ti((technology OR technologies OR treatment* OR therapy OR therapies OR lifecycle)) Grey Literature Collection (New York Academy of Medicine) May 2017 “(patient or family or caregiver or parent) AND (engagement OR participation OR involvement) AND (lifecycle OR technology OR treatment OR therapy)” 92 (4 possibly relevant) KU-UC (Reseau de recherché en santé des population du Quebec) May 2017 scanned all reports under keyword heading Technology Assessment searched keywords: “patient participation” “patient engagement” 214 1 4 UK NHS Evidence May 2017 ("patient engagement" OR "patient involvement" OR "patient participation") AND (technology OR technologies OR lifecycle) 3367 (only scanned through first 500 hits; 11 possibly relevant) The Patient – Patient-Centered Outcomes Research May 2017 Scanned tables of contents for all issues (2008-2014) 13 US Food and Drug Administration Patient-Focused Drug Development May 2017 Scanned web page and links to meeting documents & The Voice of the Patient reports 4 European Patients Forum May 2017 Scanned web page 9 European Patients’ Academy on Therapeutic Innovation May 2017 Scanned web page 1 Patient-Centered Outcomes Research Institute May 2017 Scanned web page 3 James Lind Alliance May 2017 Scanned web page 4 HTAi Patient & Citizen Involvement May 2017 Scanned web page of special interest group section 4 Google.ca May 2017 “patient focussed drug development” / “patient focused drug development” ("patient engagement" OR "patient involvement" OR "patient participation") AND (technology OR technologies OR lifecycle) 4 / 301,000 (only scanned first 100 hits) 288,000 (8 possibly relevant; only scanned first 200 hits – mainly on patient engagement & information
meeting with researchers and clinicians to develop the shared agenda was developed • A research agenda was developed Nierse (2013) [137] Netherlands NMDs Patient organizations • A staff member of VSN was a member of the team developing the agenda • A research agenda was developed (2005) [42] Germany Retinitis Pigmentosa Patient organizations • Pro Retina, a German Retinitis Pigmentosa patient group, formulated research priorities for the scientific community Not specified Initiation of research Ferguson (2002) [19] United States Chromosome 18 deletion Patients and families • A mother of a patient noticed improvement in her child’s symptoms after taking human growth hormone and developed a theory to explain her observations based on available scientific literature • Researchers tested the theory, finding it to be correct Mai (2012) [57] United States Li-Fraumeni syndrome (LFS) Patients and families • Patients participated in a workshop around clinical research on LFS at the National Institutes of Health with scientists and physicians, sharing their experiences and their goals • Patients and families identified 6 priorities for clinical research • A new advocacy group was established to facilitate effective communication between LFS families and the clinical and scientific members of the research consortium (2005) [42] Netherlands Addison’s disease; Cushing’s disease Patient organizations • The Dutch Addison and Cushing Society (NVACP) requested an independent research facility to complete a study on improved drug administration methods for the treatment of Addison’s disease • The NVACP-requested study eventually led to the establishment of a research project on delayed release tablets Panofsky (2011) [145] United States Rare diseases in general Patient organizations • Patient organizations develop close relationships with, and between, scientists (i.e., “sociability”) to influence the research process • Additional mechanisms utilized include resources; collective mobilization; timing; lay expertise; and organization controls • Note: according to the author, without sociability, these additional mechanisms are insufficient for influencing research • “Sociability” provides benefits for patient organizations and can help scientists develop stronger research networks and improved productivity Patient Partner [20] Not specified Rare diseases in general Patient organizations • Patient organizations promote the running of a trial by gathering researchers; demonstrating interest in new treatments; and bringing together research teams Not specified
information on the experiences of patients and their parents to write a white paper on the disease De Moerloose (2015) [146] Ireland Haemophilia Patient organizations • The Irish Haemophilia Society collected patient data to assess the effect of a serious bleed on health-related quality of life. • Health utility values based on the EQ-5D survey were calculated for 3 patients • This data can supplement key clinical data to inform HTA bodies and payers of the true impact of haemophilia on the day-to-day lives on patients Genetic Alliance UK (2014) [97] United Kingdom Rare diseases in general Patient organizations • Genetic Alliance UK has developed a project to explore the perspectives of patients and families affected by genetic conditions regarding willingness to accept risk or adverse outcomes with a new treatment Not specified Ferguson (2002) [19] United States Gastrointestinal stromal tumor (GIST) Patient organization • The Life Raft Group, a patient group started by the husband of a patient, published a study evaluating Gleevec’s effectiveness for GIST patients • The study collected data on quality of clinical care available at clinical trial centres; attempted to evaluate the sources of information that participants relied on; developed a methodology for participants to serve as their own control group; and introduced a new scale to rate side effect severity from the patients point of view • The leader of LifeRaftGroup.org states that patient-initiated research helps reduce the lag time that professional researchers experience, helping to get important information to patients faster Ferguson (2002) [19] United States PXE Patient organizations • PXE International was part of the research team working towards identifying the gene responsible for PXE • The gene was identified and patented by the research team Seminara (2010) [24] United States UCDs Patient organizations • Within the UCDC of the RDCRN, the National Urea Cycle Disorders Foundation has developed and distributed surveys to determine attitudes and barriers to study participation Not specified Alliance (VHLA) (2014) [40] United States Von Hippel-Lindau disease (VHL) Patient organizations • The Von-Hippel-Lindau Alliance (VHLA) developed a VHL Research Council Not specified Patient Partner Not specified Rare diseases in general Patient organizations • Patient organizations gather data or other information Not specified
(2013) [39] (PC) organizations an international collaborative network of patients, medical professionals, and scientists Schwartz (2013) [39] United States PC Patient organizations • Members of the PC Project established the International PC Consortium, which connects various researchers and specialists to conduct basic clinical research Not specified Dissemination of research-related information Ekins (2012) [144] International (Note: project started in North America) Rare diseases in general Patients, families, and patient organizations • The Open Drug Discovery Teams app collects Tweets using particular hashtags (e.g., #sanfilipposyndrome) • Anyone may use the app and curate the content by endorsing or disapproving of each Tweet (or “factoid”) • The authors anticipate that this app will be used to disseminate information on new scientific developments, to network and discover other researchers, and to provide opportunities for collaboration by highlighting conferences, publications, or laboratory data sharing JISC (2011) [54] United Kingdom NMDs Patients • Patients participated in the Talk Research focus group to discuss if the communication services provided by the Muscular Dystrophy Charity (MDC), now known as Muscular Dystrophy UK, meet the needs of patients and to provide feedback on language, content, and the structure of the MDC websites and publications • Patients involved in the Talk Research focus group have become involved in other MDC activities such as reviewing grant applications JISC (2011) [54] United Kingdom NMDs Patient organizations • The MDC communication service employs a team of people who have work in research to produce summaries of new research (e.g., lay summaries of clinical trials) • The MDC is also part of Patient Inform, which provides patients with access to research articles Not specified Landy (2012) [55] Not specified Rare diseases in general Patient organizations • Patient organizations presented research at scientific conferences, via websites and newsletters, and through the press Not specified Landy (2012) [55] Not specified Rare diseases in general Patient organizations • Patient organizations helped to prepare research reports/articles Not specified Provide funding for research Kelly (2017) [149] International Cystic Fibrosis (CF) Patient organizations • CF patient organizations fund research, including funding one larger project, funding a series of on a common theme, partially funding a research project, and indefinitely funding part of a Not specified
EURODIS (2011) [21] Europe Rare diseases in general Patient organizations • Patient organizations help to adapt the design of clinical trials to meet patients’ expectations, facilitating adherence and ensuring quality of life is taken into consideration Not specified EURODIS (2011) [21] Europe Rare diseases in general Patient organizations • Patient organizations discuss results with trial sponsors to contribute to the assessment of treatment benefits Not specified EURODIS (2011) [21] Europe Rare diseases in general Patient organizations • Patient organizations provide early information to potential participants to ensure inclusion in a trial Not specified Boon (2010) [46] Netherlands NMDs Patient organizations • The VSN is involved in assisting and initiating clinical trials Not specified Black (2011) [22] United States Severe myoclonic epilepsy of infancy (SMEI or Dravet syndrome) Patient organizations • The IDEA League provides researchers with access to a large cohort of patients for research Not specified Black (2011) [22] United States SMEI Patient organizations • Centres within the IDEA League’s Collaborative Clinical Research and Comprehensive Care Network collaborate to develop clinical research protocols Not specified Groft (2013) [23] United States Rare diseases in general Patient organizations • The Rare Diseases Clinical Research Network (RDCRN) obtains input from patient organizations on the development of informed consent statements, recruitment strategies, and protocols for research conducted within the Network Not specified Groft (2013) [23] United States Rare diseases in general Patient organizations • Patient organizations help the RDCRN to identify patient cohorts and recruit patients for studies conducted by the Network • Researchers in the RDCRN rank help with patient recruitment as one of the top three benefits of interactions with patient organizations [154] Seminara (2010) [24] United States Urea Cycle Disorders (UCDs) Patient organizations • The National Urea Cycle Disorders Foundation is involved in the Urea Cycle Disorders Consortium (UCDC) of the RDCRN • The foundation is directly involved in the development of protocols, consents, content evaluations, and progress reporting in RDCRN studies Not specified Patient Partner [20] Not specified Rare diseases in general Patient organizations • Patient organizations inform patients of opportunities for taking part in clinical trials Not specified
Patient Partner [20] Not specified Rare diseases in general Patient organizations • Patient organizations assist in the development of clinical research protocols Not specified Patient Partner [20] Not specified Rare diseases in general Patient organizations • Patient organizations review funding requests, clinical trial protocols, or patient information to be used in a trial Not specified Patient Partner [20] Not specified Rare diseases in general Patient organizations • Patient organizations provide advice or serve as advisory members on clinical research program committees for the development of a trial Not specified Patient Partner [20] Not specified Rare diseases in general Patient organizations • Patient organizations supply demographic and disease-specific information on members represented by the organization for use in a trial Not specified Provide funding for clinical trials Fajac (2013) [150] Europe CF Patient organizations • The European Cystic Fibrosis Society’s Clinical Trial Network (ECFS-CTN) is funded by the ECFS and national patient organizations through the umbrella organization CF Europe Not specified Groft (2013) [23] United States Rare diseases in general Patient organizations • Patient organizations fund research conducted within the RDCRN, including travel clinics to facilitate patient access to investigators and studies within the RDCRN • Researchers in the RDCRN ranked direct funding as one of the top three benefits of interactions with patient organizations [154] Patient Partner [20] Not specified Rare diseases in general Patient organizations • Patient organizations finance or raise funds for clinical trials Not specified Development of or involvement in clinical trial organizations/networks Fajac (2013) [150] Europe CF Patient organizations • The ECFS established a clinical trial network (ECFS-CTN) comprised of 30 selected sites across 11 European countries • Current initiatives are to set up an trial, to implement central laboratories for outcome measures, further develop new surrogate end points, and establish a quality-improvement program Not specified Fajac (2013) [150] Europe CF Patient organizations • A patient organization representative sits on the Executive Committee of the ECFS-CTN and other representatives are invited to meet with the Executive Committee once a year Not specified Fajac (2013) Europe CF Patient organizations • Patient organizations are invited to ECFS-CTN steering committee meetings twice a year Not specified
post-bereavement support, disability benefits, respite care, and housing Boon (2010) [46] Netherlands NMDs Patient organizations • The VSN keeps track of scientific development, annotates them, tries to clarify them, and reports on them with disclaimers to the patient community Not specified Black (2011) [22] United States SMEI Patient organizations • The League has a website containing information on the clinical course, etiology, epidemiology, and treatment of Dravet syndrome and provides links to other international resources and support groups, and to educational financial and health care resources for children with special needs • Information is available regarding opportunities to fund & participate in research, local events in the Dravet syndrome community, genetic testing, and regional epilepsy centers providing care to patients Not specified Dunkle (2010) [47] United States Rare diseases in general Patient organizations • Patient organizations created a database of medical information on rare diseases that both patients and health care professionals may access Not specified Groft (2013) [23] United States Rare diseases in general Patient organizations • Patient organizations are involved in educating patients, public, media, and healthcare providers within the RDCRN Not specified Groft (2013) [23] United States Rare diseases in general Patient organizations • Patient organizations provide disease-specific information for patients and families on RDCRN website Not specified Seminara (2010) [24] United States UCDs Patient organizations • The National Urea Cycle Disorders Foundation helped to design content for the UCDC website and develop and distribute brochures to patients, families, and health care professionals Not specified Alliance (VHLA) (2014) [40] United States VHL Patient organizations • The VHLA publishes a VHL Handbook, providing patients with tips on diagnosis, treatment, and ways of living with VHL Not specified Alliance (VHLA) United States VHL Patient organizations • The VHLA answers questions for physicians or passes questions along to appropriate experts Not specified
one of the largest HMOs in Israel Advocating for legislation Dunkle (2010) [47] United States Rare diseases in general Patient organizations • NORD representatives testify before Congressional committees on a regular basis •Advocacy efforts of NORD have focused on: rare disease and orphan drug legislation; ensuring adequate funding for the FDA, the NIH, and the SSA; extended patent period on orphan products; the removal of lifetime caps from health insurance policies; and for legislation to improve patient access to clinical trials • The passing of the Orphan Drug Act was due in part to the advocacy efforts of the coalition that would eventual become NORD Lasker (2005) [49] United States PBC Patient organization • The PBCers Organization advocates for those with PBC Not specified Landy (2012) [55] Not specified Rare diseases in general Patient organizations • Patient organizations lobby policy makers Not specified Awareness campaigns Black (2011) [22] United States SMEI Patient organizations • The IDEA League participates in national awareness campaigns Not specified Dunkle (2010) [47] United States Rare diseases in general Patient organizations • Patient organizations created Rare Disease Day Not specified Conferences and Workshops Participating in conferences and workshops Molster (2012) [95] Australia Rare diseases in general Patients, families and patient organizations • Patients, families, and patient organizations participated in workshops at the Australian Rare Diseases Symposium discussing the issues, goals, and actions relevant to the development of a national plan • Participants identified a range of key issues for consideration, with associated goals and actions • There was agreement that the recommendations drawn out from the outcomes of the workshops should be used to inform and frame the development of a national plan Hosting conferences and workshops De Blieck (2013) [35] United States JNCL Patient organizations • The BDRSA hosts an Annual Conference • The conference provides a venue for the URBC to enroll families in the contact registry, the natural history and disease-specific rating scale study, and/or neuropsychological investigations Lasker United States PBC Patient • The PBCers Organization hosts conferences with Not specified
(2005) [49] organization medical experts Seminara (2010) [24] United States UCDs Patient organizations • The National Urea Cycle Disorders Foundation hosts an annual conference where educational presentations are given to patients and professionals by UCDC investigators • The conference has helped to facilitate recruitment into UCDC studies Cystic Fibrosis Foundation (CFF) (2013) [34] United States CF Patient organizations • The CFF hosts the annual North American CF Conference, where updates on CF care and research are shared Not specified Alliance (VHLA) (2014) [40] United States VHL Patient organizations •Hosts the International VHL Medical Symposia, bringing together leaders in VHL basic, translational, and clinical researchers, as well as clinicians specializing in VHL diagnosis and treatment • The conference helps to stimulate research and make connections among professionals Funding conferences and workshops Groft (2013) [23] United States Rare diseases in general Patient organizations • Patient organizations fund research-based scientific conferences and meetings for patients, families, and families within the RDCRN Not specified Landy (2012) [55] Not specified Rare diseases in general Patient organizations • Patient organizations organize and support scientific conferences Not specified Patient care and support Social support for patients Frost (2008) [53] International Rare diseases in general Patients • Patients participate in Patients Like Me, an online community in which data can be entered into health profiles (e.g., symptoms, treatments, functional rating etc.) and members can communicate via forums and comments on their profiles • 3,200 members (including families, providers, and researchers) have joined since 2006, with 1,750 patient users • Data is aggregated from all health profiles to create community summaries on treatments and symptoms Hughes (2008) [158] International Rare diseases in general Patients and families • Patients and their families participate in discussion threads on RareConnect.org, an online patient forum • Posts tended to fall within one of the following themes: finances, difficulty in getting a diagnosis, feeling that doctors are not knowledgeable, and • The author concludes that rare disease patients provide their peers with social support and information • RareConnect.org provides the opportunity for general information sharing • However, due to the prevalence of moderators
(2011) [22] organizations families attend their annual “Dravet Weekend Away” JISC (2011) [54] United Kingdom NMDs Patient organizations • The MDC as a patient forum as well as a Facebook page and Twitter account Not specified Black (2011) [22] United States SMEI Patient organizations • The IDEA league established an online network allowing for social networking between families in online forums • The online family forum has helped to identify challenges in the care of Dravet syndrome patients, including access to care, difficulties in diagnosis, treatment disparities among providers, and the need to define associated comorbidities • Information gathered in the online forums has stimulated research into associated conditions (e.g., facial anomalies) Black (2011) [22] United States SMEI Patient organizations • The IDEA League has organized social gathering for patients and their families Not specified Lasker (2005) [49] United States PBC Patient organization • The PBCers Organization provides electronic mailing lists (listservs), chatrooms and message boards for use by patients and other informational resources • Patients were found to use the online resources provided by the PBCers Organization, particularly the Daily Digest, to discuss biomedical issues (e.g., medications), but framed within the context of offering support to their peers Alliance (VHLA) (2014) [40] United States VHL Patient organizations • The VHLA hosts an Annual Family Meeting Not specified Landy (2012) [55] Not specified Rare diseases in general Patient organizations • Patient organizations organize support groups and maintained toll-free support lines Not specified Financial support for patients Black (2011) [22] United Kingdom SMEI Patient organizations • The IDEA League UK has helped families obtain medical equipment by funding the purchases or helping families to obtain discounts Not specified Black (2011) [22] United States SMEI Patient organizations • The League created a medication assistance fund to help financially disadvantaged families obtain stiripentol when funding is not available Not specified
Dunkle (2010) [47] United States Rare diseases in general Patient organizations • NORD administers Patient Assistance Programs for uninsured and underinsured patients Not specified Dunkle (2010) [47] United States Rare diseases in general Patient organizations • NORD provides drugs at no charge to eligible patients through its Medication Assistance Programs Not specified Dunkle (2010) [47] United States Rare diseases in general Patient organizations • NORD provides premium and copayment funds for patients with certain disorders, who cannot afford out-of-pocket costs associated with their plans Not specified Landy (2012) [55] Not specified Rare diseases in general Patient organizations • Patient organizations provide financial assistance to patients Not specified Support for patients participating in clinical trials Bendixen (2016) [98] Proposed United States DMD Families • Families who have participated in a trial should provide peer support for those who are just entering trials Not applicable Dunkle (2010) [47] United States Rare diseases in general Patient organizations • Patient organizations provide travel and temporary housing assistance to patients and families who must travel to participate in clinical trials Not specified EURODIS (2011) [21] Europe Rare diseases in general Patient organizations • Patient organizations support patients during a study to reduce the rate of drop-outs and incomplete files Not specified Clinical care support for patients Genetic Alliance UK (2014) [97] Proposed United Kingdom Rare diseases in general Patients • Patients, in partnership with their physicians, should decide whether the benefits of a new drug outweigh its risks. Not applicable Pattacini (2009) [50] Italy Hemophilia Patients • Patients access part of their clinical records through ‘xl’Emofilia, a web-based electronic outpatients’ record, in order to consult their data and record bleeding events and home infusions • Patient-entered data is validated by doctors at a treating Hemophilia Centre • Patients receive training to use these records • Data is extracted in an anonymous format and published on a website • Some data is also processed and sent to the Italian Registry of Hemophilia and Allied Disorders • The authors state that this system has improved the management of data, facilitated work, and improved the quality of care for patients
Ferguson (2002) [19] United States Chromosome 18 deletion Patients and families • The mother of a patient developed the Chromosome 18 Registry and Research Society, uniting three independent organizations Not specified Providing guidance on establishing patient organizations Mai (2012) [57] United States LFS Patient organizations • At a workshop at the National Institutes of Health, patient organizations shared their perspectives and advice on forming support and advocacy groups Not specified Establishing international patient organization alliances Woodward (2016) [156] International aHUS Patient organizations • Representatives from 6 European countries (Belgium, France, Italy, Russia, Spain, and the UK) met and agreed to collaborate as the aHUS Alliance. Since then, another 6 organizations have become affiliated with the organization (Australia, Canada, Germany, India, Netherlands, and USA) Boon (2010) [46] Netherlands NMDs Patient organizations • The VSN helped to establish the International Pompe Association, an alliance of patient organizations Not specified Alliance (VHLA) (2014) [40] United States VHL Patient organizations • The VHLA establishes similar alliances around the world Not specified Fundraising to support further patient organization growth Alliance (VHLA) (2014) [40] United States VHL Patient organizations • The majority of funds supporting the VHLA are acquired through fundraising initiatives Not specified Regulatory decision-making Input into the regulatory decision-making process Genetic Alliance UK (2014) [97] Proposed United Kingdom Rare diseases in general Patients • Patient input on acceptable risk should be imbedded into regulatory decision-making Not applicable Pharma Letter Canada Rare diseases in general Patients and families • As part of the new Orphan Drug Framework in Canada, patients provide input on how the disease • This is a pilot project that will be used by Health Canada to assess and refine its approach
(2014) [111] Pilot project affects their ability to manage their day-to-day lives, what treatments are currently available, what therapeutic benefits are most important to them, and their risk tolerance for new treatments to collecting patient input, facilitating this process in future orphan drug authorizations EMA (2012) [69] [Website] European Union Rare diseases in general Patient organizations • Patient organizations are consulted on requests by the European Medicines Agency’ scientific committees and working parties • They take part in discussions on the development and authorization of medicines Not specified EMA (2012) [69] [Website] European Union Rare diseases in general Patient organizations • Patient organizations review written information on medicines prepared by the EMA Not specified EMA (2012) [69] [Website] European Union Rare diseases in general Patient organizations • Patient organizations are involved in the preparation of EMA guidelines Not specified EMA (2012) [69] [Website] European Union Rare diseases in general Patient organizations • Patient organizations take part in EMA conferences and workshops Not specified FDA (2017) [159] United States Rare diseases in general Patients and families • Patients and families attend public meetings hosted by the FDA where they have the chance to discuss their disease and its impact on their daily lives, the types of treatment benefits that matter to them most, and their perspectives on the adequacy of current therapies Not specified FDA (2017) [159] United States Rare diseases in general Patients and families • As part of the FDA’s Patient Representative Program, patients and families provide input on disease-specific issues relating to medical products in various stages of development, review and approval Not specified Black (2011) [22] United States SMEI Patient organizations • The IDEA League contributed data used in the orphan drug designation application for stiripentol in the United States • Stiripentol received orphan drug designation in the United States Dunkle (2010) [47] United States Rare diseases in general Patient organizations • NORD provides input into FDA processes, such as reviewing applications and standards for drug and device testing Not specified Membership on a regulatory decision-making or advisory committee
endpoints Reimbursement decision-making Input into the reimbursement decision-making process O’Mahony (2014) [96] Proposed European Union Haemophilia Patients • It was stated at the EHC Congress satellite symposium that it is necessary to begin engaging HTA agencies, possibly through EU Network for HTA (EUnetHTA), to allow for patient input at the earliest stage of the HTA process •It was also suggested by a panel member that there is an opportunity for patient engagement in EUnetHTA’s current efforts to develop common practices across the EU that ensure consistency of data collection • e.g., it is essential that the system is capable of understanding the impact of an intervention in the context of the experience of hemophilia patients Not applicable O’Mahony (2014) [96] Proposed European Union Haemophilia Patients, families, and patient organizations • To ensure that health technology assessments (HTAs) consider factors beyond cost (i.e., factors that matter to patients), a panel member at the EHC Congress satellite symposium suggested that patient advocates and clinicians must work together to create a shared consensus on how to effectively evaluate the efficacy of therapies and what experiential data are of the greatest value Not applicable Genetic Alliance UK (2014) [97] Proposed United Kingdom Rare diseases in general Patients • Nominated patient experts should be permitted to present a summary of the patient evidence submission to the Evaluation Committee Not applicable Genetic Alliance UK (2014) [97] Proposed United Kingdom Rare diseases in general Patients • Patient should be formally consulted during the National Institute of Health and Care Excellence (NICE) Highly Specialized Technology (HST) evaluation process, prior to the final Committee meeting, to outline the nature and timeline of any post-evaluation research recommended by NICE Not applicable Genetic Alliance UK (2014) United Kingdom Rare diseases in general Patients • If the benefit/risk of a product is to be by NICE, patient input should be included and all information from patient Not applicable