1. Market Research
  2. > Pharmaceutical
  3. > Therapy Market Trends

Commercial Excellence in Rare Diseases and Orphan Drugs

  • June 2015
  • Firstword Pharma
Report ID: 3090980

Summary

Table of Contents






Developing therapies for orphan diseases is attracting much industry interest with the promise of profits. But what are the specific R&D, regulatory, stakeholder and market access issues you need to understand and practically address?


Commercial Excellence in Rare Diseases and Orphan Drugs is a highly-detailed report for industry management who must evaluate, plan, execute, manage and deliver profit from orphan drug programmes. Enriched with case studies, the report reveals the real-world experience and actionable insights of eight senior industry experts who possess a deep knowledge of the challenging and highly-demanding operating environment for orphan drug development and commercialisation.









Gain Answers to Key Questions



How can patients and patient advocacy groups play an enhanced role throughout a product’s development cycle from research funding to market access?
Why is early engagement with payers and KOLs critical in ensuring wide market access and uptake?
To what extent is the accurate assessment of unmet clinical need and establishing potential patient population critical and what are the benchmarks?
With approximately 7,000 identified orphan diseases, how do you select research targets for development?
Clinical trial design and recruitment are challenges for orphan drug developers: what needs to be taken into account and how have companies approached this?
Orphan drug regulation is relatively relaxed, but what more could regulators do and how can you influence them?
Small/Big pharma collaborations: when is the optimum time for engagement and what can each learn from each other?





Top Benefits



Understand how patient support is a must-have for industry and identify the areas where they are most influential
Formulate communication strategies to ensure wide clinical buy-in and support
Address effectively the anxieties of payers who must justify orphan drug spend in their budgets
Examine critical orphan drug candidate selection and overcome challenges of designing and conducting clinical trials
Understand what is considered to be commercial excellence for orphan drugs and what are the hurdles for success
Gain insight into current company structures for orphan drugs and the way in which rare disease teams should be structured in order to maximise commercial success
Understand the regulatory structures that determine orphan drug research/approval and learn where and how regulators can be influenced




Key Takeaways


Orphan drugs offer a positive opportunity for Pharma, but companies must be realistic about their potential and the challenges – old models of assessment used for drugs targeting large patient populations are not fit for purpose
Engagement with key stakeholders at an early stage is critical to ultimate commercial success
Patient Groups, more than any other area, are critical to success and hugely influential in realising a product’s commercial potential
Orphan drugs are often very expensive: make your case to payers, and make it early to ensure buy in
Identifying appropriate research candidates for late phase investment requires a clear understanding of unmet needs and patient population – and how you will access them
Regulators may look positively on orphan drug applications but there are still challenges to overcome
The internal organisation of orphan drug teams varies and the assessment of different models is critical






Report Features – Not Available Elsewhere



Knowledgeable and detailed insights and opinions of eight industry experts working daily in developing and commercialising orphan disease drugs in the US and Europe


Extensive case studies demonstrating different approaches and experiences for ensuring commercial effectiveness


Critical insights for corporate, research, planning, marketing and communications management




Experts Interviewed


Business Unit Head, Rare diseases, Top 10 pharmaceutical company

Senior Director, Top 10 pharmaceutical company

Marketing Director, Mid-size European pharmaceutical company

Sales Director, European pharmaceutical company

Senior Product Manager, Top 10 pharmaceutical company

Director, Rare disease pharmaceutical company

Marketing Director, Top 10 pharmaceutical company

Marketing Director, Top 10 pharmaceutical company






About FirstWord Reports

FirstWord is an innovative industry intelligence leader serving over 240,000 Pharma and MedTech professionals worldwide. FirstWord offers a range of products and services designed to help your company gain a competitive edge by making key business decisions with speed and confidence



FirstWord Pharma PLUS is a personalised and comprehensive intelligence service delivering up-to-the-minute pharma news, insight, analysis and expert views of importance to your company’s success.
FirstWord Reports deliver timely, need-to-know intelligence about your products, your competitors and your markets. Covering biosimilars, market access, medical affairs, sales & marketing, technology and therapy areas, FirstWord Reports provide expert views and intelligence on the challenges facing pharma today.


Get Industry Insights. Simply.

  • Latest reports & slideshows with insights from top research analysts
  • 60 Million searchable statistics with tables, figures & datasets
  • More than 25,000 trusted sources
  • Single User License — provides access to the report by one individual.
  • Department License — allows you to share the report with up to 5 users
  • Site License — allows the report to be shared amongst all employees in a defined country
  • Corporate License — allows for complete access, globally.

Ahmad helps you find the right report:

Testimonials

The research specialist advised us on the best content for our needs and provided a great report and follow-up, thanks very much we shall look at ReportLinker in the future.

Kate Merrick

Global Marketing Manager at
Eurotherm by Schneider Electric

We were impressed with the support that ReportLinker’s research specialists’ team provided. The report we purchased was useful and provided exactly what we want.

Category Manager at
Ikea

ReportLinker gave access to reliable and useful data while avoiding dispersing resources and spending too much time on unnecessary research.

Executive Director at
PwC Advisory

The customer service was fast, responsive, and 100% professional in all my dealings (...) If we have more research needs, I'll certainly prioritize working with ReportLinker!

Scott Griffith

Vice President Marketing at
Maurice Sporting Goods

The research specialist provided prompt, helpful instructions for accessing ReportLinker's product. He also followed up to make sure everything went smoothly and to ensure an easy transition to the next stage of my research

Jessica P Huffman

Research Associate at
American Transportation Research Institute

Excellent customer service. Very responsive and fast.

Director, Corporate Strategy at
Ingredion

I reached out to ReportLinker for a detailed market study on the Air Treatment industry. The quality of the report, the research specialist’s willingness to solve my queries exceeded my expectations. I would definitely recommend ReportLinker for in-depth industry information.

Mariana Mendoza

Global Platform Senior Manager at
Whirlpool Corporation

Thanks! I like what you've provided and will certainly come back if I need to do further research works.

Bee Hin Png

CEO at
LDR Pte Ltd

The research specialist advised us on the best content for our needs and provided a great report and follow-up, thanks very much we shall look at ReportLinker in the future.

Kate Merrick

Global Marketing Manager at
Eurotherm by Schneider Electric

Purchase Reports From Reputable Market Research Publishers

Clinical Chemistry Reagents and Analyzers Market 2021: A 74-Country Analysis--Supplier Shares and Strategies, Volume and Sales Segment Forecasts, Technology and Instrumentation Review

  • $ 75000
  • Industry report
  • May 2017
  • by Venture Planning Group

This new 74-country report from VPGMarketResearch.com is available by region, country, market segment, section, or individual test. Please contact Client Services at reports@vpgcorp.com or +1 212 564 ...

Immunodiagnostics Analyzers and Reagents Market 2021: A 74-Country Analysis--Supplier Shares and Strategies, Volume and Sales Segment Forecasts, Technology and Instrumentation Review

  • $ 75000
  • Industry report
  • July 2017
  • by Venture Planning Group

This new 74-country survey from VPGMarketResearch.com provides a granular strategic analysis of over 100 clinical chemistry, TDM, endocrine, cancer, immunoprotein and abused drug assays performed in both ...

Cancer Diagnostics Market 2021: A 74-Country Analysis--Supplier Shares and Strategies, Emerging Tumor Markers, Volume and Sales Segment Forecasts, Technology and Instrumentation Review

  • $ 52400
  • Industry report
  • May 2017
  • by Venture Planning Group

This new 74-country report from VPGMarketResearch.com is available by region, country, market segment, section, or individual test. This report is designed to assist diagnostics industry executives, as ...


Download Unlimited Documents from Trusted Public Sources

Vaccine Market and Influenza Statistics in India

  • September 2017
    6 pages
  • Vaccine  

    Influenza  

    Diabetes  

  • India  

    United States  

View report >

Health Services Industry in the UK

  • September 2017
    19 pages
  • Cardiology  

    Neurology  

    Hospital  

  • United Kingdom  

    Europe  

View report >

Hormone and Therapy Market in Pakistan and the US

  • September 2017
    7 pages
  • Hormone  

    Therapy  

    Diabetes  

  • Pakistan  

    United States  

View report >

Therapy Market

1 day ago

ref:plp2015

Reportlinker.com © Copyright 2017. All rights reserved.

ReportLinker simplifies how Analysts and Decision Makers get industry data for their business.