||
| 952 | |d 53 |j 2019 |e 3 |b 23 |c 05 |h 364-373 | ||