CINXE.COM

<!DOCTYPE HTML>     <html class="no-js" lang="en-US">  <head> <title>NHS England » Why working with patient groups is critical for providing effective treatments and services</title> <meta name="title" property="og:title" content="NHS England » Why working with patient groups is critical for providing effective treatments and services"> <script type="text/javascript"> var addthis_config = { ui_tabindex:0 } </script> <meta charset="utf-8"> <meta http-equiv="cleartype" content="on"> <meta name="description" property="og:description" content="When there are limited options available for treating a rare disease, new therapies can transform care for patients with the condition. For young children with Spinal Muscular Atrophy (SMA), a rare genetic condition that causes progressive muscle weakness and wasting, NHS England has managed to secure three treatments in less than three years, where previously […]"> <meta name="author" content="NHS England"> <meta property="og:image" content="https://www.england.nhs.uk/wp-content/themes/nhsengland/static/img/nhs-england-logo-export.png"> <meta property="og:url" content="https://www.england.nhs.uk/blog/why-working-with-patient-groups-is-critical-for-providing-effective-treatments-and-services/"> <link rel="alternate" type="application/rss+xml" title="RSS2.0" href="https://www.england.nhs.uk/feed/" /> <meta name='robots' content='max-image-preview:large' /> <link rel='stylesheet' id='wp-block-library-css' href='https://www.england.nhs.uk/wp-includes/css/dist/block-library/style.min.css?ver=6.7' type='text/css' media='all' /> <style id='co-authors-plus-coauthors-style-inline-css' type='text/css'> .wp-block-co-authors-plus-coauthors.is-layout-flow [class*=wp-block-co-authors-plus]{display:inline} </style> <style id='co-authors-plus-avatar-style-inline-css' type='text/css'> .wp-block-co-authors-plus-avatar :where(img){height:auto;max-width:100%;vertical-align:bottom}.wp-block-co-authors-plus-coauthors.is-layout-flow .wp-block-co-authors-plus-avatar :where(img){vertical-align:middle}.wp-block-co-authors-plus-avatar:is(.alignleft,.alignright){display:table}.wp-block-co-authors-plus-avatar.aligncenter{display:table;margin-inline:auto} </style> <style id='co-authors-plus-image-style-inline-css' type='text/css'> .wp-block-co-authors-plus-image{margin-bottom:0}.wp-block-co-authors-plus-image :where(img){height:auto;max-width:100%;vertical-align:bottom}.wp-block-co-authors-plus-coauthors.is-layout-flow .wp-block-co-authors-plus-image :where(img){vertical-align:middle}.wp-block-co-authors-plus-image:is(.alignfull,.alignwide) :where(img){width:100%}.wp-block-co-authors-plus-image:is(.alignleft,.alignright){display:table}.wp-block-co-authors-plus-image.aligncenter{display:table;margin-inline:auto} </style> <style id='classic-theme-styles-inline-css' type='text/css'> /*! This file is auto-generated */ .wp-block-button__link{color:#fff;background-color:#32373c;border-radius:9999px;box-shadow:none;text-decoration:none;padding:calc(.667em + 2px) calc(1.333em + 2px);font-size:1.125em}.wp-block-file__button{background:#32373c;color:#fff;text-decoration:none} </style> <style id='global-styles-inline-css' type='text/css'> :root{--wp--preset--aspect-ratio--square: 1;--wp--preset--aspect-ratio--4-3: 4/3;--wp--preset--aspect-ratio--3-4: 3/4;--wp--preset--aspect-ratio--3-2: 3/2;--wp--preset--aspect-ratio--2-3: 2/3;--wp--preset--aspect-ratio--16-9: 16/9;--wp--preset--aspect-ratio--9-16: 9/16;--wp--preset--color--black: #000000;--wp--preset--color--cyan-bluish-gray: #abb8c3;--wp--preset--color--white: #ffffff;--wp--preset--color--pale-pink: #f78da7;--wp--preset--color--vivid-red: #cf2e2e;--wp--preset--color--luminous-vivid-orange: #ff6900;--wp--preset--color--luminous-vivid-amber: #fcb900;--wp--preset--color--light-green-cyan: #7bdcb5;--wp--preset--color--vivid-green-cyan: #00d084;--wp--preset--color--pale-cyan-blue: #8ed1fc;--wp--preset--color--vivid-cyan-blue: #0693e3;--wp--preset--color--vivid-purple: #9b51e0;--wp--preset--gradient--vivid-cyan-blue-to-vivid-purple: linear-gradient(135deg,rgba(6,147,227,1) 0%,rgb(155,81,224) 100%);--wp--preset--gradient--light-green-cyan-to-vivid-green-cyan: linear-gradient(135deg,rgb(122,220,180) 0%,rgb(0,208,130) 100%);--wp--preset--gradient--luminous-vivid-amber-to-luminous-vivid-orange: linear-gradient(135deg,rgba(252,185,0,1) 0%,rgba(255,105,0,1) 100%);--wp--preset--gradient--luminous-vivid-orange-to-vivid-red: linear-gradient(135deg,rgba(255,105,0,1) 0%,rgb(207,46,46) 100%);--wp--preset--gradient--very-light-gray-to-cyan-bluish-gray: linear-gradient(135deg,rgb(238,238,238) 0%,rgb(169,184,195) 100%);--wp--preset--gradient--cool-to-warm-spectrum: linear-gradient(135deg,rgb(74,234,220) 0%,rgb(151,120,209) 20%,rgb(207,42,186) 40%,rgb(238,44,130) 60%,rgb(251,105,98) 80%,rgb(254,248,76) 100%);--wp--preset--gradient--blush-light-purple: linear-gradient(135deg,rgb(255,206,236) 0%,rgb(152,150,240) 100%);--wp--preset--gradient--blush-bordeaux: linear-gradient(135deg,rgb(254,205,165) 0%,rgb(254,45,45) 50%,rgb(107,0,62) 100%);--wp--preset--gradient--luminous-dusk: linear-gradient(135deg,rgb(255,203,112) 0%,rgb(199,81,192) 50%,rgb(65,88,208) 100%);--wp--preset--gradient--pale-ocean: linear-gradient(135deg,rgb(255,245,203) 0%,rgb(182,227,212) 50%,rgb(51,167,181) 100%);--wp--preset--gradient--electric-grass: linear-gradient(135deg,rgb(202,248,128) 0%,rgb(113,206,126) 100%);--wp--preset--gradient--midnight: linear-gradient(135deg,rgb(2,3,129) 0%,rgb(40,116,252) 100%);--wp--preset--font-size--small: 13px;--wp--preset--font-size--medium: 20px;--wp--preset--font-size--large: 36px;--wp--preset--font-size--x-large: 42px;--wp--preset--spacing--20: 0.44rem;--wp--preset--spacing--30: 0.67rem;--wp--preset--spacing--40: 1rem;--wp--preset--spacing--50: 1.5rem;--wp--preset--spacing--60: 2.25rem;--wp--preset--spacing--70: 3.38rem;--wp--preset--spacing--80: 5.06rem;--wp--preset--shadow--natural: 6px 6px 9px rgba(0, 0, 0, 0.2);--wp--preset--shadow--deep: 12px 12px 50px rgba(0, 0, 0, 0.4);--wp--preset--shadow--sharp: 6px 6px 0px rgba(0, 0, 0, 0.2);--wp--preset--shadow--outlined: 6px 6px 0px -3px rgba(255, 255, 255, 1), 6px 6px rgba(0, 0, 0, 1);--wp--preset--shadow--crisp: 6px 6px 0px rgba(0, 0, 0, 1);}:where(.is-layout-flex){gap: 0.5em;}:where(.is-layout-grid){gap: 0.5em;}body .is-layout-flex{display: flex;}.is-layout-flex{flex-wrap: wrap;align-items: center;}.is-layout-flex > :is(*, div){margin: 0;}body .is-layout-grid{display: grid;}.is-layout-grid > :is(*, div){margin: 0;}:where(.wp-block-columns.is-layout-flex){gap: 2em;}:where(.wp-block-columns.is-layout-grid){gap: 2em;}:where(.wp-block-post-template.is-layout-flex){gap: 1.25em;}:where(.wp-block-post-template.is-layout-grid){gap: 1.25em;}.has-black-color{color: var(--wp--preset--color--black) !important;}.has-cyan-bluish-gray-color{color: var(--wp--preset--color--cyan-bluish-gray) !important;}.has-white-color{color: var(--wp--preset--color--white) !important;}.has-pale-pink-color{color: var(--wp--preset--color--pale-pink) !important;}.has-vivid-red-color{color: var(--wp--preset--color--vivid-red) !important;}.has-luminous-vivid-orange-color{color: var(--wp--preset--color--luminous-vivid-orange) !important;}.has-luminous-vivid-amber-color{color: var(--wp--preset--color--luminous-vivid-amber) !important;}.has-light-green-cyan-color{color: var(--wp--preset--color--light-green-cyan) !important;}.has-vivid-green-cyan-color{color: var(--wp--preset--color--vivid-green-cyan) !important;}.has-pale-cyan-blue-color{color: var(--wp--preset--color--pale-cyan-blue) !important;}.has-vivid-cyan-blue-color{color: var(--wp--preset--color--vivid-cyan-blue) !important;}.has-vivid-purple-color{color: var(--wp--preset--color--vivid-purple) !important;}.has-black-background-color{background-color: var(--wp--preset--color--black) !important;}.has-cyan-bluish-gray-background-color{background-color: var(--wp--preset--color--cyan-bluish-gray) !important;}.has-white-background-color{background-color: var(--wp--preset--color--white) !important;}.has-pale-pink-background-color{background-color: var(--wp--preset--color--pale-pink) !important;}.has-vivid-red-background-color{background-color: var(--wp--preset--color--vivid-red) !important;}.has-luminous-vivid-orange-background-color{background-color: var(--wp--preset--color--luminous-vivid-orange) !important;}.has-luminous-vivid-amber-background-color{background-color: var(--wp--preset--color--luminous-vivid-amber) !important;}.has-light-green-cyan-background-color{background-color: var(--wp--preset--color--light-green-cyan) !important;}.has-vivid-green-cyan-background-color{background-color: var(--wp--preset--color--vivid-green-cyan) !important;}.has-pale-cyan-blue-background-color{background-color: var(--wp--preset--color--pale-cyan-blue) !important;}.has-vivid-cyan-blue-background-color{background-color: var(--wp--preset--color--vivid-cyan-blue) !important;}.has-vivid-purple-background-color{background-color: var(--wp--preset--color--vivid-purple) !important;}.has-black-border-color{border-color: var(--wp--preset--color--black) !important;}.has-cyan-bluish-gray-border-color{border-color: var(--wp--preset--color--cyan-bluish-gray) !important;}.has-white-border-color{border-color: var(--wp--preset--color--white) !important;}.has-pale-pink-border-color{border-color: var(--wp--preset--color--pale-pink) !important;}.has-vivid-red-border-color{border-color: var(--wp--preset--color--vivid-red) !important;}.has-luminous-vivid-orange-border-color{border-color: var(--wp--preset--color--luminous-vivid-orange) !important;}.has-luminous-vivid-amber-border-color{border-color: var(--wp--preset--color--luminous-vivid-amber) !important;}.has-light-green-cyan-border-color{border-color: var(--wp--preset--color--light-green-cyan) !important;}.has-vivid-green-cyan-border-color{border-color: var(--wp--preset--color--vivid-green-cyan) !important;}.has-pale-cyan-blue-border-color{border-color: var(--wp--preset--color--pale-cyan-blue) !important;}.has-vivid-cyan-blue-border-color{border-color: var(--wp--preset--color--vivid-cyan-blue) !important;}.has-vivid-purple-border-color{border-color: var(--wp--preset--color--vivid-purple) !important;}.has-vivid-cyan-blue-to-vivid-purple-gradient-background{background: var(--wp--preset--gradient--vivid-cyan-blue-to-vivid-purple) !important;}.has-light-green-cyan-to-vivid-green-cyan-gradient-background{background: var(--wp--preset--gradient--light-green-cyan-to-vivid-green-cyan) !important;}.has-luminous-vivid-amber-to-luminous-vivid-orange-gradient-background{background: var(--wp--preset--gradient--luminous-vivid-amber-to-luminous-vivid-orange) !important;}.has-luminous-vivid-orange-to-vivid-red-gradient-background{background: var(--wp--preset--gradient--luminous-vivid-orange-to-vivid-red) !important;}.has-very-light-gray-to-cyan-bluish-gray-gradient-background{background: var(--wp--preset--gradient--very-light-gray-to-cyan-bluish-gray) !important;}.has-cool-to-warm-spectrum-gradient-background{background: var(--wp--preset--gradient--cool-to-warm-spectrum) !important;}.has-blush-light-purple-gradient-background{background: var(--wp--preset--gradient--blush-light-purple) !important;}.has-blush-bordeaux-gradient-background{background: var(--wp--preset--gradient--blush-bordeaux) !important;}.has-luminous-dusk-gradient-background{background: var(--wp--preset--gradient--luminous-dusk) !important;}.has-pale-ocean-gradient-background{background: var(--wp--preset--gradient--pale-ocean) !important;}.has-electric-grass-gradient-background{background: var(--wp--preset--gradient--electric-grass) !important;}.has-midnight-gradient-background{background: var(--wp--preset--gradient--midnight) !important;}.has-small-font-size{font-size: var(--wp--preset--font-size--small) !important;}.has-medium-font-size{font-size: var(--wp--preset--font-size--medium) !important;}.has-large-font-size{font-size: var(--wp--preset--font-size--large) !important;}.has-x-large-font-size{font-size: var(--wp--preset--font-size--x-large) !important;} :where(.wp-block-post-template.is-layout-flex){gap: 1.25em;}:where(.wp-block-post-template.is-layout-grid){gap: 1.25em;} :where(.wp-block-columns.is-layout-flex){gap: 2em;}:where(.wp-block-columns.is-layout-grid){gap: 2em;} :root :where(.wp-block-pullquote){font-size: 1.5em;line-height: 1.6;} </style> <link rel='stylesheet' id='contact-form-7-css' href='https://www.england.nhs.uk/wp-content/plugins/contact-form-7/includes/css/styles.css?ver=5.9.8' type='text/css' media='all' /> <link rel='stylesheet' id='jquery-ui-css' href='https://www.england.nhs.uk/wp-content/themes/nhsengland/static//lib/jquery-ui/themes/smoothness/jquery-ui.css?ver=6.7' type='text/css' media='screen' /> <link rel='stylesheet' id='main-css' href='https://www.england.nhs.uk/wp-content/themes/nhsengland/static/main-1ce80937921c73ff41efd2f36eb817fdc2d34e1e.min.css?ver=6.7' type='text/css' media='screen' /> <link rel='stylesheet' id='print-css' href='https://www.england.nhs.uk/wp-content/themes/nhsengland/static/print-fe4e4682bcb96cc3674bd9e62072e3d81c0553ab.min.css?ver=6.7' type='text/css' media='print' /> <meta name="viewport" content="width=device-width, initial-scale=1.0"> <link rel="dns-prefetch" href="//fast.fonts.com"> <link rel="dns-prefetch" href="//www.google-analytics.com"> <link rel="shortcut icon" href="https://www.england.nhs.uk/wp-content/themes/nhsengland/static/img/favicon.ico" type="image/x-icon"> <link rel="apple-touch-icon-precomposed" sizes="180x180" href="https://www.england.nhs.uk/wp-content/themes/nhsengland/static/img/apple-touch-icon-180x180.png"> <link rel="apple-touch-icon-precomposed" sizes="152x152" href="https://www.england.nhs.uk/wp-content/themes/nhsengland/static/img/apple-touch-icon-152x152.png"> <link rel="apple-touch-icon-precomposed" sizes="120x120" href="https://www.england.nhs.uk/wp-content/themes/nhsengland/static/img/apple-touch-icon-120x120.png"> <link rel="apple-touch-icon-precomposed" sizes="76x76" href="https://www.england.nhs.uk/wp-content/themes/nhsengland/static/img/apple-touch-icon-76x76.png"> <link rel="apple-touch-icon-precomposed" href="https://www.england.nhs.uk/wp-content/themes/nhsengland/static/img/apple-touch-icon.png"> <script type="text/javascript" src="https://www.england.nhs.uk/wp-content/themes/nhsengland/static/lib/jquery.min.js?ver=6.7" id="jquery-js"></script> <script type="text/javascript" src="https://www.england.nhs.uk/wp-content/themes/nhsengland/static/lib/modernizr.min.js?ver=6.7" id="modernizr-js"></script> <script type="text/javascript" src="https://cc.cdn.civiccomputing.com/8/cookieControl-8.x.min.js?ver=6.7" id="cookieControl-js"></script> <link rel="canonical" href="https://www.england.nhs.uk/blog/why-working-with-patient-groups-is-critical-for-providing-effective-treatments-and-services/" /> <style type="text/css"> .header.nhs-e { background: #005eb8; } .nhs-e.top-nav-container { background: #0072ce; } .nhs-e.navigation li > a:hover, .nhs-e.navigation li > a.open, .nhs-e.navigation li.current-menu-item > a, .nhs-e.navigation li.current-menu-parent > a, .nhs-e.navigation li.current_page_item > a, .nhs-e.navigation li.current-page-ancestor > a { background: #0072ce; } </style> <style type="text/css" id="wp-custom-css"> /*@media print { .rc-anchor, #rc-anchor-alert, .grecaptcha-badge { display: none !important; } }*/ </style> </head> <body class="blog-template-default single single-blog postid-199798 group"> <div class="skip-link"> <a href="#main-content" tabindex="1">Skip to main content</a> </div> <div class="cookie-banner-container" style="display: none"> <div class="cookie-banner row"> <h1>Cookies on the NHS England website</h1> <div class="rich-text"> <p>We’ve put some small files called cookies on your device to make our site work.</p> <p>We’d also like to use analytics cookies. These send information about how our site is used to a service called Google Analytics. We use this information to improve our site.</p> <p>Let us know if this is OK. We’ll use a cookie to save your choice. You can <a id="nhsuk-cookie-banner__link" tabindex="1" href="https://www.england.nhs.uk/privacy-policy/">read more about our cookies</a> before you choose.</p> <p> </p> </div> <p> <button class="preferences">Change my preferences</button> <button class="ok">I'm OK with analytics cookies</button> </p> </div> </div> <div class="nhs-e top-nav-container group"> <div class="row group"> <nav class="secondary-navigation" role="navigation"> <ul id="menu-top-header-navigation" class="nav-menu"><li id="menu-item-74795" class="menu-item menu-item-type-custom menu-item-object-custom menu-item-home menu-item-74795"><a href="https://www.england.nhs.uk/">Home</a></li> <li id="menu-item-74797" class="menu-item menu-item-type-custom menu-item-object-custom menu-item-74797"><a href="https://www.england.nhs.uk/news/">News</a></li> <li id="menu-item-74798" class="menu-item menu-item-type-custom menu-item-object-custom menu-item-74798"><a href="https://www.england.nhs.uk/publication/">Publications</a></li> <li id="menu-item-74799" class="menu-item menu-item-type-custom menu-item-object-custom menu-item-74799"><a href="https://www.england.nhs.uk/statistics/">Statistics</a></li> <li id="menu-item-85510" class="menu-item menu-item-type-post_type menu-item-object-page menu-item-85510"><a href="https://www.england.nhs.uk/blogs/">Blogs</a></li> <li id="menu-item-74802" class="menu-item menu-item-type-custom menu-item-object-custom menu-item-74802"><a href="https://www.england.nhs.uk/events/">Events</a></li> <li id="menu-item-74803" class="menu-item menu-item-type-custom menu-item-object-custom menu-item-74803"><a href="https://www.england.nhs.uk/contact-us/">Contact us</a></li> </ul> </nav> </div> </div> <header class="nhs-e header group" role="banner"> <div class="row group"> <div class="nhs-e logo"> <a href="https://www.england.nhs.uk"> <img src="https://www.england.nhs.uk/wp-content/themes/nhsengland/static/img/nhs-england-white.svg" alt="NHS England" /> </a> </div> <div class="nhs-e header-search"> <form role="search" id="search-form" class="search-form" method="get" action="https://www.england.nhs.uk"> <label class="hide" for="s" id="search-label">Search</label> <input type="search" name="s" aria-labelledby="search-label" class="search-input"> <label class="hide" for="search" id="search-button">Search</label> <input type="submit" id="search" aria-labelledby="search-button" value="Search" class="button"> </form> </div> </div> <div class="nhs-e nav-container group"> <button type="button" href="#" class="nav-toggle" id="js-nav-toggle"> <svg xmlns="http://www.w3.org/2000/svg" viewBox="0 0 18 18"><path class="menu-icon" fill="#ffffff" d="M.1 3.6C0 2.7 0 1.7.1.7.1.3.5 0 1 0h16.1c.5 0 .9.3.9.7.1 1 .1 1.9 0 2.9 0 .4-.4.7-.9.7H1c-.5 0-.9-.3-.9-.7zm.9 7.6c-.5 0-.9-.3-.9-.7-.1-1-.1-2 0-2.9 0-.4.4-.7.9-.7h16.1c.5 0 .9.3.9.7.1 1 .1 1.9 0 2.9 0 .4-.4.7-.9.7H1zM1 18c-.5 0-.9-.3-.9-.7-.1-1-.1-1.9 0-2.9 0-.4.4-.7.9-.7h16.1c.5 0 .9.3.9.7.1 1 .1 1.9 0 2.9 0 .4-.4.7-.9.7H1z"/></svg> <span>Menu</span> </button> <nav class="nhs-e navigation group" role="navigation"> <ul id="menu-secondary-nav" class="nav-menu"><li id="menu-item-85700" class="menu-item menu-item-type-post_type menu-item-object-page menu-item-85700"><a href="https://www.england.nhs.uk/about/" title="About NHS England">About us</a></li> <li id="menu-item-85701" class="menu-item menu-item-type-post_type menu-item-object-page menu-item-85701"><a href="https://www.england.nhs.uk/ourwork/">Our work</a></li> <li id="menu-item-85755" class="menu-item menu-item-type-post_type menu-item-object-page menu-item-85755"><a href="https://www.england.nhs.uk/ourwork/commissioning2/">Commissioning</a></li> <li id="menu-item-93612" class="menu-item menu-item-type-post_type menu-item-object-page menu-item-93612"><a href="https://www.england.nhs.uk/get-involved/">Get involved</a></li> </ul> </nav> </div> </header> <main class="main group" role="main"> <div id="chat" class="intelecomchat"> </div> <div class="row"> <div class="single-post"> <div class="single-post-article group" id="main-content"> <header> <h1>Blog</h1> </header> <article class="post group"> <header> <h2>Why working with patient groups is critical for providing effective treatments and services</h2> <div class="entry-meta group"> <time class="post-meta" datetime="2022-08-22"> <svg xmlns="http://www.w3.org/2000/svg" viewBox="0 0 16 16"><path class="icon" d="M15.47 3v11.61a.8.8 0 0 1-.63.78A36 36 0 0 1 8 16a36 36 0 0 1-6.83-.6.8.8 0 0 1-.63-.78V3a.8.8 0 0 1 .63-.78c.05 0 .6-.12 1.5-.23v.72A1.35 1.35 0 0 0 4.09 4a1.08 1.08 0 0 1-.35-.8V1.07a1.07 1.07 0 1 1 2.13 0v.58h3.2v1.02A1.35 1.35 0 0 0 10.49 4a1.08 1.08 0 0 1-.35-.8V1.07a1.07 1.07 0 1 1 2.13 0v.75c1.53.17 2.48.35 2.57.37a.8.8 0 0 1 .63.81zm-1.6 11V6.4H2.14V14a36.28 36.28 0 0 0 5.86.48 36.28 36.28 0 0 0 5.87-.48zM3.74 12.8v-2.14h2.13v2.14zm5.33-3.2H6.94V7.46h2.13zm-2.13 3.2v-2.14h2.13v2.14zm5.33-3.2h-2.13V7.46h2.13zm-2.13 3.2v-2.14h2.13v2.14z"/></svg> 22 August 2022 </time> <p class="post-author"> <svg xmlns="http://www.w3.org/2000/svg" viewBox="0 0 16 16"><path class="icon" d="M3.74 11.2A24.31 24.31 0 0 1 .39 11a.43.43 0 0 1-.3-.2.38.38 0 0 1-.09-.34c.22-.95.17-1.27 1.07-1.9s1.8-1 2.12-1.37A.78.78 0 0 0 3.27 6a3.17 3.17 0 0 1-.78-3.58A2.48 2.48 0 0 1 4.8 1.07a2.48 2.48 0 0 1 2.32 1.36 3 3 0 0 1 .27 1.2 3.56 3.56 0 0 0-2.82 2 4.32 4.32 0 0 0 .72 4.6c-.15.1-.33.2-.5.3-.34.22-.7.42-1.05.67zm-.15 3.55a.43.43 0 0 1-.3-.2.38.38 0 0 1-.07-.35c.22-.95.17-1.27 1.07-1.9s1.8-1 2.12-1.37a.78.78 0 0 0 .07-1.18 3.17 3.17 0 0 1-.78-3.58A2.48 2.48 0 0 1 8 4.8a2.48 2.48 0 0 1 2.32 1.37 3.17 3.17 0 0 1-.78 3.58.78.78 0 0 0 .07 1.18c.32.35 1.32.8 2.12 1.37s.85.95 1.07 1.9a.44.44 0 0 1-.07.35.43.43 0 0 1-.3.2 31.21 31.21 0 0 1-4.43.18 30.9 30.9 0 0 1-4.41-.18zm12.33-3.93a.43.43 0 0 1-.3.2 24.67 24.67 0 0 1-3.35.18c-.35-.25-.72-.45-1-.65a5.21 5.21 0 0 1-.52-.3 4.27 4.27 0 0 0 .7-4.6 3.52 3.52 0 0 0-2.8-2 3 3 0 0 1 .27-1.2 2.48 2.48 0 0 1 2.28-1.38 2.48 2.48 0 0 1 2.32 1.37A3.17 3.17 0 0 1 12.74 6a.78.78 0 0 0 .06 1.2c.32.35 1.32.8 2.12 1.37s.85.95 1.07 1.9a.44.44 0 0 1-.07.34z"/></svg> <a href="https://www.england.nhs.uk/author/fiona-marley/" title="Posts by Fiona Marley" class="author url fn" rel="author">Fiona Marley</a> and <a href="https://www.england.nhs.uk/author/liz-ryburn/" title="Posts by Liz Ryburn" class="author url fn" rel="author">Liz Ryburn</a> </p> <ul class="topics"> <li><a href="https://www.england.nhs.uk/blog/?filter-category=cyp" rel="tag">Children and young people</a></li><li><a href="https://www.england.nhs.uk/blog/?filter-category=comm" rel="tag">Commissioning</a></li><li><a href="https://www.england.nhs.uk/blog/?filter-category=get-involved" rel="tag">Get involved</a></li><li><a href="https://www.england.nhs.uk/blog/?filter-category=ltc" rel="tag">Long term conditions</a></li> </ul> </div> </header> <div class="rich-text"> <p><strong>When there are limited options available for treating a rare disease, new therapies can transform care for patients with the condition. For young children with Spinal Muscular Atrophy (SMA), a rare genetic condition that causes progressive muscle weakness and wasting, NHS England has managed to secure three treatments in less than three years, where previously none were available. Here, we are keen to share some of the lessons learned along the way, working together with patient groups to maximise the impact for those affected.</strong></p> <p>In 2019, there were no NHS-approved treatments for children with SMA. However, later that year, after working with NICE and patient groups, such as SMA UK, NHS England was able to successfully negotiate a deal to make a new treatment, nusinersen (also known as Spinraza®) available for patients.</p> <p>The deal, a Managed Access Agreement (MAA), stipulated a time-limited period of funding, while collecting further evidence on effectiveness of the treatment. A model that would bring the treatment to patients faster and without delay, with approval from patient groups as signatories on the agreement.</p> <p>This agreement helped to pave the way for two more innovative treatments, onasemnogene abeparvovec (Zolgensma®) and risdiplam (Evrysdi®), to be secured for NHS patients in March and then November 2021. At the time of approval, Zolgensma®, a cutting-edge gene therapy, was internationally reported to be ‘the most expensive drug in the world’.</p> <p>Thanks to the close working of commissioning experts with those impacted by the condition, a functioning specialised service was rapidly put into place for patients across England. Zolgensma® has now been given to nearly 70 children with dozens more children with SMA expected to benefit from the gene therapy every year.</p> <p>Working effectively with patient groups, it was possible to develop a line of communication between NHS England and those affected by SMA, to ensure services would meet the demands of the SMA community while also supporting effective utilisation of NHS resources and clinical expertise.</p> <p>Regular meetings with patient representatives gave the opportunity to pass on frank advice on the commissioned services from families of children who have SMA and adults who have SMA. These open and candid discussions also enabled questions from the community about the treatments and how the MAA would work to be answered – or explored further when and where necessary</p> <p>One member of the SMA UK patient group, David from Northamptonshire, said his family felt “reassured that our concerns about our adult son’s difficulties gaining access to treatment were clearly articulated to the commissioners, which in turn enabled all parties to work together to try to address the block we were experiencing”. David added, “having the opportunity to input via the patient group gave those working to set up the service a clearer understanding of the issues families and adults faced. It also highlighted what needed to be done to make sure the arrangements would work so treatments would reach whoever was eligible in the SMA community.”</p> <p>The results of these conversations – about all three treatments and commercial agreements – gave patient groups the information needed to produce valuable materials for the patient community on the new drugs, posting the details on their websites and receiving thousands of views.</p> <p>Working together, the information was shared far more widely than it would have if left to one organisation alone. For example, an SMA UK webinar on Zolgensma® in 2021, inviting NHS experts to discuss the new treatment, likely reached all eligible families in the country at the time.</p> <p>With continued interest in innovative and high-cost treatments, it is essential that service delivery, communications and eligibility assessments meet the needs of providers and patients. This collaborative model, engaging the patient community from the start, supported delivery of equitable treatment provision and well-timed, appropriate information via community channels that was better able to reach those that needed it.</p> <p>Involving patient groups is essential to developing effective NHS services. The lessons learned working with the SMA community reinforce the value of working relationships and have contributed to improving patient outcomes for hundreds of children with SMA in England.</p> <p>NHS England works with a range of Patient and Public Voice (PPV) partners at all levels on top of collaborations with organisations like SMA UK. The latest list of <a href="https://www.england.nhs.uk/get-involved/get-involved/opportunities/">opportunities to get involved can be found on the NHS England website</a>.</p> </div> <div class="author-meta group"> <div class="avatar-photo"> <img width="140" height="140" src="https://www.england.nhs.uk/wp-content/uploads/2022/08/fiona-marley-150x150.jpg" class="avatar avatar-140 photo wp-post-image" alt="Fiona Marley" decoding="async" srcset="https://www.england.nhs.uk/wp-content/uploads/2022/08/fiona-marley-150x150.jpg 150w, https://www.england.nhs.uk/wp-content/uploads/2022/08/fiona-marley.jpg 234w" sizes="(max-width: 140px) 100vw, 140px" /> </div> <div class="biography"> <h5><a href="https://www.england.nhs.uk/author/fiona-marley/" title="Posts by Fiona Marley" class="author url fn" rel="author">Fiona Marley</a></h5> <div class="rich-text"> <p>Fiona Marley heads up the Highly Specialised Commissioning Team in NHS England and has worked for the NHS since 1991 in a number of roles, including in primary care and health improvement.</p> <p>Fiona’s team commissions over 80 highly specialised services, which are typically delivered in small numbers of expert centres. The portfolio includes a number of transplant services, services for patients with rare cancers and services for patients who have rare diseases. Commissioning services in this way ensures that patients have: excellent clinical outcomes; equitable access to services regardless of where they live; and access to clinical trials. In 2021, Fiona commissioned the service to deliver onasemnogene abeparvovec for Spinal Muscular Atrophy.</p> </div> </div> </div> <div class="author-meta group"> <div class="avatar-photo"> <img width="140" height="140" src="https://www.england.nhs.uk/wp-content/uploads/2022/08/liz-ryburn-150x150.jpg" class="avatar avatar-140 photo wp-post-image" alt="Liz Ryburn" decoding="async" loading="lazy" srcset="https://www.england.nhs.uk/wp-content/uploads/2022/08/liz-ryburn-150x150.jpg 150w, https://www.england.nhs.uk/wp-content/uploads/2022/08/liz-ryburn.jpg 234w" sizes="auto, (max-width: 140px) 100vw, 140px" /> </div> <div class="biography"> <h5><a href="https://www.england.nhs.uk/author/liz-ryburn/" title="Posts by Liz Ryburn" class="author url fn" rel="author">Liz Ryburn</a></h5> <div class="rich-text"> <p>Liz has been Support Team Manager at Spinal Muscular Atrophy UK (SMA UK) since May 2011. She has a long professional background in social work and disability rights/services in both the voluntary and statutory sector in the UK and New Zealand. She now works on Information Coordination at SMA UK – a patient group/charity that has been providing information and support and funding and supporting research-related initiatives since 1985. The charity actively advocates tor access to treatments and better health and social care with and for the SMA community. </p> </div> </div> </div> <div class="metabar metabar--footer"> <div class="article-date"> <div class="article-date__pub"> <span class="article-date__pub label">Date published</span>: 22 August, 2022 </div> <div class="article-date__last"> <span class="article-date__last label">Date last updated</span>: 22 August, 2022 </div> </div> </div> <div class="entry-meta entry-meta--topics"> <h4>Topics</h4> <ul class="topics"><li><a href="https://www.england.nhs.uk/blog/?filter-category=cyp" rel="tag">Children and young people</a></li><li><a href="https://www.england.nhs.uk/blog/?filter-category=comm" rel="tag">Commissioning</a></li><li><a href="https://www.england.nhs.uk/blog/?filter-category=get-involved" rel="tag">Get involved</a></li><li><a href="https://www.england.nhs.uk/blog/?filter-category=ltc" rel="tag">Long term conditions</a></li></ul> </div> </article> <div class="comment-area"> </div> </div> <aside class="sidebar blog-sidebar group" role="complementary"> <div class="blog-sidebar--related-posts"> <h2>Latest posts</h2> <ul class="related-posts"> <li> <a href="https://www.england.nhs.uk/blog/early-diagnosis-saved-my-life/">Early diagnosis saved my life</a> </li> <li> <a href="https://www.england.nhs.uk/blog/millions-of-people-have-diabetes-but-how-much-do-you-know-about-it/">Millions of people have diabetes, but how much do you know about it?</a> </li> <li> <a href="https://www.england.nhs.uk/blog/working-with-people-and-communities-to-improve-general-practice-in-primary-care/">Working with people and communities to improve general practice in primary care</a> </li> <li> <a href="https://www.england.nhs.uk/blog/improving-health-equity-through-the-london-inspire-programme/">Improving health equity through the London Inspire Programme</a> </li> <li> <a href="https://www.england.nhs.uk/blog/mental-health-in-the-workplace-caring-for-staff/">Mental health in the workplace – caring for staff</a> </li> </ul> </div> </aside> </div> </div> </main> <footer class="footer group" role="contentinfo"> <div class="row"> <div class="footer-links"> <nav class="footer-navigation"> <ul id="menu-footer" class="menu"><li id="menu-item-29149" class="menu-item menu-item-type-custom menu-item-object-custom menu-item-29149"><a href="https://www.england.nhs.uk/terms-and-conditions-2/">Terms and conditions</a></li> <li id="menu-item-29151" class="menu-item menu-item-type-custom menu-item-object-custom menu-item-29151"><a href="https://www.england.nhs.uk/privacy-policy/">Privacy and cookies</a></li> <li id="menu-item-29152" class="menu-item menu-item-type-custom menu-item-object-custom menu-item-29152"><a href="https://www.england.nhs.uk/terms-and-conditions/comment-policy/">Social media and comment moderation</a></li> <li id="menu-item-159089" class="menu-item menu-item-type-post_type menu-item-object-page menu-item-159089"><a href="https://www.england.nhs.uk/contact-us/feedback/">Website feedback</a></li> <li id="menu-item-29153" class="menu-item menu-item-type-custom menu-item-object-custom menu-item-29153"><a href="https://www.england.nhs.uk/accessibility/">Accessibility statement</a></li> <li id="menu-item-74806" class="menu-item menu-item-type-custom menu-item-object-custom menu-item-74806"><a href="http://www.nationalarchives.gov.uk/doc/open-government-licence/version/3/">Open Government Licence v3.0</a></li> </ul> </nav> <div class="social-networks"> <ul> <li> <a href="https://www.england.nhs.uk/email-bulletins/" title="Sign up to our email bulletins" target="_blank"> <span class="hide">Sign up to our email bulletins</span> </a> </li> <li> <a href="http://www.twitter.com/NHSEngland" title="Follow us on X" target="_blank"> <span class="hide">Follow us on X</span> </a> </li> <li> <a href="https://www.facebook.com/NHSEngland/" title="Follow us on Facebook" target="_blank"> <span class="hide">Follow us on Facebook</span> </a> </li> <li> <a href="https://www.instagram.com/nhsengland" title="Find us on Instagram" target="_blank"> <span class="hide">Find us on Instagram</span> </a> </li> <li> <a href="https://www.linkedin.com/company/nhsengland" title="Visit us on LinkedIn" target="_blank"> <span class="hide">Visit us on LinkedIn</span> </a> </li> <li> <a href="http://www.youtube.com/thenhsengland" title="Watch videos on YouTube" target="_blank"> <span class="hide">Watch videos on YouTube</span> </a> </li> </ul> </div> </div> <div class="footer-logo"> <a href="https://www.nhs.uk/" title="NHS website"> <img src="https://www.england.nhs.uk/wp-content/themes/nhsengland/static/img/nhs-uk-logo.svg" alt="NHS website"> </a> </div> </div> <div id="__ba_panel"></div> </footer> <script type="text/javascript" src="https://www.england.nhs.uk/wp-includes/js/dist/hooks.min.js?ver=4d63a3d491d11ffd8ac6" id="wp-hooks-js"></script> <script type="text/javascript" src="https://www.england.nhs.uk/wp-includes/js/dist/i18n.min.js?ver=5e580eb46a90c2b997e6" id="wp-i18n-js"></script> <script type="text/javascript" id="wp-i18n-js-after"> /* <![CDATA[ */ wp.i18n.setLocaleData( { 'text direction\u0004ltr': [ 'ltr' ] } ); /* ]]> */ </script> <script type="text/javascript" src="https://www.england.nhs.uk/wp-content/plugins/contact-form-7/includes/swv/js/index.js?ver=5.9.8" id="swv-js"></script> <script type="text/javascript" id="contact-form-7-js-extra"> /* <![CDATA[ */ var wpcf7 = {"api":{"root":"https:\/\/www.england.nhs.uk\/wp-json\/","namespace":"contact-form-7\/v1"}}; /* ]]> */ </script> <script type="text/javascript" src="https://www.england.nhs.uk/wp-content/plugins/contact-form-7/includes/js/index.js?ver=5.9.8" id="contact-form-7-js"></script> <script type="text/javascript" src="https://www.england.nhs.uk/wp-content/plugins/dxw-comment-length-limiter/assets/dxw-comment-length-limiter.js?ver=6.7" id="dxw-comment-length-limiter-js"></script> <script type="text/javascript" id="main-js-extra"> /* <![CDATA[ */ var cookieControlSettings = {"apiKey":"90adfdad163e4c52a4794c7aa4ea95a84732ae3f","product":"PRO_MULTISITE"}; /* ]]> */ </script> <script type="text/javascript" src="https://www.england.nhs.uk/wp-content/themes/nhsengland/static/main-b952ae9fc371b7f02c44521aacc475379c82b1ca.min.js?ver=6.7" id="main-js"></script> <script type="text/javascript" src="https://www.england.nhs.uk/wp-content/plugins/page-links-to/dist/new-tab.js?ver=3.3.7" id="page-links-to-js"></script> <script type="text/javascript" src="https://www.google.com/recaptcha/api.js?render=6Ld9JegkAAAAAEzna4HCHXBNyzb5k82ivMDZLstF&ver=3.0" id="google-recaptcha-js"></script> <script type="text/javascript" src="https://www.england.nhs.uk/wp-includes/js/dist/vendor/wp-polyfill.min.js?ver=3.15.0" id="wp-polyfill-js"></script> <script type="text/javascript" id="wpcf7-recaptcha-js-extra"> /* <![CDATA[ */ var wpcf7_recaptcha = {"sitekey":"6Ld9JegkAAAAAEzna4HCHXBNyzb5k82ivMDZLstF","actions":{"homepage":"homepage","contactform":"contactform"}}; /* ]]> */ </script> <script type="text/javascript" src="https://www.england.nhs.uk/wp-content/plugins/contact-form-7/modules/recaptcha/index.js?ver=5.9.8" id="wpcf7-recaptcha-js"></script> <script> // nhs england markExternalLinks(); showDownloadableFileIcons(); // Browsealoud var _baTheme=0, _baUseCookies=true, _baHideOnLoad=true; </script> </body> </html>