CRA's Project LEOPARD Survey

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* 1. Please provide us with your name, company, job title, and email.

Name

Company & Job Title

Email Address

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* 2. How do you prefer to receive updates and thought pieces on rare disease?

Articles

Infographics

Podcasts & videos

Webinars & roundtables

Other (please specify)

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* 3. What rare disease topics do you want to hear more about?

Trends in Orphan Medicinal Product (OMP) policy/assessment

How to find rare disease patients

Optimization of rare disease care pathways

Justifying OMP value with evidence

Other (please specify)

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* 4. For established/addressed rare diseases with product(s) indicated, please select the top three future trends you consider most important for rare disease companies

Higher bar for evidence

Need for innovative forms of engagement to differentiate

Increasing price pressure

Subject to restriction or tendering

Commercial opportunity trade-offs given market maturity

LoE exacerbating competition

Proactive targeting of niche/subpopulation

Other (please specify)

Does not apply

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* 5. For unaddressed rare diseases with high unmet needs, please select the top three future trends you consider most important for rare disease companies

Lower evidence expectations for first to market

Alternative access approaches to alleviate uncertainty

Increased payer scrutiny of “rare” or “orphan”

Competitive role for patient finding to accelerate access & uptake

Increasing RD affordability concerns

Need to establish patient support “beyond the pill”

Other (please specify)

Does not apply

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* 6. For rare subcategories/segments of non-rare conditions, please select the top three future trends you consider most important for rare disease companies

Need to show incremental value in rare sub-category

Dissociating messaging from parent non-rare disease

Strict management approaches to prevent spillover

Reliable diagnosis and precision medicine expectations

Development of distinct care pathways

Other (please specify)

Does not apply

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* 7. Of the rare disease critical success factors, which (up to three) would you like to hear more about?

Identifying unmet need

Precision medicine

Evidence generation

Market shaping

Stakeholder engagement

Go-to-market strategy

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* 8. Would you be open to a follow up relating to Project Leopard? If yes, we will use the email address provided above. If you prefer a different email address, please provide below.

Yes

Best email address for contact