18 June 2015 Rare Disease Site & Patient Recruitment KJA
-
Upload
kevin-j-anderson -
Category
Documents
-
view
190 -
download
4
Transcript of 18 June 2015 Rare Disease Site & Patient Recruitment KJA
![Page 1: 18 June 2015 Rare Disease Site & Patient Recruitment KJA](https://reader034.fdocuments.net/reader034/viewer/2022042604/587b1eda1a28ab736c8b61ef/html5/thumbnails/1.jpg)
Site & Patient Recruitment in (Early Phase) Rare Disease Studies
Kevin J. Anderson, MBA
Associate Director, Clinical Operations
![Page 2: 18 June 2015 Rare Disease Site & Patient Recruitment KJA](https://reader034.fdocuments.net/reader034/viewer/2022042604/587b1eda1a28ab736c8b61ef/html5/thumbnails/2.jpg)
2
Agenda
• Challenges in rare diseases
• Key site recruitment assumptions
• In-house approaches to site identification
• Outsourced approaches to site identification
• Incorporating evidence-based methods into site recruitment
• Lessons learned in rare disease site recruitment
• Approaches to patient recruitment
• Lessons learned in patient recruitment
• Conclusions
![Page 3: 18 June 2015 Rare Disease Site & Patient Recruitment KJA](https://reader034.fdocuments.net/reader034/viewer/2022042604/587b1eda1a28ab736c8b61ef/html5/thumbnails/3.jpg)
The Prevalence of Rare Disease
3
rare diseases 7000
of people with rare diseases are children 2/3
people are affected with a rare disease
1 in
affected globally
25
350m
The Global Genes Project. RARE Facts and Statistics. http://globalgenes.org/rare-diseases-facts-statistics/ Accessed June 2015. National Institutes of Health, National Human Genome Research Institute. Rare Diseases Day 2012. http://www.genome.gov/27547266/. Accessed June 2015.
![Page 4: 18 June 2015 Rare Disease Site & Patient Recruitment KJA](https://reader034.fdocuments.net/reader034/viewer/2022042604/587b1eda1a28ab736c8b61ef/html5/thumbnails/4.jpg)
4
Challenges in Rare Disease Studies
• Little is known about these diseases:
○ Data on the disease prevalence and incidence may be unavailable, making it difficult to determine an appropriate study size1
• Extremely difficult to find patients:
○ May be in volatile regions with complex requirements
• Treating physicians often don’t fit into specialty ‘buckets’, and it can be difficult to identify the specialties
• Principal Investigators may be far from a patient’s location:
○ Travel logistics can be complex and difficult for patients and their families
• Rare disease patients are an inherently vulnerable population:
○ The majority of rare diseases affect children, who are considered a vulnerable population – need to ensure naïve sites are well trained1
Patient
1. Registries and Rare Diseases, Applied Clinical Trials, Richard Gliklich, MD, Michelle Leavy, 1 March 2011
![Page 5: 18 June 2015 Rare Disease Site & Patient Recruitment KJA](https://reader034.fdocuments.net/reader034/viewer/2022042604/587b1eda1a28ab736c8b61ef/html5/thumbnails/5.jpg)
5
Challenges in Rare Disease Studies (cont.)
• Majority of PIs are KOLs
○ Can be expensive and difficult to work with
• Many PIs are inexperienced:
○ In North America and Europe, PIs often think they are experienced but are
not
○ In ROW, most sites are inexperienced and only nominally familiar with
ICH/GCPs
• Data integrity can be challenging:
○ Given the small patient populations, every data point counts
○ Every error/misinterpretation confounds the data
• Key Performance Indicators can be very different from industry KPIs:
○ Little rare disease benchmarking data available
• Diagnosis may be complex, particularly in cases where there is no
definitive test – therefore, having the right sites is critical
![Page 6: 18 June 2015 Rare Disease Site & Patient Recruitment KJA](https://reader034.fdocuments.net/reader034/viewer/2022042604/587b1eda1a28ab736c8b61ef/html5/thumbnails/6.jpg)
6
Key Site Recruitment Assumptions
countries 10–15
patient travel
Considerable vendor staffing
for small studies
All study costs will be
patients 20–30 Travel vendor need to support
high
![Page 7: 18 June 2015 Rare Disease Site & Patient Recruitment KJA](https://reader034.fdocuments.net/reader034/viewer/2022042604/587b1eda1a28ab736c8b61ef/html5/thumbnails/7.jpg)
7
Key Site Recruitment Assumptions
• Costs will be very high for every study:
○ 50–75% of sites will not enroll any patients
○ Despite site projections, most sites will enroll no more than one
patient
• A wide net must be cast, with many countries involved:
○ 10–15 countries may be involved in a study with 20–30 patients
• Will require considerable vendor staffing for small studies:
○ Multiple regulatory and EC submissions occurring simultaneously
• Travel vendor needed to support patient travel:
○ Some patients will require cross-country or international travel for
every treatment – need sites that can accommodate this
![Page 8: 18 June 2015 Rare Disease Site & Patient Recruitment KJA](https://reader034.fdocuments.net/reader034/viewer/2022042604/587b1eda1a28ab736c8b61ef/html5/thumbnails/8.jpg)
8
In-house Approaches to Site Identification
• Reach out globally within the company across therapeutic
areas to all country managers, Commercial, MSLs, Med.
Affairs MDs, COLs for recommendations
• Secure funding for major global feasibility studies:
○ May need to contact 500+ sites to identify 20–30 for participation
• Ensure any sites involved in appropriate registries are
included in feasibility
• Search Engine Optimization and Paid Search are critical –
these can drive sites to you
• Social Media Outreach to find potential sites
![Page 9: 18 June 2015 Rare Disease Site & Patient Recruitment KJA](https://reader034.fdocuments.net/reader034/viewer/2022042604/587b1eda1a28ab736c8b61ef/html5/thumbnails/9.jpg)
9
Outsourced Approaches to Site Identification
• Run global feasibility, reaching out to hundreds of sites worldwide
• Reach out to Patient Advocacy Groups if applicable
• Contact organizations such as Patients Like Me, who can assist you in bringing disparate groups together – can locate sites that see the patients
• Medical Informatics – some vendors can locate sites based on treatment patterns and insurance information
• Consider hiring a Clinical Trial Educator or equivalent, who can:
○ Dedicate time to searching authoritative text articles/journal articles for study authors
○ Find investigators from similar trials posted on ClinicalTrials.gov and other public sources
○ Search through key medical associations (e.g. ASH/ASN/ACR) for speakers and authors on related disease topics
![Page 10: 18 June 2015 Rare Disease Site & Patient Recruitment KJA](https://reader034.fdocuments.net/reader034/viewer/2022042604/587b1eda1a28ab736c8b61ef/html5/thumbnails/10.jpg)
10
Incorporating Evidence-Based Methods into Site Recruitment
• Utilize medical informatics data on treatment/insurance claim
patterns in determining appropriate sites
• Use outcomes data from key academic papers
• Leverage market research data on key prescribers for drugs in a
similar therapeutic area
• Use publically available information to identify sites with
successful enrollment in similar disease areas
• Utilize CRO/internal recommended sites with prior history of
successful enrollment
![Page 11: 18 June 2015 Rare Disease Site & Patient Recruitment KJA](https://reader034.fdocuments.net/reader034/viewer/2022042604/587b1eda1a28ab736c8b61ef/html5/thumbnails/11.jpg)
11
Incorporating Evidence-Based Methods into Site Recruitment (cont.)
• Challenge estimates provided by sites in global feasibility
• Use de-identified pre-screen logs:
○ Checklist of key inclusion/exclusion criteria to be met
• Send logs to sites in advance of Site Selection Visit (SSV)
• Review potential patient profiles at SSV
• Ensure patient profiles are a key driver for site selection decision
![Page 12: 18 June 2015 Rare Disease Site & Patient Recruitment KJA](https://reader034.fdocuments.net/reader034/viewer/2022042604/587b1eda1a28ab736c8b61ef/html5/thumbnails/12.jpg)
12
Lessons Learned in Rare Disease Site Recruitment
• From the outset:
○ Run global feasibility
○ Include a Clinical Trial Educator or equivalent
○ Develop a search engine optimization and social media outreach
plan
○ Reach out to Patient Advocacy groups
• Recruit sites from a large number of countries and be willing to work in
volatile regions or countries with complex requirements
• Utilize medical informatics data for site identification
• Use outcomes research in site selection
• Ensure you have at least 2 sites for every patient
• Require sites to show evidence of patient population prior to selecting
![Page 13: 18 June 2015 Rare Disease Site & Patient Recruitment KJA](https://reader034.fdocuments.net/reader034/viewer/2022042604/587b1eda1a28ab736c8b61ef/html5/thumbnails/13.jpg)
13
Approaches to Patient Recruitment
• Study landing page with a 1-800 number
• Outreach to patient advocacy groups to promote study
• Search engine optimization:
○ Link landing page to Patient Advocacy webpages
○ Paid search
○ Banner ads on Healthgrades
○ Targeted banner ads on LinkedIn
• Send ‘Dear Doctor’ letters to MDs local to study sites
○ Purchase physician mailing list from major medical organization
• Create HCP- and patient-focused posters and brochures
![Page 14: 18 June 2015 Rare Disease Site & Patient Recruitment KJA](https://reader034.fdocuments.net/reader034/viewer/2022042604/587b1eda1a28ab736c8b61ef/html5/thumbnails/14.jpg)
14
Approaches to Patient Recruitment (cont.)
• Employ a Clinical Trial Educator1 to:
○ Educate the principal investigator and site staff in successful enrollment
techniques
○ Network with healthcare facilities/providers that may refer potential
study patients
○ Reduce screening loss of otherwise excluded patients from trials through
identification prior to initiation of other therapies
○ Share best practices in focus groups of study coordinators or
investigators
○ Offer creative solutions to recruitment barriers and provide suggestions
to the trial team for development of recruitment tools
1. Quintiles Clinical Trial Educator Proposal, 22 August 2014
![Page 15: 18 June 2015 Rare Disease Site & Patient Recruitment KJA](https://reader034.fdocuments.net/reader034/viewer/2022042604/587b1eda1a28ab736c8b61ef/html5/thumbnails/15.jpg)
15
Lessons Learned in Patient Recruitment
• Employ a Clinical Trial Educator prior to study kickoff meeting
• Build a study landing page at the time of protocol synopsis
• Develop a comprehensive social media plan from the start
• Involve Patient Advocacy groups from the start
• Reach out to physicians via ‘Dear Doctor’ letters using targeted
MD lists purchased from major medical organizations
• Consider building a study app to track recruitment efforts
![Page 16: 18 June 2015 Rare Disease Site & Patient Recruitment KJA](https://reader034.fdocuments.net/reader034/viewer/2022042604/587b1eda1a28ab736c8b61ef/html5/thumbnails/16.jpg)
16
In Conclusion
• There are many challenges
• There are many current solutions
• There are rapidly evolving solutions for the future
• Engage in as many approaches to site/patient
recruitment as you can from the start
• We are all constantly learning in this arena
• It is important to share our knowledge so others
can succeed – our patients depend on it!