527 lines
40 KiB
Text
527 lines
40 KiB
Text
<!DOCTYPE html>
|
||
<html lang="en" dir="ltr" prefix="og: https://ogp.me/ns#">
|
||
<head>
|
||
<meta charset="utf-8" />
|
||
<meta name="description" content="NCATS Toolkit provides information and resources about the growing role of patient voices from rare disease communities in treatment development." />
|
||
<meta name="keywords" content="rare diseases, patient groups, therapy development, FDA approval" />
|
||
<link rel="canonical" href="http://ncats.nih.gov/research/research-resources/ncats-toolkit/about" />
|
||
<meta name="google" content="PROqkJCbtNawPO29xzL4ENjcb0_EckySw96i97QXx8s" />
|
||
<meta name="google-site-verification" content="PROqkJCbtNawPO29xzL4ENjcb0_EckySw96i97QXx8s" />
|
||
<meta name="Generator" content="Drupal 10 (https://www.drupal.org)" />
|
||
<meta name="MobileOptimized" content="width" />
|
||
<meta name="HandheldFriendly" content="true" />
|
||
<meta name="viewport" content="width=device-width, initial-scale=1.0" />
|
||
<link rel="icon" href="/themes/custom/d9_subtheme/assets/images/favicon.ico" type="image/vnd.microsoft.icon" />
|
||
|
||
<title>About NCATS Toolkit | National Center for Advancing Translational Sciences</title>
|
||
<link rel="stylesheet" media="all" href="/libraries/drupal-superfish/css/superfish.css?srmo5o" />
|
||
<link rel="stylesheet" media="all" href="/core/modules/system/css/components/ajax-progress.module.css?srmo5o" />
|
||
<link rel="stylesheet" media="all" href="/core/modules/system/css/components/align.module.css?srmo5o" />
|
||
<link rel="stylesheet" media="all" href="/core/modules/system/css/components/autocomplete-loading.module.css?srmo5o" />
|
||
<link rel="stylesheet" media="all" href="/core/modules/system/css/components/fieldgroup.module.css?srmo5o" />
|
||
<link rel="stylesheet" media="all" href="/core/modules/system/css/components/container-inline.module.css?srmo5o" />
|
||
<link rel="stylesheet" media="all" href="/core/modules/system/css/components/clearfix.module.css?srmo5o" />
|
||
<link rel="stylesheet" media="all" href="/core/modules/system/css/components/details.module.css?srmo5o" />
|
||
<link rel="stylesheet" media="all" href="/core/modules/system/css/components/hidden.module.css?srmo5o" />
|
||
<link rel="stylesheet" media="all" href="/core/modules/system/css/components/item-list.module.css?srmo5o" />
|
||
<link rel="stylesheet" media="all" href="/core/modules/system/css/components/js.module.css?srmo5o" />
|
||
<link rel="stylesheet" media="all" href="/core/modules/system/css/components/nowrap.module.css?srmo5o" />
|
||
<link rel="stylesheet" media="all" href="/core/modules/system/css/components/position-container.module.css?srmo5o" />
|
||
<link rel="stylesheet" media="all" href="/core/modules/system/css/components/progress.module.css?srmo5o" />
|
||
<link rel="stylesheet" media="all" href="/core/modules/system/css/components/reset-appearance.module.css?srmo5o" />
|
||
<link rel="stylesheet" media="all" href="/core/modules/system/css/components/resize.module.css?srmo5o" />
|
||
<link rel="stylesheet" media="all" href="/core/modules/system/css/components/sticky-header.module.css?srmo5o" />
|
||
<link rel="stylesheet" media="all" href="/core/modules/system/css/components/system-status-counter.css?srmo5o" />
|
||
<link rel="stylesheet" media="all" href="/core/modules/system/css/components/system-status-report-counters.css?srmo5o" />
|
||
<link rel="stylesheet" media="all" href="/core/modules/system/css/components/system-status-report-general-info.css?srmo5o" />
|
||
<link rel="stylesheet" media="all" href="/core/modules/system/css/components/tabledrag.module.css?srmo5o" />
|
||
<link rel="stylesheet" media="all" href="/core/modules/system/css/components/tablesort.module.css?srmo5o" />
|
||
<link rel="stylesheet" media="all" href="/core/modules/system/css/components/tree-child.module.css?srmo5o" />
|
||
<link rel="stylesheet" media="all" href="/modules/contrib/extlink/extlink.css?srmo5o" />
|
||
<link rel="stylesheet" media="all" href="/core/modules/layout_discovery/layouts/onecol/onecol.css?srmo5o" />
|
||
<link rel="stylesheet" media="all" href="/modules/contrib/paragraphs/css/paragraphs.unpublished.css?srmo5o" />
|
||
<link rel="stylesheet" media="all" href="https://cdn.jsdelivr.net/npm/@uswds/uswds@3.3.0/dist/css/uswds.min.css" />
|
||
<link rel="stylesheet" media="all" href="https://cdn.jsdelivr.net/npm/bootstrap@5.0.2/dist/css/bootstrap.min.css" />
|
||
<link rel="stylesheet" media="all" href="https://cdn.jsdelivr.net/npm/bootstrap-icons@1.11.1/font/bootstrap-icons.css" />
|
||
<link rel="stylesheet" media="all" href="/themes/custom/d9_subtheme/css/styles.css?srmo5o" />
|
||
|
||
<script src="https://dap.digitalgov.gov/Universal-Federated-Analytics-Min.js?agency=HHS&subagency=NIH" id="_fed_an_ua_tag" async type="text/javascript"></script>
|
||
|
||
</head>
|
||
<body class="path-node page-node-type-landing-page-new-">
|
||
<a href="#main-content" class="usa-sr-only focusable">
|
||
Skip to main content
|
||
</a>
|
||
<noscript><iframe src="https://www.googletagmanager.com/ns.html?id=GTM-55CGV66V"
|
||
height="0" width="0" style="display:none;visibility:hidden"></iframe></noscript>
|
||
|
||
<div class="dialog-off-canvas-main-canvas" data-off-canvas-main-canvas>
|
||
|
||
<section class="navigation-top">
|
||
<div class="container">
|
||
<div class="row">
|
||
<div class="col-12">
|
||
<div class="link-small d-flex">
|
||
|
||
<a href="http://www.hhs.gov/" title="Go to the hhs.gov" class="" target="_blank">
|
||
<img src="/sites/default/files/2023-10/hhs-white.png" alt="HHS Logo">
|
||
<span class="d-xl-inline d-lg-inline d-none small">U.S. Department of Health & Human Services</span>
|
||
</a>
|
||
|
||
<a href="http://www.nih.gov/" title="National Institutes of Health | Homepage" class="" target="_blank">
|
||
<img src="/sites/default/files/2023-10/nih-logo.png" alt="NIH Logo">
|
||
<span class="d-xl-inline d-lg-inline d-none small">National Institutes of Health</span>
|
||
</a>
|
||
|
||
</div>
|
||
</div>
|
||
</div>
|
||
</div>
|
||
</section>
|
||
|
||
|
||
<div class="usa-overlay"></div>
|
||
|
||
<header class="usa-header usa-header--extended usa-header--extended-megamenu" id="header" role="banner">
|
||
|
||
|
||
|
||
|
||
|
||
<div class="region region-header grid-container">
|
||
<div class="grid-row grid-gap-1"><div class="desktop:grid-col-3 header-first">
|
||
<div class="usa-logo site-logo" id="logo">
|
||
|
||
<em class="usa-logo__text">
|
||
<a class="logo-img" href="/" accesskey="1" title="Home" aria-label="Home">
|
||
<img src="/themes/custom/d9_subtheme/logo.png" alt="Home" />
|
||
</a>
|
||
|
||
|
||
</em>
|
||
|
||
</div>
|
||
<div class="search-icon d-md-none"><a class="toggle-search" href="#"><i class="bi bi-search"></i></a></div>
|
||
|
||
</div><div class="desktop:grid-col-7">
|
||
<div id="block-navigation--2" class="block block-superfish block-superfishnavigation">
|
||
|
||
|
||
|
||
<ul id="superfish-navigation" class="menu sf-menu sf-navigation sf-horizontal sf-style-none">
|
||
|
||
<li id="navigation-menu-link-contentaed842ec-f581-4cea-b0b0-4a85c50def86" class="active-trail sf-depth-1 menuparent"><a href="/research" class="sf-depth-1 menuparent">Research</a><ul><li id="navigation-menu-link-content5a3b6aac-7ae9-45d7-929f-fbf776db20f0" class="sf-depth-2 sf-no-children"><a href="/research/overview" class="sf-depth-2">Research Overview</a></li><li id="navigation-menu-link-contentd0507d8b-7b88-453c-908a-2b7e961c0479" class="sf-depth-2 sf-no-children"><a href="/research/our-impact" class="sf-depth-2">Our Impact</a></li><li id="navigation-menu-link-contented530d1e-2ec3-46db-a529-9029be3f3fb4" class="sf-depth-2 sf-no-children"><a href="/research/research-activities" class="sf-depth-2">Research Activities</a></li><li id="navigation-menu-link-content2dac8545-38f2-47c0-8993-ea0cae65e123" class="active-trail sf-depth-2 sf-no-children"><a href="/research/research-resources" class="sf-depth-2">Research Resources</a></li><li id="navigation-menu-link-content311ec98e-6232-4e22-9966-4a9ac796870a" class="sf-depth-2 sf-no-children"><a href="/research/training-education" class="sf-depth-2">Training & Education</a></li><li id="navigation-menu-link-content52b1ba41-94ea-4d84-9db7-7482b8f78f16" class="sf-depth-2 sf-no-children"><a href="/research/alliances" class="sf-depth-2">Alliances</a></li><li id="navigation-menu-link-contenta185af75-90a5-4cc4-813f-ef66f35e21ea" class="sf-depth-2 sf-no-children"><a href="/research/collaborate-with-ncats" class="sf-depth-2">Collaborate With NCATS</a></li></ul></li><li id="navigation-menu-link-content84a3cc1f-203d-4de6-ab26-d80c256b96dc" class="sf-depth-1 menuparent"><a href="/funding" class="sf-depth-1 menuparent">Funding</a><ul><li id="navigation-menu-link-content0a09bed2-3d99-4faf-b3c5-fc9a4878629a" class="sf-depth-2 sf-no-children"><a href="/funding/overview" class="sf-depth-2">Funding Overview</a></li><li id="navigation-menu-link-contentd85a1f38-7893-4e4a-8514-5b9e767983e2" class="sf-depth-2 sf-no-children"><a href="/funding/funding-opportunities" class="sf-depth-2">Open Funding Opportunities</a></li><li id="navigation-menu-link-content81e84c00-ef73-4aff-a15f-7728cc398801" class="sf-depth-2 sf-no-children"><a href="/funding/small-business-programs" class="sf-depth-2">Small Business Programs</a></li><li id="navigation-menu-link-content631a166b-650b-450f-97b4-9014d14935e9" class="sf-depth-2 sf-no-children"><a href="/funding/funding-policy-operating-guidelines" class="sf-depth-2">Funding Policy & Operating Guidelines</a></li><li id="navigation-menu-link-content5d56ebe7-b3e8-452a-bd30-df92b0deabef" class="sf-depth-2 sf-no-children"><a href="/funding/grant-information" class="sf-depth-2">Grant Information</a></li><li id="navigation-menu-link-content4429fdc9-e911-4022-b3dc-3f6a4980b9ff" class="sf-depth-2 sf-no-children"><a href="/funding/challenges" class="sf-depth-2">Challenges and Prize Competitions</a></li><li id="navigation-menu-link-content943fe682-94ff-4fcf-a6b0-32096d2b2d0e" class="sf-depth-2 sf-no-children"><a href="/funding/scientific-review" class="sf-depth-2">Scientific Review</a></li><li id="navigation-menu-link-content10496705-320e-4437-b2ba-ea2830b293d1" class="sf-depth-2 sf-no-children"><a href="/funding/conference-grant-information" class="sf-depth-2">Conference Grant Information</a></li></ul></li><li id="navigation-menu-link-contentea91f2ef-1b8d-4b1c-83e7-5830769dd505" class="sf-depth-1 menuparent"><a href="/resources" class="sf-depth-1 menuparent">Resources For You</a><ul><li id="navigation-menu-link-content567e64e3-685d-41bc-8bf4-130c7689d6a2" class="sf-depth-2 sf-no-children"><a href="/resources/overview" title="Resources for You" class="sf-depth-2">Resources for You</a></li><li id="navigation-menu-link-content786ce1f5-ff7a-463a-bb05-c01882062426" class="sf-depth-2 sf-no-children"><a href="/resources/for-researchers" title="Resources for Researchers" class="sf-depth-2">Resources for Researchers</a></li><li id="navigation-menu-link-contente737ec67-ecd0-4f18-915d-b61660cb524f" class="sf-depth-2 sf-no-children"><a href="/resources/for-trainees" title="Resources for Trainees" class="sf-depth-2">Resources for Trainees</a></li><li id="navigation-menu-link-content8812fdb6-0fb7-4b27-8401-2fc5b3f41ff1" class="sf-depth-2 sf-no-children"><a href="/resources/for-rare-disease-patients-and-advocates" title="Resources for Rare Disease Patients and Advocates" class="sf-depth-2">Resources for Rare Disease Patients and Advocates</a></li></ul></li><li id="navigation-menu-link-contenta8527040-df23-4117-a5a7-566bd83e2c7f" class="sf-depth-1 menuparent"><a href="/news-events" class="sf-depth-1 menuparent">News & Events</a><ul><li id="navigation-menu-link-contentf6836a81-a42a-4528-b569-18939d8451da" class="sf-depth-2 sf-no-children"><a href="/news-events/overview" class="sf-depth-2">News & Events Overview</a></li><li id="navigation-menu-link-content9f44edc6-e5b7-4a92-bcb3-c0a8777461f4" class="sf-depth-2 sf-no-children"><a href="/news-events/news" class="sf-depth-2">News</a></li><li id="navigation-menu-link-content402cff9f-12b7-459b-9f0f-56606930399c" class="sf-depth-2 sf-no-children"><a href="/news-events/newsletter" class="sf-depth-2">Newsletters</a></li><li id="navigation-menu-link-content6cd76cbe-053e-4e3b-8961-c9f5def57ba6" class="sf-depth-2 sf-no-children"><a href="/news-events/events" class="sf-depth-2">Events</a></li><li id="navigation-menu-link-content0a211675-2fb5-45cc-b443-c7946e7d9120" class="sf-depth-2 sf-no-children"><a href="/news-events/science-publications" class="sf-depth-2">Scientific Publications</a></li><li id="navigation-menu-link-contente6d4ed4f-826f-41b4-a9e2-728c2fb4ca62" class="sf-depth-2 sf-no-children"><a href="/news-events/multimedia" class="sf-depth-2">Multimedia</a></li></ul></li><li id="navigation-menu-link-contentf1eb1fa8-5813-433c-9bcc-dc56f2e258f3" class="sf-depth-1 menuparent"><a href="/about" class="sf-depth-1 menuparent">About NCATS</a><ul><li id="navigation-menu-link-contentada9a195-ae5a-446a-9014-c1232164e6db" class="sf-depth-2 sf-no-children"><a href="/about/ncats-overview" class="sf-depth-2">NCATS Overview</a></li><li id="navigation-menu-link-contentd5e0369a-aded-4348-84f7-463c64498447" class="sf-depth-2 sf-no-children"><a href="/about/about-translational-science" class="sf-depth-2">About Translational Science</a></li><li id="navigation-menu-link-contente298b7b2-724e-408b-9bb9-afcaac57a751" class="sf-depth-2 sf-no-children"><a href="/about/advisory-committees" class="sf-depth-2">Advisory Committees</a></li><li id="navigation-menu-link-contentbc9c91b7-8d3d-4d2c-af65-c25ef894d269" class="sf-depth-2 sf-no-children"><a href="/about/budget" class="sf-depth-2">Budget</a></li><li id="navigation-menu-link-contenta4ed4439-05db-4a41-9357-731c2ff74543" class="sf-depth-2 sf-no-children"><a href="/about/director" class="sf-depth-2">Director's Corner</a></li><li id="navigation-menu-link-contentb50f96af-3723-4634-8566-4e60c2639491" class="sf-depth-2 sf-no-children"><a href="/about/divisions-and-offices" class="sf-depth-2">Divisions & Offices</a></li><li id="navigation-menu-link-content41d11ad4-c576-46be-a326-1f8cf5059354" class="sf-depth-2 sf-no-children"><a href="/about/our-staff" class="sf-depth-2">Our Staff</a></li></ul></li>
|
||
</ul>
|
||
|
||
</div>
|
||
</div>
|
||
<div class="desktop:grid-col-2 search-block">
|
||
<form class="usa-search usa-search--small" action="/search/node" method="get" id="search-block-form" accept-charset="UTF-8">
|
||
<div role="search">
|
||
|
||
<input class="usa-input" id="extended-search-field-small" type="search" name="keys" />
|
||
<button class="usa-button" type="submit">
|
||
<img src="/themes/custom/d9_subtheme/node_modules/@uswds/uswds/dist/img/usa-icons-bg/search--white.svg" class="usa-search__submit-icon" alt="Search">
|
||
</button>
|
||
</div>
|
||
</form>
|
||
</div></div>
|
||
</div>
|
||
|
||
|
||
<nav class="usa-nav" role="navigation">
|
||
<div class="usa-nav__inner">
|
||
|
||
<button type="button" class="usa-nav__close">
|
||
<img src="/themes/custom/d9_subtheme/node_modules/@uswds/uswds/dist/img/usa-icons/close.svg" alt="close"/>
|
||
</button>
|
||
|
||
|
||
|
||
|
||
|
||
|
||
</div>
|
||
</nav>
|
||
|
||
|
||
</header>
|
||
|
||
<main class="main-content usa-layout-docs usa-section " role="main"
|
||
id="main-content">
|
||
|
||
<div class="grid-container-">
|
||
|
||
<div class="grid-row">
|
||
|
||
</div>
|
||
|
||
<div class="">
|
||
|
||
</div>
|
||
|
||
<div class="grid-row ">
|
||
|
||
</div>
|
||
|
||
<div class="grid-row grid-gap">
|
||
|
||
<div class="usa-layout-docs__main desktop:grid-col-fill">
|
||
<div class="">
|
||
<div data-drupal-messages-fallback class="hidden"></div>
|
||
<div id="block-d9-subtheme-content" class="block block-system block-system-main-block">
|
||
|
||
|
||
<article data-history-node-id="27617">
|
||
|
||
|
||
|
||
|
||
|
||
<div>
|
||
<div class="container layout layout--onecol">
|
||
<div class="layout__region layout__region--content">
|
||
<div class="block block-system block-system-breadcrumb-block">
|
||
|
||
|
||
<nav class="usa-breadcrumb" role="navigation" aria-labelledby="system-breadcrumb">
|
||
<h2 id="system-breadcrumb" class="usa-sr-only">Breadcrumb</h2>
|
||
<ol class="usa-breadcrumb__list">
|
||
<li class="usa-breadcrumb__list-item">
|
||
<a href="/" class="usa-breadcrumb__link"><span>Home</span></a>
|
||
</li>
|
||
<li class="usa-breadcrumb__list-item">
|
||
<a href="/research/overview" class="usa-breadcrumb__link"><span>Research</span></a>
|
||
</li>
|
||
<li class="usa-breadcrumb__list-item">
|
||
<a href="/research/research-resources" class="usa-breadcrumb__link"><span>Research Resources</span></a>
|
||
</li>
|
||
<li class="usa-breadcrumb__list-item">
|
||
<a href="/research/research-resources/ncats-toolkit" class="usa-breadcrumb__link"><span>NCATS Toolkit For Patient-Focused Therapy Development</span></a>
|
||
</li>
|
||
<li class="usa-breadcrumb__list-item">
|
||
<span>About NCATS Toolkit</span>
|
||
</li>
|
||
</ol>
|
||
</nav>
|
||
|
||
</div>
|
||
<div class="block block-layout-builder block-field-blocknodelanding-page-new-field-metatag">
|
||
|
||
|
||
|
||
</div>
|
||
|
||
</div>
|
||
</div>
|
||
<div class="layout layout--onecol">
|
||
<div class="layout__region layout__region--content">
|
||
<div class="block block-layout-builder block-field-blocknodelanding-page-new-field-landing-page-components">
|
||
|
||
|
||
|
||
|
||
|
||
<section class="main-hero" style="background-image:
|
||
linear-gradient(to left, rgba(255,255,255, 0.1), rgba(0, 0, 0, 0.99)),
|
||
url('/sites/default/files/2023-10/1440x350%20Toolkit%20Banner_0.png');">
|
||
<div class="container">
|
||
<div class="row">
|
||
<div class="col-12">
|
||
<div class="hero-content">
|
||
<h1> About NCATS Toolkit </h1>
|
||
<p class="text-white">
|
||
|
||
Our toolkit (NCATS Toolkit) provides information and resources about the growing role of patient voices from rare disease communities in treatment development.
|
||
</p>
|
||
</div>
|
||
</div>
|
||
</div>
|
||
</div>
|
||
</section>
|
||
<style type="text/css">
|
||
article {position: relative;}
|
||
.block-system-breadcrumb-block{
|
||
position:absolute;
|
||
top:0;
|
||
color:#fff;
|
||
}
|
||
.block-system-breadcrumb-block .usa-breadcrumb__list, .block-system-breadcrumb-block .usa-breadcrumb__list a {
|
||
color:#fff;
|
||
}
|
||
.block-system-breadcrumb-block .usa-breadcrumb__list .usa-breadcrumb__list-item:after {
|
||
background-color:#fff !important;
|
||
}
|
||
</style>
|
||
<div class="container subnav">
|
||
|
||
|
||
<nav role="navigation" aria-labelledby="block-navigation-menu" id="block-navigation">
|
||
|
||
<h2 class="usa-sr-only" id="block-navigation-menu">Navigation</h2>
|
||
|
||
|
||
|
||
<ul class="menu menu--navigation nav">
|
||
<li class="active active-trail first">
|
||
<a href="/research/research-resources/ncats-toolkit/about" class="active active-trail first active-trail is-active" data-drupal-link-system-path="node/27617">About NCATS Toolkit</a>
|
||
</li>
|
||
<li>
|
||
<a href="/research/research-resources/ncats-toolkit/share-resources" data-drupal-link-system-path="node/43157">Share Resources</a>
|
||
</li>
|
||
<li>
|
||
<a href="/research/research-resources/ncats-toolkit/toolkit-getting-started" data-drupal-link-system-path="node/27698">Getting Started</a>
|
||
</li>
|
||
<li>
|
||
<a href="/research/research-resources/ncats-toolkit/discovery" data-drupal-link-system-path="node/27619">Discovery</a>
|
||
</li>
|
||
<li>
|
||
<a href="/research/research-resources/ncats-toolkit/toolkit-to-prepare-clinical-trials" data-drupal-link-system-path="node/27706">Prepare for Clinical Trials</a>
|
||
</li>
|
||
<li>
|
||
<a href="/research/research-resources/ncats-toolkit/toolkit-clinical-trials-and-fda-review" data-drupal-link-system-path="node/27694">Clinical Trials and FDA Review</a>
|
||
</li>
|
||
<li class="last">
|
||
<a href="/research/research-resources/ncats-toolkit/toolkit-after-fda-approval" class="last" data-drupal-link-system-path="node/27692">After FDA Approval</a>
|
||
</li>
|
||
</ul>
|
||
|
||
|
||
</nav>
|
||
|
||
|
||
</div>
|
||
|
||
<section>
|
||
<div class="container news-content Advancing">
|
||
|
||
|
||
<div class="paragraph paragraph--type--body-content paragraph--view-mode--default">
|
||
|
||
|
||
<h2>About NCATS Toolkit </h2><p>The tips, strategies and many of the resources are presented from the perspective of rare disease patient group leaders with the goal of empowering these groups to actively participate in each stage of the process. The level of participation of patient groups in the therapy development process varies for many reasons, including the level of resources and experience of the group. One of the goals of NCATS Toolkit is to help even young, newly forming groups with limited resources and experience find ways to have a voice in the process. <br> </p><h3>What is the NCATS Toolkit? </h3><p>NCATS Toolkit is a website that provides rare disease patient group leaders, like you, with reliable information about the stages of the therapy development process and the ways your group can become involved. The ultimate goal is to help your group become a driving force behind the development of therapies and/or a cure for your disease(s). In fact, of the 7,000-plus recognized rare diseases, only about 5% have a <a href="https://toolkit.ncats.nih.gov/glossary/u-s-food-and-drug-administration/">U.S. Food and Drug Administration</a> (FDA)–approved treatment. Involving the patient voice in the therapy development process is recognized by the FDA, researchers and <a href="https://toolkit.ncats.nih.gov/glossary/pharmaceutical-industry/">pharmaceutical industry</a> as a way to increase the pace of therapy development for rare diseases. </p><h3>Where do I start? </h3><p><a href="https://toolkit.ncats.nih.gov/module/getting-started/">Getting Started</a> provides information that can help a patient group become involved in the therapy development process. The remaining sections provide a more in-depth look into the different stages of treatment development, including what happens after a therapy is approved by the FDA. The tips and strategies in these sections highlight ways patient groups, like your own, can add vital information and resources to the process. You may also find the <a href="https://toolkit.ncats.nih.gov/glossary/">Glossary</a> helpful. </p><p>The organization of the NCATS Toolkit may make the process of therapy development seem like a series of steps to be followed one after the other, but it is not a straight line from start to finish. In fact, at any given time, different potential therapies for your disease may be at different stages of development. Restarting the process is not uncommon, as not every promising therapy is found to be safe and effective. In addition, not every task or research tool will be needed for every disease. Therefore, we suggest skimming through the website to decide which areas may be important to the development of a therapy for your disease, and then reading those sections and their listed resources in depth. </p><h3>Do you perform research? </h3><p>NCATS Toolkit is solely an information website and is not involved in research.</p><h3>Is there information for patients and their families? </h3><p>NCATS Toolkit focuses on patient groups, and the tips and strategies were written with patient group leaders in mind. However, the information about how a therapy is developed for a rare disease may be helpful to any person or family affected by a rare disease. If you are a patient who has a genetic or rare disease or a family member or <a href="https://toolkit.ncats.nih.gov/glossary/caregiver/">caregiver</a> and would like to support the efforts for the development of a therapy for your disease, the NCATS Toolkit has two topic areas that can help get you started: “<a href="https://toolkit.ncats.nih.gov/module/getting-started/empower-your-patient-community-voice/finding-a-group/">Finding a Group</a>” and “<a href="https://toolkit.ncats.nih.gov/module/getting-started/empower-your-patient-community-voice/starting-a-patient-group/">Starting a Patient Group</a>.” In addition, NCATS has another program, the Genetic and Rare Diseases Information Center (GARD), that is directed towards individuals, families and caregivers: <a href="https://rarediseases.info.nih.gov/about-gard/pages/23/about-gard">About GARD</a>. The GARD website provides access to information about rare or genetic diseases. If you can’t find the information you need on the GARD website, you may <a href="https://rarediseases.info.nih.gov/about-gard/contact-gard">contact a GARD Information Specialist</a>, who can direct you to disease information, research studies, clinical trials, patient advocacy organizations (supportive resources), medical journal articles and genetic services.</p><h3>Where do the resources and content come from? </h3><p>The resources on the NCATS Toolkit are collected from rare disease patient groups, academia, industry and government agencies, such as the National Institutes of Health (NIH) and the FDA. The content is developed using the listed resources for that topic area and by engaging with key stakeholders at conferences and through one-on-one meetings and informal conversations. </p><p>The information and resources in the NCATS Toolkit are intended to help you better target your conversations and efforts. NCATS Toolkit is not intended to replace talking with your own Board of Directors and Medical/Scientific Advisory Boards, other patient group leaders, rare disease umbrella organizations, the FDA, researchers, industry and other stakeholders. </p><h3>Can I contribute resources?</h3><p>NCATS Toolkit welcomes your patient group to submit resources that you found helpful at any stage of the therapy development process by emailing <a href="mailto:ncatstoolkit@nih.gov">NCATS Toolkit</a>.</p><p><a href="/research/research-resources/ncats-toolkit/share-resources">Learn more</a> about our list of goals, what kinds of resources might helpful, and our review criteria for evaluating resources to be included in the NCATS Toolkit.</p><h3>What is the history of the website? </h3><p>NCATS Toolkit was created in response to requests from rare disease patient groups to develop a resource repository to help other patient groups become more involved in the therapy development process. Leading a working group of NCATS staff and various patient groups for nearly two years, NCATS Toolkit co-chairs Ronald J. Bartek (co-founder of the Friedreich’s Ataxia Research Alliance) and Annie Kennedy (senior vice president of legislation and public policy for Parent Project Muscular Dystrophy) identified and evaluated existing resources. The original NCATS Toolkit was launched September 8, 2017. In 2019, in response to feedback from patient groups, content and resources were added and the website was restructured. NCATS Toolkit was relaunched on Rare Disease Day 2020.</p><h3>What’s next for the NCATS Toolkit?</h3><p>The role of the patient groups in therapy development continues to expand. NCATS Toolkit is meant to be a living website that will add new content as it becomes available and as the therapy development process adapts to scientific advancements and regulatory changes.</p><h3>Who oversees the development of the website?</h3><p><a href="https://ncats.nih.gov/staff/sidew">Dr. Eric Sid</a> oversees the NCATS Toolkit management and development team.</p><p>Eric Sid, M.D., M.H.A. <br>Program Officer <br>National Institutes of Health <br>NCATS Division of Rare Diseases Research Innovation</p><p>Email: <a href="mailto:eric.sid@nih.gov">eric.sid@nih.gov</a></p><h3>What can the NCATS Toolkit do better?</h3><p><a href="https://docs.google.com/forms/d/e/1FAIpQLSeM-iy2blyU3174Jh5nOoIrbCaEhDVsNumhDV4MqN5sBTeirg/viewform">Provide Your Feedback </a></p><p>Please use our feedback form to submit comments on existing information or resources. You can also let us know if there are topics or areas you would like to see added. Your feedback will help us improve the NCATS Toolkit for the whole rare disease community.</p><h3>Request Assistance or Submit Resources</h3><p>You can use the <a href="https://contact.rarediseases.info.nih.gov/Gard/s/?language=en_US">contact form</a> for the GARD Information Center to reach an Information Specialist if you need help navigating the NCATS Toolkit. You can also suggest the addition of a resource your group found helpful. Or you may wish to share resources your group developed, such as a meeting agenda template or a checklist for reviewing clinical trials to post on your website. Thank you in advance for helping us keep the NCATS Toolkit up to date.</p>
|
||
|
||
</div>
|
||
|
||
|
||
|
||
<hr class="section-divider">
|
||
</div>
|
||
</section>
|
||
|
||
|
||
</div>
|
||
<div class="block block-layout-builder block-extra-field-blocknodelanding-page-new-content-moderation-control">
|
||
|
||
|
||
|
||
</div>
|
||
|
||
</div>
|
||
</div>
|
||
|
||
</div>
|
||
|
||
</article>
|
||
|
||
</div>
|
||
<div id="block-footercss" class="block block-block-content block-block-content2db33373-6fb7-49c5-be2f-83266eddea39">
|
||
|
||
|
||
|
||
|
||
<style type="text/css">
|
||
ul#superfish-navigation li { line-height:1.25; }
|
||
.region-header .block-superfish ul.sf-menu li a {padding-right:40px !important;}
|
||
@media (min-width: 1200px) {
|
||
ul.sf-menu.sf-navigation li.menuparent ul {width:280px !important;}
|
||
}
|
||
.main-content article .menu--navigation {
|
||
display:flex;
|
||
flex-wrap: nowrap;
|
||
justify-content: space-between;
|
||
align-items: stretch;
|
||
}
|
||
.main-content article .menu--navigation li {
|
||
flex: 1 1 0;
|
||
width: 0;
|
||
align-items: center;
|
||
display: flex;
|
||
justify-content: center;
|
||
}
|
||
.main-content article .menu--navigation li .dropdown-toggle {
|
||
white-space: unset;
|
||
}
|
||
.image-box img,
|
||
div.col-md-4.col-lg-2 img {
|
||
height: auto;
|
||
}
|
||
.caption-left-md {
|
||
float: left;
|
||
width: 50%;
|
||
margin: 0 1% 2% 0;
|
||
border: 1px solid #ddd;
|
||
}
|
||
.caption-left-md img {
|
||
width: 100%!important;
|
||
height: auto;
|
||
}
|
||
div.block.block-layout-builder.block-field-blocknodestafffield-divisions-office {
|
||
display: none
|
||
}
|
||
@media (max-width: 992px) {
|
||
.main-content article .menu--navigation {
|
||
flex-direction:column;
|
||
}
|
||
.main-content article .menu--navigation li {
|
||
display:inline-block;
|
||
margin:0;
|
||
padding:.25rem;
|
||
width: auto;
|
||
text-align:left;
|
||
}
|
||
.main-content article .menu--navigation li:hover, .main-content article .menu--navigation li.active {
|
||
border-bottom: 4px solid transparent !important;
|
||
}
|
||
.main-content article .menu--navigation li:hover a, .main-content article .menu--navigation li.active a {
|
||
border-bottom:4px solid #00626f !important;
|
||
padding-bottom:4px;
|
||
}
|
||
}
|
||
p.last-update-container {
|
||
font-size: 15px;
|
||
font-style: italic;
|
||
}
|
||
</style>
|
||
|
||
</div>
|
||
|
||
</div>
|
||
</div>
|
||
</div>
|
||
</div>
|
||
</main>
|
||
<div>
|
||
<div id="block-d9-subtheme-lastupdateblock" class="block block-ncats-common block-last-update-block">
|
||
|
||
|
||
<div class="container"><p class="last-update-container">Last updated on April 20, 2024</p></div>
|
||
</div>
|
||
|
||
</div>
|
||
|
||
<footer class="pt-4 footer mt-4">
|
||
<div class="container">
|
||
<div class="row">
|
||
<div class="col-12 py-4">
|
||
<img src="/sites/default/files/2023-10/ncats-logo-footer.png" class="img-fluid" alt="NCATS">
|
||
</div>
|
||
</div>
|
||
|
||
<div class="row">
|
||
<div class="container">
|
||
<div class="row justify-content-between">
|
||
<div class="col-xl-4">
|
||
<div class="pt-4">
|
||
<address>
|
||
9609 Medical Center Drive<br>
|
||
Rockville, MD 20850-9793<br>
|
||
301-594-8966<br>
|
||
<a href="mailto:ncatsinfo@nih.gov">ncatsinfo@nih.gov</a>
|
||
</address>
|
||
</div>
|
||
|
||
<div class="text-white social-links mt-4">
|
||
<h5>Connect with us</h5>
|
||
<ul id="social-media-icons" class="list-unstyled d-flex mb-4">
|
||
<li><a href="http://www.facebook.com/ncats.nih.gov" title="Visit us on Facebook" target="_blank"><i class="bi bi-facebook"></i></a></li>
|
||
<li><a href="https://www.linkedin.com/company/nih-ncats" title="Visit us on LinkedIn" target="_blank"><i class="bi bi-linkedin"></i></a></li>
|
||
<li><a href="http://www.twitter.com/ncats_nih_gov" title="Visit us on X" target="_blank"><i class="bi bi-twitter-x"></i></a></li>
|
||
<li><a href="http://www.youtube.com/user/ncatsmedia" title="View our videos on YouTube" target="_blank"><i class="bi bi-youtube"></i></a></li>
|
||
<li><a href="https://www.flickr.com/photos/64860478@N05/" target="_blank" title="View our photos on Flickr"><img src="/sites/default/files/2023-10/flickr.png" alt="View our photos on Flickr" style="height:24px;"></a></li>
|
||
</ul>
|
||
<p class="small">For <a href="http://edi.nih.gov/consulting/language-access-program/about" target="_blank">language access assistance</a>, contact the <a href="mailto:ncatsinfo@nih.gov">NCATS Public Information Officer</a></p>
|
||
</div>
|
||
|
||
</div>
|
||
<div class="col-xl-3 text-white mt-5 d-lg-none d-sm-none d-none d-xl-block">
|
||
<div id="mc_embed_shell">
|
||
|
||
<div id="mc_embed_signup">
|
||
<form action="https://nih.us5.list-manage.com/subscribe/post?u=e06bd55ccbb41f1a362306dff&id=d32fa404b4&f_id=0087b1edf0" method="post" id="mc-embedded-subscribe-form" name="mc-embedded-subscribe-form" class="validate" target="_blank">
|
||
<h5>Subscribe to NCATS News</h5>
|
||
<p>If you would like to stay up to date with the latest information from us, please sign up for NCATS News.</p>
|
||
<div id="mce-responses" class="clear">
|
||
<div class="response" id="mce-error-response" style="display: none;"></div>
|
||
<div class="response" id="mce-success-response" style="display: none;"></div>
|
||
</div>
|
||
<div class="input-group mb-3">
|
||
<input type="email" name="EMAIL" class="required email form-control" id="mce-EMAIL" required="" value="" placeholder="Enter Email" aria-label="Recipient's username" aria-describedby="button-addon2" tabindex="-1">
|
||
|
||
<div aria-hidden="true" style="position: absolute; left: -5000px;"><input type="text" name="b_e06bd55ccbb41f1a362306dff_d32fa404b4" tabindex="-1" value=""></div>
|
||
<button type="submit" name="subscribe" id="mc-embedded-subscribe" class="button btn" value="Subscribe">Submit</button>
|
||
</div>
|
||
|
||
</form>
|
||
</div>
|
||
<script type="text/javascript" src="//s3.amazonaws.com/downloads.mailchimp.com/js/mc-validate.js"></script><script type="text/javascript">(function($) {window.fnames = new Array(); window.ftypes = new Array();fnames[0]='EMAIL';ftypes[0]='email';fnames[1]='FNAME';ftypes[1]='text';fnames[2]='LNAME';ftypes[2]='text';fnames[3]='SIGNUP';ftypes[3]='text';}(jQuery));var $mcj = jQuery.noConflict(true);</script></div>
|
||
|
||
</div>
|
||
</div>
|
||
</div>
|
||
</div>
|
||
|
||
</div>
|
||
<!-- footer menu section -->
|
||
|
||
<section class="footer-menu py-3 mb-xl-0 mb-lg-0 mb-sm-5 mt-4">
|
||
<div class="container">
|
||
<div class="row">
|
||
<div class="col-12">
|
||
<ul class="list-unstyled d-xl-flex d-lg-flex d-sm-block d-block justify-content-center mb-0">
|
||
<li><a href="https://ncats.nih.gov/">NCATS Home</a></li>
|
||
<li><a href="/sitemap">Site Map</a></li>
|
||
<li><a href="/privacy">Privacy Notice</a></li>
|
||
<li><a href="/comments">Comment Policy</a></li>
|
||
<li><a href="/accessibility">Accessibility</a></li>
|
||
<li><a href="/foia">FOIA</a></li>
|
||
<li><a href="http://oig.hhs.gov/" target="_blank">OIG</a></li>
|
||
<li class="border-0"><a href="https://www.hhs.gov/vulnerability-disclosure-policy/index.html" target="_blank">HHS Vulnerability Disclosure</a></li>
|
||
</ul>
|
||
</div>
|
||
</div>
|
||
</div>
|
||
</section>
|
||
</footer>
|
||
|
||
</div>
|
||
|
||
|
||
<script type="application/json" data-drupal-selector="drupal-settings-json">{"path":{"baseUrl":"\/","pathPrefix":"","currentPath":"node\/27617","currentPathIsAdmin":false,"isFront":false,"currentLanguage":"en"},"pluralDelimiter":"\u0003","suppressDeprecationErrors":true,"ajaxPageState":{"libraries":"eJx1kFGOwzAIRC_kjb_3NBaJqeMWGwtw29y-jtTutqvsz2jmjQCJ-B20z7ZiQQ-qaOriG8K7oVSgLxiyWV7UDUS5XnyU3oCmZ3SJOREGg-TTkL95gjPcP2FxBBt3CzHrwleUzXPFhck1EEgCbdXXlV8y9dr6TFlXjE57QzkN73_cEQvrvj5Xw2qHvRYg0kUQ63G_u_EU_a9trNkyj-lNDYufQdF1vUUNu_UngYI3lssDdIKSjQ","theme":"d9_subtheme","theme_token":null},"ajaxTrustedUrl":[],"gtm":{"tagId":null,"settings":{"data_layer":"dataLayer","include_classes":false,"allowlist_classes":"","blocklist_classes":"","include_environment":false,"environment_id":"","environment_token":""},"tagIds":["GTM-55CGV66V"]},"gtag":{"tagId":"G-ZZQ8H5G8ZX","otherIds":[],"events":[],"additionalConfigInfo":[]},"data":{"extlink":{"extTarget":true,"extTargetNoOverride":false,"extNofollow":false,"extNoreferrer":true,"extFollowNoOverride":false,"extClass":"ext","extLabel":"(link is external)","extImgClass":false,"extSubdomains":true,"extExclude":"","extInclude":"","extCssExclude":"","extCssExplicit":"","extAlert":true,"extAlertText":"You are leaving the NIH website.\r\n\r\nThis external link provides additional information that is consistent with the intended purpose of this site. NIH cannot attest to the accuracy of a non-federal site.\r\n\r\nLinking to a non-federal site does not constitute an endorsement by NIH or any of its employees of the sponsors or the information and products presented on the site. You will be subject to the destination site\u0027s privacy policy when you follow the link.","mailtoClass":"mailto","mailtoLabel":"(link sends email)","extUseFontAwesome":false,"extIconPlacement":"append","extFaLinkClasses":"fa fa-external-link","extFaMailtoClasses":"fa fa-envelope-o","whitelistedDomains":[]}},"superfish":{"superfish-navigation":{"id":"superfish-navigation","sf":{"animation":{"opacity":"show","height":"show"},"speed":"fast"},"plugins":{"smallscreen":{"mode":"window_width","accordionButton":0,"title":"Navigation"},"supposition":true,"supersubs":true}}},"user":{"uid":0,"permissionsHash":"24b2a698a72e7a44d4f2d94d9880a6ba82bfbc8b8200dee23422144335254cbc"}}</script>
|
||
<script src="/core/assets/vendor/jquery/jquery.min.js?v=3.7.1"></script>
|
||
<script src="/core/assets/vendor/once/once.min.js?v=1.0.1"></script>
|
||
<script src="/core/misc/drupalSettingsLoader.js?v=10.2.3"></script>
|
||
<script src="/core/misc/drupal.js?v=10.2.3"></script>
|
||
<script src="/core/misc/drupal.init.js?v=10.2.3"></script>
|
||
<script src="/core/assets/vendor/tabbable/index.umd.min.js?v=6.2.0"></script>
|
||
<script src="https://cdn.jsdelivr.net/npm/@uswds/uswds@3.3.0/dist/js/uswds.min.js"></script>
|
||
<script src="/core/misc/progress.js?v=10.2.3"></script>
|
||
<script src="/core/assets/vendor/loadjs/loadjs.min.js?v=4.2.0"></script>
|
||
<script src="/core/misc/debounce.js?v=10.2.3"></script>
|
||
<script src="/core/misc/announce.js?v=10.2.3"></script>
|
||
<script src="/core/misc/message.js?v=10.2.3"></script>
|
||
<script src="/core/misc/ajax.js?v=10.2.3"></script>
|
||
<script src="/themes/custom/d9_subtheme/js/script.js?v=10.2.3"></script>
|
||
<script src="/modules/contrib/extlink/extlink.js?v=10.2.3"></script>
|
||
<script src="/modules/contrib/google_tag/js/gtm.js?srmo5o"></script>
|
||
<script src="/modules/contrib/google_tag/js/gtag.js?srmo5o"></script>
|
||
<script src="/modules/contrib/google_tag/js/gtag.ajax.js?srmo5o"></script>
|
||
<script src="/libraries/drupal-superfish/superfish.js?srmo5o"></script>
|
||
<script src="/libraries/drupal-superfish/jquery.hoverIntent.minified.js?srmo5o"></script>
|
||
<script src="/libraries/drupal-superfish/sfsmallscreen.js?srmo5o"></script>
|
||
<script src="/libraries/drupal-superfish/supposition.js?srmo5o"></script>
|
||
<script src="/libraries/drupal-superfish/supersubs.js?srmo5o"></script>
|
||
<script src="/modules/contrib/superfish/js/superfish.js?v=2.0"></script>
|
||
|
||
</body>
|
||
</html>
|