Product launches represent a huge investment for pharmaceutical organizations. They also offer the potential for major new revenue streams, and yet the majority of launches fail to meet expectations. By surveying more than 50 individuals with launch experience from within the pharmaceutical community, Huron highlights areas that companies could revisit and potentially improve.

The research provides insights on whether:

• Teams developed sufficiently nuanced market knowledge
• There were proper linkages between launch plans and portfolio strategy and/or functional area plans
• Appropriate processes, resources/training were in place to execute a successful launch
• Best practices were applied across launches

The survey also teases out differences among respondents from different functional groups, (such as marketing, market access and other roles) and those in global verses affiliate roles.

Click here to read the research paper and see what lessons the findings may have for your company.

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To provide insight on the many burdens patients, caregivers, and families must manage when living with a rare diagnosis; while educating the broader rare disease ecosystem regarding the needs necessary to maintain and/or improve quality of life.

Download the complimentary white paper here >>

Paper Insights & Learning:

• The financial implications of rare disease that go beyond affording treatment
• Addressing lifelong habits and the changes necessary to managing a rare condition
• The family dynamic and living with rare challenges from childhood to adulthood
  • The implications of living with a rare condition has on quality of life
  • How rare disease knows no limits and effects each member of the family
• Social and Psychological Implications of rare disease
  • How patients manage the holistic toll of living with a rare condition
• The role stakeholders play in assisting patients overcome routine challenges born by a rare disease

To learn more about MaxifyLife, visit their website.

The post [[ Whitepaper ]] Support Beyond Therapeutic Products appeared first on Orphan Drugs.

The case study draws upon Boston healthcare’s experience working with an orphan drug manufacturer to prepare for the launch of a novel drug by developing a reimbursement strategy and identifying evidence requirements in order to support product adoption.

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Our case study will assist manufacturers of orphan and ultra-orphan drugs to recognize the varying degrees of influence that key stakeholders and the patient care paradigm have throughout the development and commercialization process, as well as the impact this can have in developing a successful market access strategy.

To learn more about Boston Healthcare visit their website.

The post [[ Whitepaper ]] Optimal Pathways to Commercial Success for Orphan Drug Innovators appeared first on Orphan Drugs.

This paper focuses on the critical need for development and refinement of effective tools to identify patient-reported outcomes (PRO) in pre-approval assessments of orphan drugs. Background is provided on the dearth of PROs in the labels of pharmaceuticals for rare diseases and challenges inherent to orphan drug analyses, such as limited and heterogeneous patient populations. However, the author identifies and discusses innovative approaches now being used to develop and implement new tools for PROs, including improvements in qualitative and psychometric/statistical approaches, which can address the unique requirements of orphan drug analyses.

This white paper may have utility for research professionals involved in the development and oversight of patient-reported outcomes and clinical outcome assessments of drugs under development for rare diseases.

Download the complimentary white paper here >>

Readers can expect to:

• Understand the background and challenges inherent to orphan drug assessment, for both patient and clinical outcome analyses
• Describe the progress being made to improve and use PRO measurement tools for orphan drug studies
• Identify examples of innovative tools and approaches to improve PROs in orphan drug analyses

To learn more about Pharmerit, visit their website.

The post [[ Whitepaper ]] Patient-Reported Outcomes in Orphan Drug Clinical Trials appeared first on Orphan Drugs.

The biopharmaceutical industry is increasingly incorporating the voice of the patient in the drug development process, but how effective are these interactions from the perspective of rare disease patients? To explore this question, Covance recently spoke with four leaders who have established their own patient advocacy groups and foundations. 

We wanted to hear about their unique experiences – both as people living with rare diseases and trailblazers for patient advocacy – to gather practical recommendations for the drug development industry.

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This article focuses on how to strengthen the connections between rare disease communities and a network of stakeholders, specifically examining how to best create mutually beneficial relationships of members of the patient community with contract research organizations (CROs) and Sponsors.

To learn more about Covance , visit their website.

The post [[ Whitepaper ]] Connecting With Rare Disease Patient Communities: Patient Advocates appeared first on Orphan Drugs.

Regardless of how innovative a new technology may be, all therapeutic approaches face increasing pressure on target product profiles. Determining what benchmarks novel therapies will need to demonstrate in order to prove differentiation and therapeutic value and compete in the changing rare disease landscape should be an early priority for developers. The transformation of the hemophilia market provides an important paradigm for the development of other rare disease treatment landscapes.

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Cello Health experts provide in-depth analysis and best practice insights to inform early asset development and commercialization.

For more information on Cello Health, visit the website.

The post [[Whitepaper ]] Disruptive Innovation in Rare Disease Markets appeared first on Orphan Drugs.

Innovative, more patient-centric approaches to early phase study design, especially in oncology and rare diseases have become critical, given the increasing complexity of early phase studies and the focus on small patient populations. As participating sites strive to achieve timely and adequate recruitment of eligible participants for these studies, there are new challenges and approaches to consider to ensure success.

During this webinar, PAREXEL’s team of Early Phase experts will share their insights and best practices for complex clinical trials management.

• Increasing study complexity: multiple questions addressed under a single protocol, multiple third party vendors
• Recruitment challenges for targeted populations: for example, enrollment rate forecasting challenges in “basket” patient populations and the importance of determining rate limiting cohorts
• Taking a patient centric approach: for example, selecting sites based on patient location for rare diseases
• Best practices for complex studies site management: the critical need for close collaboration, pro-activity and significant relevant experience

View PAREXEL’s on-demand webinar: Early Phase Insights on Complex Studies and Patient-Centricity. The webinar recording can be accessed here.

You can also access additional drug development strategies on PAREXEL’s exclusive Clinical Development Content Portal. The portal has timely content on a range of topics affecting clinical development professionals.

The post [[ Webinar]] Early Phase Insights on Complex Studies and Patient-Centricity appeared first on Orphan Drugs.

This shift from blockbuster to niche has operated in parallel with the rise of the empowered patient, and increasingly, fund-raising patient advocates, adding further complexity to the landscape.

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The paper also outlines the approach needed to distribute specialty medicines and the challenges of operating within Europe, including the impact of a post-Brexit European Union.

To learn more about CHAPPER Healthcare, visit the website.

The post [[ Whitepaper ]] Meeting Needs: Making A Big Difference To Small Patient Populations appeared first on Orphan Drugs.

Improving Regulatory and Operational Performance in Orphan Drug Development

The environment for developing and marketing orphan drugs is more favorable than at any time in history, thanks largely to a regulatory climate that offers a progressive platform for innovation.  

Download the complimentary white paper now >

The post [[ Whitepaper ]] Regulatory Optimization in Orphan Drugs appeared first on Orphan Drugs.

Download the complimentary white paper  >>

Over the past 10 years, ICON’s highly experienced clinical and therapeutic teams have conducted numerous rare disease trials across a wide range of therapeutic areas, rendering us experts in the field. Learn more about the challenge of rare disease drug development, and how our experienced team can help you to navigate them.

To learn more about ICON, visit the website.

The post [[ Whitepaper ]] Overcoming the Challenges of Rare Disease Drug Development appeared first on Orphan Drugs.