Prepared byQueta Bond & Elaine Gallin

QE Philanthropic AdvisorsDecember 2011

Partnering with Patient Advocacy Groups & Voluntary Health Organizations Can Bridge

Gaps in Clinical Research

• The Clinical Research Forum (CRForum) is a non-profit organization that comprises academic health centers, professional societies, and industry.

• CRForum’s mission is to provide leadership to the national clinical & translational research enterprise and promote understanding and support for clinical research and its impact on health and health care.

www.clinicalresearchforum.org

BACKGROUND

BACKGROUND• CRForum has identified enhancing collaborations between academic health

centers (AHCs) and patient advocacy groups/voluntary health organizations (PAG/VHOs) as a priority.

• The goal of the Partnering with PAG/VHO Initiative is to enhance translational and clinical research collaborations between AHCs & PAG/VHOs.– Steering Committee members: Bill Crowley, Pamela Davis, Richard Galbraith,

Albert Reese

• PAG/VHOs are public charities with active fundraising components that work to improve health by supporting advocacy, education & training, health services, and/or research.

2011: LANDSCAPE & TRENDS

• Ongoing federal and state budget shortfalls• Retrenchment of pharmaceutical industry R&D• Increasing globalization & expensive U.S. clinical trials• Difficulty recruiting U.S. patients for clinical trials• Inadequate pipeline of clinical researchers & insufficient career

paths • Poor harmonization of requirements leading to increased

regulatory burden

Responding to these challenges requires increased collaboration across all sectors.

Role of PAG/VHOs in Clinical Research

• Substantial political clout & credible advocates• Source of community education & outreach• Research funders• Access to special patient groups, registries & samples• Bridge to industry

A paradigm shift is occurring, with PAG/VHOs playing greater roles in setting the clinical research agenda in many disease areas.

Estimated 2010 U.S. Health Research Expenditures

Expenditures in Millions of Dollars

Industry - $76.5MFederal & State Gov't - $49.5MUniversities & Research Institutes -$12.5MPhilanthropic Foun-dations - $1.13MVHOs - $0.88M

Data from Research!America

PAG/VHOs provide only a small portion of total U.S. health research expenditures.

• Well over 10,000 organizations with local, national, or international reach

• Annual budgets range from less than $100K to well over $1 billion

• The activities of these organizations and the approaches they use range widely.

PAG/VHO Sector: Size & Diversity

• Traditional PAG/VHOs (support broad range of activities

including research & training to help build fields):• March of Dimes, American Cancer Society, American Heart

Association, the Arthritis Foundation • Entrepreneurial (venture) PAG/VHOs (focus primarily on

developing products):• Multiple Myeloma Foundation, Myelin Repair Foundation,

Cystic Fibrosis Foundation • Groups using mixed approaches:

• Juvenile Diabetes Foundation, Susan G. Komen for the Cure

PAG/VHO Sector: Types of Organizations

Sector is very heterogeneous—one of its great strengths.

Strengths of PAG/VHO Sector

• Number & diversity of organizations• Flexibility & ability to move quickly• Laser focus on specific patient interests• When needed, willingness to work on tough

problems & make long-term investments• Freedom from for-profit & political pressures• Breadth of activities supported: Funding

research, patient care, education, and/or advocacy

Umbrella Organizations

• National Health Council• Health Research Alliance• Research!America• National Organization for Rare Disorders• Genetic Alliance• Faster Cures

A number of umbrella groups exist, but no single group represents a majority of PAG/VHOs. Nonetheless, such groups are potential partners for the CRForum and AHCs.

Government Programs Foster Partnerships with PAG/VHOs

Examples include:• Office of Rare Disease Research

– Supports consortia that foster collaboration between the rare-diseases community & AHCs

• Clinical & Translational Science Award Program– Requires community engagement

Examples of Productive Partnerships Between PAG/VHOs & AHCs:

Supporting Career Development & Training

• American Heart Association (AHA)– Supports early career development & mentoring programs– Currently funds >2,000 researchers

• American Society of Clinical Oncology (ASCO)– Offerings include loan repayment program & awards for

investigators in developing countries

Examples of Productive Partnerships: Creating Research Networks to Foster Data Sharing & Interactions

with Industry

• Cystic Fibrosis Foundation– Funded network of AHC research hubs with strong

emphasis on developing drug pipeline of for CF patients

• Multiple Myeloma Research Foundation– Created collaborative research network linked to

industry to launch clinical trials & accelerate drug development

Examples of Productive Partnerships: Creating Centralized Resources for Rare-disease Research

• Many researchers at AHCs use patient registries, shared databases & tissue banks provided by PAG/VHOs to study patients with rare diseases. PAG/VHOs offering such resources include: – Progeria Research – Pseudoxanthoma Elasticum International– Lymphangioleiomyomatosis Foundation

Examples of Productive Partnerships:Developing Biomarkers & Diagnostics

• Alzheimer’s Association

– World Wide Alzheimer’s Disease Neuroimaging Initiative

Examples of Productive Partnerships:Training Volunteers to Become Research Advocates

• Susan G. Komen for the Cure– Created a community of advocates to participate in

a variety of scientific activities, such as grant review & advocacy.

• Activities: Outreach to PAG/VHO community to identify issues of concern & share information with AHCs– Phone & in-person interviews – Panels at annual CRForum meetings– Web survey designed with input from the Health

Research Alliance and Genetic Alliance and sent to their members

– Products: white paper & related manuscript for publication, as well as this slide presentation

Update on CRForum Initiative: Partnering with PAG/VHOs

Feedback from PAG/VHOs: Results of Phone Interviews & Meetings

• In winter and spring of 2010, QE Philanthropic Advisors conducted: – Phone interviews with ~10 PAG/VHOs & 2

foundations– In-person discussion with 3 umbrella groups

Feedback from PAG/VHOs: Results of Phone Interviews & Meetings

• Contracting processes are cumbersome and often take too long.

• Communication between PIs and contracting offices is often inadequate.

• Many PAG/VHOs want “return on investment” and include requirements for profit sharing, IP sharing & “reach-through clauses” in their contracts; this prolongs negotiations.

• Designed with Health Research Alliance (HRA) & Genetic Alliance; distributed to their members

• Survey responses collected 8/2011 to 9/2011• 37 respondents

– 27 PAG/VHOs; 20 focused on single disease or disease area

– 10 foundations

Feedback from PAG/VHOs: Formal Web Survey (Summer 2011)

• Out of 27 PAG/VHOs:– Total 2010 budgets ranged from $0.2-$272 M– 20 focused on single disease or disease area– 23 funded health research – % of clinical health research funded in 2010

ranged from 25%-100% – 9/23 supported development of young

investigators

Feedback from PAG/VHOs: Formal Web Survey

Survey Questions : What is your organization’s total budget for 2010?

What is your organization’s total health research budget for 2010?

0%

10%

20%

30%

40%

50%

60%

70%

80%

90%

100%

The percent budget spent on health research by the 23 responding PAG/VHOs varied from ~9% to 90%.

100% to >85% 85% to >50% 50% <50% to 25% <25%05

101520253035404550

% R

espo

ndin

g VH

Os

Percentage of total research funded in 2010 that was clinical and/or translational ranged widely for the 23 PAG/VHOs responding to the survey.

Survey Question: What % of research supported by your organization

is clinical and/or translational research?

Most 50% < 50% Little None NA0

10

20

30

40

50

60

70Research

Capacity Building & Training

% R

espo

ndin

g VH

Os

Survey Request: For each area of work, indicate how much of the work your organization funds (whether conducted directly by your organization or through

indirect grants or contracts to other organizations) is carried out with AHCs.

83% of respondents indicated ≥50% of research they support is carried out with AHCs, while only 23% of respondents carry out most of their capacity- building and training activities with AHCs.

Most 50% < 50% Little None N/A0

10

20

30

40

50

60

Patient Registries & Biorepositories

Patient Services

Advocacy & Edu-cation%

Res

pond

ing

VHO

s

Survey Request: For each area of work, indicate how much of the work your organization funds (whether conducted directly by your organization or through

indirect grants or contracts to other organizations) is carried out with AHCs.

Only a few organizations carried out most of their activities relating to patient registries, patient services & advocacy with AHCs.

Survey Request: Assess the quality of interactions between your organization & AHCs

for each area of work supported by your organization.

Consistently

Excel...

Usually

Excelle

nt

Excelle

nt 50% of ..

.

Usually

inadequate NA

02468

10121416

Research

Capacity Building & Training

For research & capacity building/training activities, respondents indicated their interactions with AHCs were consistently or usually excellent.

Survey Request: Assess the quality of interactions between your organization & AHCs

for each area of work supported by your organization.

Consistently

Excelle

nt

Usually

Excelle

nt

Excelle

nt 50% of T

ime

Usually

inadequate NA

0

5

10

15

20

Patient Registries &Repositories

Advocacy & Public Eucation

Patient Services

For non-research and training activities, respondents’ assessments of the quality of their interactions with AHCs were mixed.

Survey Question: Have any of the factors listed below significantly slowed down and/or

negatively impacted your interactions with AHCs?

01020304050607080

% r

espo

ndin

g V

HO

s

Most respondents identified bureaucratic processes, negotiating intellectual property & IRB delays as often problematic.

• Improve communications and linkages.– Provide timely feedback from clinical trials to

patients & PAG/VHOs. – Seek feedback and advice from patients and

PAG/VHOs on research required to address patient needs, and the experiences of clinical trial participants.

– Work with PAG/VHOs to establish educational processes that systematically reach out to, support & educate for these roles.

What Can/Should AHCs Do Now?

• Partner with PAG/VHOs using existing PAG/VHO educational materials and community programs.

• Conduct systems/engineering process exercises to streamline bureaucratic processes.

• Work with PAG/VHOs supporting career development programs to develop career paths and incentives to retain clinician-scientists.

What Can/Should AHCs Do Now?

What Can/Should AHCs Do Now?

• Engage PAG/VHOs in helping simplify informed consent documents.

• Explore ways to help PAG/VHOs recruit investigators to study the diseases on which they focus by developing careers of physician-scientists & building fields of research.

Summary & Conclusions

• Partnerships & collaborations can bridge different parts of the clinical research enterprise and facilitate translation & application of new knowledge.

• Such partnerships and collaborations can help AHCs innovate to enhance organizational effectiveness & achieve their goals.

Summary & Conclusions• PAG/VHOs are natural partners for AHCs: both have a

deep commitment to improving health for their communities.

• PAG/VHOs not only provide research funds, but also help recruit patients to trials, link to industry, catalyze innovation, & support cross-sector research networks to facilitate data sharing & speed product development.

• PAG/VHOs strengths should be fully leveraged by AHCs and the nation as a whole.