1. Market Research
  2. > Pharmaceutical
  3. > Therapy Market Trends
Orphan Drug Market Access: Payer Insights 2016

Orphan Drug Market Access: Payer Insights 2016

  • October 2016
  • ID: 4275619
  • Format: PDF

Summary

Table of Contents

Description

How to ensure positive payer engagement for orphan drug market access

The explosion in orphan drug approvals and high prices are causing increasing concern to payers.
When there were few orphan drugs for small patient populations the cost was less of an issue. But with the orphan drug bill pushing 10% of drug spending to treat less than 1% of patients, hard questions are being asked and tough choices being made.
The current trends are unsustainable and payers are looking to rein in costs through vigorous HTA assessment, innovative contracting, and higher proof of value. Orphan drug developers need to take seriously the issues payers face and Orphan Drug Market Access: Payer Insights is a “must read” report that shows how positively working with them will have real market access benefits.

Key Features
- In-depth interviews reveal the insights of leading payers in the US, France, Germany, Italy, Spain and the UK gained from frontline experience
- SWOT of the orphan drug market access landscape in 2016
- Graphs and tables defining orphan drug product and developer status
- Charts outlining US/EU orphan drug designation process
- Case study of NICE rejection of Alexion Pharma’s Kanuma
- At-a-glance summaries of all the main conclusions

Key Benefits
- Understand current market dynamics and why the growth in the number of orphan products and the rise in prices is unsustainable
- Easily evaluate the players and products that are leading the the orphan drug sector
- Review in detail the regulatory incentives and support for orphan drugs in the US and EU-5.
- Appreciate how the orphan drug market access landscape in the EU5 countries differ and the challenges which must be overcome at the national level
- Assess US payer responses to the growing cost burden of orphan drugs that threatens the free pricing model
- Drive payer engagement practices that build supportive relationships
- Know how payers view the role patients can play in the orphan drug space.

Key Questions Answered By This Report:
- Defining “Orphan”: Is there a need to more narrowly redefine what an orphan drug actually is?
- Regulation: Regulatory incentives are driving rare disease drug development but how do they differ in the US and EU and what’s on offer?
- EU Reimbursement: EMA approval doesn’t necessarily translate into positive reimbursement at the national level. How is this affecting market access post-approval and where?
- US Pricing: Which legal ruling has impacted pricing of orphan drugs in non-orphan indications and why is this a concern to US payers?
- New Working Models: What do payers want in terms of pricing, innovative contracting, post-marketing research and engagement with pharma?
- The Patient Voice: What role would payers like to see patient groups take on in orphan drugs?

Expert Views
To ensure candid views were expressed, payer names have been kept anonymous, but each has been selected for their experience and detailed current knowledge of orphan drug issues.
- French Payer - Head of the Department of Pharmacy at a major university hospital in Paris and a Professor of Clinical Pharmacy and Biotechnology at the Faculty of Pharmacy at his university. He is President of a national hospital formulary group covering almost all therapy areas, including many that have an ‘orphan’ designation.
- German Payer - His role involves managing the drug budget, including negotiating with insurance companies, as well as negotiating the federal state budget for specialty groups. His organisation reimburses orphan drugs and he is a voting member of the Federal Joint Committee on the assessment of orphan drugs.
- Italian Payer - The Pharmacy Director of a hospital which is the referral centre for specific metabolic rare diseases with budget responsibility for all of the drugs prescribed in the hospital. She helps physicians identify the most appropriate drugs, particularly in situations where no specific treatments are available for a particular rare condition.
- Spanish Payer - The Director of a Pharmacy Service and the Chief of the Pharmacy and Therapeutic Committee at a large teaching hospital in Valencia who has experience in the use, financing, pricing, reimbursement and market access of medications in Spain including orphan drugs
- UK Payer - A regional pharmacy payer with a role involving implementation of NICE guidelines, allocation of health technology resources and formulary guidelines, reimbursement policy and procedures for Commissioning Groups in hospitals
- US Payer 1 - Executive Vice President of a national plan and has responsibility for the oversight of manufacturer relationships, formulary development, reporting and analytics. This includes sitting on committees that review the appropriateness, coverage and contracting for orphan drugs.
- US Payer 2 – A senior manager in a large national payer who is a member of the Formulary Pharmacy and Therapeutics Committee and has been engaged in formulary management, specialty injectables, manufacture contracting and benefit designs for 14 years.
- US Payer 3 – The Chief Medical Officer and Chair of the Pharmacy and Therapeutics Committee at his organisation. He is responsible for the entire pharmacy budget and is involved in decisions regarding coverage and restrictions on drugs, formulary placement and contracting.
“I don't think it's fair for the payer system to inherit an orphan-like drug price for a non-orphan disease.”
- US Payer 1
“We cannot decide at the hospital level to choose between patients. If you have no money to pay for very expensive drugs, the government says, ‘okay, you have to manage the problems yourself.’ But we cannot manage the problems ourselves, because we have a lot of patients coming into the hospitals.”
- “I think a good model is having companies which can maybe arrange face to face interviews, advisory forums, payer forums where they have more in-depth, deep-dive discussions. I think there's a few companies doing that. Basically they need to do that more now.” UK Payer

Who will benefit from this report?
- Market access professionals needing to secure orphan drug market entry
- MSL teams needing knowledge of orphan drug pricing and reimbursement issues
- HEOR teams building value and evidence portfolios for orphan drugs
- Brand/KAM teams charged with presenting commercial propositions for orphan drugs to payers
- Regulatory affairs professionals tracking orphan drug regulation and incentives
- National and regional payers having to negotiate pricing and reimbursement with companies
- Health technology experts reviewing the safety, efficacy and value of orphan drugs
- Patient advocacy groups wanting to widen market access and availability.

Content Highlights
- Market dynamics
- Market size and growth forecast
- The key players
- Regulatory environment
- Orphan drug regulation in the US
- Orphan drug regulation in the EU
- International harmonisation
- Incentives for orphan drug development
- Should orphan drugs be more narrowly defined? – Payer views
- Pricing, reimbursement and market access
- Pricing and reimbursement issues in the US
- Pricing and reimbursement in the EU
- Challenges and opportunities for orphan drug market access
- The need to differentiate as the number of orphan drugs rises
- High prices are unsustainable
- Innovative contracting models are needed
- Establishing relationships with payers
- Payers need greater access to relevant data
- Patient associations can be influential in gaining market access
- Payers suggest patients should be heard
- Opportunities for future orphan drug development

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
Coronary Artery Disease (CAD) (Ischemic Heart Disease) - Pipeline Review, H1 2019

Coronary Artery Disease (CAD) (Ischemic Heart Disease) - Pipeline Review, H1 2019

  • $ 2000
  • April 2019

Coronary Artery Disease (CAD) (Ischemic Heart Disease) - Pipeline Review, H1 2019SummaryGlobal Markets Direct’s latest Pharmaceutical and Healthcare disease pipeline guide Coronary Artery Disease (CAD) ...

Respiratory Assist Devices - Medical Devices Pipeline Assessment, 2019

Respiratory Assist Devices - Medical Devices Pipeline Assessment, 2019

  • $ 4000
  • April 2019

Respiratory Assist Devices - Medical Devices Pipeline Assessment, 2019SummaryGlobalData’s Medical Devices sector report, “Respiratory Assist Devices - Medical Devices Pipeline Assessment, 2019" provides ...

Neuronal Acetylcholine Receptor Subunit Alpha 7 (CHRNA7) - Pipeline Review, H1 2019

Neuronal Acetylcholine Receptor Subunit Alpha 7 (CHRNA7) - Pipeline Review, H1 2019

  • $ 3500
  • April 2019

Neuronal Acetylcholine Receptor Subunit Alpha 7 (CHRNA7) - Pipeline Review, H1 2019SummaryNeuronal Acetylcholine Receptor Subunit Alpha 7 (CHRNA7) - Neuronal acetylcholine receptor subunit alpha-7 is a ...


ref:plp2016

Reportlinker.com © Copyright 2019. 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