1. Market Research
  2. > Pharmaceutical
  3. > Therapy Market Trends
The ROI of Orphan Drugs-- Ensuring optimal returns (2017)

The ROI of Orphan Drugs-- Ensuring optimal returns (2017)

  • December 2017
  • ID: 5251969
  • Format: PDF
  • By Firstword Pharma

Summary

Table of Contents

Orphan drugs are at the forefront of medical science and offer a chance for the pharma industry to make a real difference to people’s lives; there’s also the lure of potentially lucrative commercial benefits for those that succeed. But finding the right balance between return on investment (ROI) and optimum patient access is proving difficult. And with only 415 orphan drugs in the marketplace, it’s still early days.

The ROI of Orphan Drugs: Ensuring Optimal Returns centres on the commercial perspective and provides the inside view from experts already wrestling with the issues arising in the emerging world of rare disease treatments

Discover on this page…
• Key themes covered in the report
• What you will learn from the report
• Detailed table of contents
• Research objectives and methodologies employed producing in the report

Reasons to buy this report

The orphan drugs market is lucrative and growing, however its particular challenges require ’go to market’ approaches that differ substantially from the norm – especially if there is any hope of ROI being achieved. This places the pharma industry at a crossroads on many issues. Should orphan drug prices reflect the level of unmet need, or be built from the bottom up to recoup R&D investment? What ethical stance should the industry be adopting? Are protracted legal battles about exclusivity setting the right tone? Launching an orphan drug and then repurposing it may make sense commercially but is it the right path in the longer term?

Ultimately, how the fledging orphan drugs market resolves these issues will do plenty to influence the reputation of pharma. Will that be for better or worse?

This report will enable you to:
• Get up to speed on how the orphan drugs market has developed since The Orphan Drug Act came into force.
• Understand in detail why ROI is so difficult to achieve for rare disease treatments.
• Explore the commercial potential of orphan drugs including financial and non-financial market incentives.
• Breakdown the key challenges to market success and hear expert views on how these are being overcome.
• Understand where the patient perspective sits alongside the commercial push to meet ROI targets.
• Discover how market frontrunners are using stakeholder engagement strategies to meet ROI targets.
• Review the various business models that drive ROI and gain insight into how these may evolve in the future.

Table of Contents
Executive summary
Research objectives
Experts interviewed

Emergence of the orphan drug market
• Key Insights
• What are orphan drugs?
• Meeting unmet medical needs
• The Orphan Drug Act
• Return on investment

The commercialisation of orphan drugs - viability and attractiveness
• Key Insights
• Incentives – financial and non-financial

Challenges and how companies are overcoming them
• Key Insights
• Research and development costs
• Repurposing existing compounds
• Clinical trial recruitment
• Pricing
• Demonstrating value and effectiveness with RWE
• Healthcare budgets and orphan drug funding
• Ethical considerations when achieving ROI

Optimising ROI via engagement with stakeholders
• Key Insights
• Payers
• Healthcare Professionals
• Patients

Business models that drive ROI
• Key Insights
• Future orphan drug business models – a new approach
• SWOT analysis

Conclusion
• Achieving ROI and meeting patients’ needs
• What does the future hold?
Biographies

Research Methodology and Objectives

This report puts the commercial focus on orphan drugs and looks at how return on investment can be achieved whilst still driving optimum patient access.

Insights are drawn from in-depth interviews with 8 pharmaceutical company experts with a particular interest in orphan drugs who are currently working in commercial strategy, market access or senior management. The interviews were conducted between September and November 2017. One interviewee chose to remain anonymous.
• Danil Blinov, Marketing Lead, Rare Disease - International Developed Markets, Pfizer, UK
• Brian Go, Senior Manager, Alnylam Pharmaceuticals, US

Patrik Grandits, Head of Commercial Operations and Prokurist, Oncology Europe, Daiichi Sankyo Oncology, Europe GmbH
• Philippe Ledru, Senior Director, Global Business Lead, Novartis, US
• Luigi Longinotti, Head of Portfolio Managementr - Orphan Drugs, Recordati, Italy
• Rick Polio, BS, CMR, MBA, Senior Regional Manager, Pulmonary & Biologics of Pulmonary, Autoimmune Disorders, Rare Disease, and Orphan & Biologics at a major biotech company, US
• Gaël Le Rouzo, Oncology Scientific & Market Access Manager, EUSA Pharma, France
• Anonymous, Pharma executive within a global pharmaceutical company

Key questions explored in this report include:
• Are there particular disease areas where it is easier to achieve ROI?
• What impact have orphan drug regulations had on ROI?
• Do investors value the FDA orphan drug designation?
• Do payers value rarity?
• What is the best pricing model to ensure ROI?
• What are the obstacles to achieving ROI and do these vary geographically?
• How do ethical issues come into play within orphan drug ROI?
• What evidence is needed to demonstrate the value of orphan drugs?
• What is the future of orphan drugs given the difficulty in achieving ROI?
• Is a new business model needed for orphan drugs and what might it look like?

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
  • 150+ 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

FAQ
  • How we can help
    • I am not sure if the report I am interested in will fulfill my needs. Can you help me?
    • Yes, of course. You can call us at +33(0) 4 37 65 17 03 or drop us an email at researchadvisor@reportlinker.com to let us know more about your requirements.
    • We buy reports often - can ReportLinker get me any benefits?
    • Yes. Set up a call with a Senior Research Advisor to learn more - researchadvisor@reportlinker.com or +33(0) 4 37 65 17 03.
    • I have had negative experiences with market research reports before. How can you avoid this from happening again?
    • We advise all clients to read the TOC and Summary and list your questions so that we can get more insight for you before you make any purchase decision. A research advisor will accompany you so that you can compare samples and reports from different sources, and choose the study that is right for you.

  • Report Delivery
    • How and when I will receive my Report?
    • Most reports are delivered right away in a pdf format, while others are accessed via a secure link and access codes. Do note that sometimes reports are sent within a 12 hour period, depending on the time zones. However, you can contact us to escalate this. Should you need a hard copy, you can check if this option is offered for the particular report, and pay the related fees.
  • Payment conditions
    • What payment methods do you accept?
      1. Credit card : VISA, American Express, Mastercard, or
      2. You can download an invoice to pay by wire transfer, check, or via a Purchase Order from your company, or
      3. You can pay via a Check made out in US Dollars, Euros, or British Pounds for the full amount made payable to ReportLinker
    • What are ReportLinker’s Payment Terms?
    • All payments must normally be submitted within 30 days. However, you can let us know if you need extended time.
    • Are Taxes and duties included?
    • All companies based in France must pay a 20% tax per report. The same applies to all individuals based in the EU. All EU companies must supply their VAT number when purchasing to avoid this charge.
    • I’m not satisfied. Can I be refunded?
    • No. Once your order has been processed and the publisher has received a notification to send you the report, we cannot issue any refund or cancel any order. As these are not ‘traditional’ products that can be returned, reports that are dispatched are considered to be ‘consumed’.
  • User license
    • The license that you should acquire depends on the number of persons that need to access the report. This can range from Single User (only one person will have the right to read or access the report), or Department License (up to 5 persons), to Site License (a group of persons based in the same company location), or Corporate License (the entire company personnel based worldwide). However, as publishers have different terms and conditions, we can look into this for you.
Purchase Reports From Reputable Market Research Publishers
Elephantiasis (Lymphatic Filariasis) - Pipeline Review, H1 2020

Elephantiasis (Lymphatic Filariasis) - Pipeline Review, H1 2020

  • $ 2000
  • January 2020

Elephantiasis (Lymphatic Filariasis) - Pipeline Review, H1 2020SummaryThe latest Pharmaceutical and Healthcare disease pipeline guide Elephantiasis (Lymphatic Filariasis) - Pipeline Review, H1 2020, provides ...

Infertility Treatment Market - Global Outlook and Forecast 2019-2024

Infertility Treatment Market - Global Outlook and Forecast 2019-2024

  • $ 4000
  • January 2020

The infertility treatment market is expected to grow at a CAGR of over 6% during the forecast period 2018–2024.The growing prevalence of infertility among men and women is increasing the demand for sterility ...

Human Papillomavirus Infections Global Clinical Trials Review, H1, 2020

Human Papillomavirus Infections Global Clinical Trials Review, H1, 2020

  • $ 2500
  • January 2020

Human Papillomavirus Infections Global Clinical Trials Review, H1, 2020Summaryclinical trial report, “Human Papillomavirus Infections Global Clinical Trials Review, H1, 2020 provides an overview of Human ...


ref:plp2017

Reportlinker.com © Copyright 2020. All rights reserved.

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

Make sure you don’t miss any news and follow us on