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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






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Table Of Contents

Commercial Excellence in Rare Diseases and Orphan Drugs
1.Executive summary
2.Research objectives and methodology
3.Contributors
4.Orphan drugs overview
4.1.The evolution of the orphan drugs market
4.2.The orphan drugs market is expected to undergo continued growth
4.3.What is commercial excellence in orphan drugs?
4.4.Traditional measures of success are not appropriate for orphan drugs
4.5.One of the most important factors is the number of patients that have been helped
4.6.Access is seen as a critical success factor for orphan drugs
4.7.Why is commercial excellence in orphan drugs important?
4.8.What are the key hurdles for orphan drugs?
4.9.Identifying the appropriate unmet need
4.10.The execution of effective clinical trials
4.11.Improvement is still needed in regulatory processes
4.12.Market access is a particular issue for orphan drugs
4.13.Even after a successful launch, there are still challenges for orphan drugs

5.Stakeholder engagement
5.1.Why is stakeholder engagement important?
5.2.The treatment of orphan diseases tends to be concentrated amongst a small number of experts
5.3.Case study 1: Birmingham Health Partners Rare Disease Centre initiative
5.4.Key stakeholder groups that should be engaged
5.5.Patients should be engaged throughout the lifecycle
5.6.Patient advocacy groups
5.7.Case study 2: PPMD submits patient advocacy initiated draft guidance
for a rare disease
5.8.Patients are critical in providing insight into the disease
5.9.Staying close to patients can serve as a reminder of the importance of
orphan drugs4
5.10.Patient engagement must be conducted in a sensitive manner
5.11.Patient engagement needs to start as early as possible
5.12.Physicians are critical to orphan drug success
5.13.Rare disease KOLs communicate and work with one another
5.14.KOLs have a critical role to play in the development and pre-launch phase for
orphan drugs
5.15.Post-launch, KOL engagement is vital for commercial success
5.16.KOL engagement should be as early as possible
5.17.Payers are equally as important as patients and physicians
5.18.Although market access is not currently a major challenge,
5.19.orphan drugs still have to demonstrate value
5.20.Payers are frequently being engaged in order to ensure clinical trials
are designed appropriately
5.21.When is the best time to engage payers?
5.22.The regulatory authorities are seen by many as an important stakeholder
for orphan drugs

6.Commercialisation of orphan drugs
6.1.Collaboration and acquisition is driving the landscape for orphan drugs
6.2.Big pharma is expected to dominate in orphan drugs
6.3.The structure of orphan drugs teams in big pharma
6.4.Specific rare diseases teams
6.5.Absorbing orphan drugs into therapy area teams
6.6.Maintaining acquired orphan drug specialists as separate entities
6.7.A separate rare diseases operation is seen as the most effective structure
for big pharma
6.8.The benefits of being a smaller pharmaceutical company
6.9.Alliances and co-operation tends to be favoured
6.10.Reasons for collaboration in orphan drugs
6.11.Consolidation and co-development likely to continue in the orphan drugs space

7.Best practices
7.1.Case study 3: Genzyme - an orphan drugs pioneer still leading the way
7.2.Factors for Genzyme's success
7.3.Case study 4: Shire - putting rare disease at the centre
7.4.Shire's acquisition activity has established its orphan drug presence
7.5.Shire will continue to be a leader in orphan diseases
7.6.Case study 5: Roche activities in orphan disease
7.7.The Genentech acquisition
7.8.Genentech has a strong history with orphan drugs
7.9.Roche is building on its successful Genentech merger with other acquisitions
in the orphan drugs area

8.The future outlook
8.1.Market access is expected to become more challenging
8.2.Patient involvement in orphan drugs will continue to grow
8.3.Venture philanthropy is an area where patient advocates are increasingly involved
8.4.The Cystic Fibrosis Foundation courts controversy with Kalydeco
8.5.Smaller companies will continue to be of critical importance
8.6.Smaller biotech companies will have to continue to make alliances in order to succeed
8.7.Recommendations to companies in orphan drugs
8.8.Developing a “fit for purpose” orphan drug is key
8.9.An accurate assessment of unmet need is critical
8.10.Flexibility during commercialisation is needed
8.11.Early engagement with all stakeholders is fundamental to commercial excellence
8.12.Collaboration will help boost commercial excellence

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