Landmark Month Sees Second Gene Therapy Approved and Other Wins

RxC International Gene Therapy Newsletter

October was a landmark month for gene therapies with the approval of the second-ever gene therapy in the US, Yescarta (axicabtagene ciloleucel) from Kite and Gilead.  The prior approval of Novartis’ Kymriah is more proof that gene therapies are set to shake up the pharma industry.  Gene therapies saw other wins as Luxturna, Spark’s treatment for Leber congenital amaurosis, received the FDA’s recommendation for approval and Bluebird Bio’s interim data report for its gene therapy Lenti-D to treat cerebral adrenoleukodystrophy showed promising results.

That’s not to say it was all smooth sailing.  Pricing remains forefront in the discussion of gene therapies’ value and potential to “break the bank.”  And not all therapies are succeeding as Alnylam suspended its RNAi fitursiran trial after a patient death.

RxC International will continue to closely monitor gene therapy developments to provide readers with news and insights into this ever-evolving market.


Pricing Debate Intensifies with Approval of First Gene Therapies

The recent approvals of the first two gene therapies in the United States have intensified the debates over pricing and the financial burden these treatments may have on the healthcare system.

While gene therapies are emerging onto the commercial market, the debate surrounding affordability is not new.  Dating back to the era of Lipitor and other blockbuster PCP-driven drugs to the expansion of the oncology market to the more recent emergence of multiple rare disease treatments, the question has always been the same: “How can we afford them?”  The real question, however, is not whether we a society can afford gene therapy treatments, but whether we can afford not to pursue treatments that extend or improve people’s lives.


Gene Therapy Pricing and Target Patient Population


The article offers some important insight into the pricing debate, but at the same time makes the fundamental mistake of grouping all gene therapies into a single category.  Gene therapies are far from a homogeneous group so must be thoughtfully segmented to fully understand the different dynamics at play for each product.

The chart seems to purport an overall correlation between pricing and target patient population, however it doesn’t take into account the basic HEOR measure of cost per year of life saved.  While gene therapies targeting rare diseases may have a smaller patient pools, they often target childhood diseases with the potential to add decades to each patient’s life. Oncology is a very different dynamic.


Harvard Scientists Reveal New Gene Editor

Using CRISPR-Cas9 technology, scientists can now change a single DNA letter.  This advancement expands the capabilities of the first base editor, created last year, by converting additional bases.

While CRISPR continues to make strides for gene therapy advancement, it is still a long way from creating commercial therapies.


Research Updates

Second Gene Therapy Treatment Approved

The October 18th approval of Yescarta (axicabtagene ciloleucel), a CAR-T treatment for adults with relapsed or refractory large B-cell lymphoma, places Kite and Gilead in the new market of approved US gene therapies. Novartis’ Kymriah, the first gene therapy to gain approval in the US, is also a CAR-T therapy.  While currently aimed at different oncology segments, the two CAR-T treatments could become competitors as both brands seek to expand product labels. 


Fitusiran's Recent Failure Opens Door for Additional Therapies

The September suspension of trials for RNAi fitursiran following the death of a patient in one of the studies has opened the door for two competing gene therapies that offer long-term benefit from one administration.


Gene Therapy Receives Unanimous Recommendation for Approval

Luxturna, a treatment for a hereditary form of blindness, received recommendation for approval from an FDA advisory committee.  Spark Therapeutics, the treatment’s developer, is expected to receive a decision from the FDA by January.


Encouraging Results for Gene Therapy Treatment of Fatal Neurodegenerative Disease

Interim data for Bluebird Bio’s gene therapy treatment Lenti-D for boys suffering from cerebral adrenoleukodystrophy (CALD) showed encouraging results.  Of the 17 boys who received treatment as part of Bluebird’s trial, 15 were alive and stable after 24 months. "The clinical experience with Lenti-D demonstrates the potential for gene therapy to benefit patients with CALD," said David Davidson, MD, chief medical officer of Bluebird Bio.  The company is currently considering its plans for FDA approval.

About RxC International

Understanding the subtleties of the gene therapies is critical for bringing these treatments to market. RxC International has extensive experience successfully commercializing and launching new drug products, developing innovative solutions, realizing a product’s best potential, and working across organizations to achieve common goals.

To receive updates from RxC International, follow us on LinkedIn. Visit our Gene Therapy Resource Center for other posts and resources on launching and commercializing gene therapies.

About the Author

Richard Tinsley is a Strategy Partner at RxC International with over 20 years of life sciences consulting experience. He is a recognized leader in strategy, commercialization, life cycle management, and sophisticated forecast modeling. Rich has deep expertise in specialty markets such as rare diseases, HIV, oncology, neurology, and diagnostics.

Effective Pharmaceutical Life Cycle Management Planning: Optimizing Product Value over a Drug's Lifespan

Effective Pharmaceutical Life Cycle Management Planning: Optimizing Product Value over a Drug's Lifespan

Developing an effective drug life cycle management plan is critical for maximizing the value of a pharma product over the course of its commercial lifespan. Drawing from our deep expertise of pharmaceutical product lifecycle management, RxC International has identified key success factors and industry best practices as well as common pitfalls to avoid when developing and implementing LCM plans.

Partnering for Successful Life Cycle Management: 5 Reasons to Seek Outside Help

RxC International Life Cycle Management.jpg

Pharmaceutical companies with effective Life Cycle Management (LCM) planning typically work with outside partners to develop and execute LCM plans.  Working with a third-party partner adds outside thinking and best practices to an organization.  “When you can [bring] in the experience that you need, you’ll benefit from the hard work that others have done, shorten your time to market for new products, and introduce new talent to your company,” writes Madeline Bennett of The Telegraph.

Below are the five most valuable reasons to bring in outside help to assist with product life cycle management.

Top Reasons to Engage Outside Resources

1. Knowledge of Industry Best Practices and Innovative Solutions

Hiring professionals who routinely help drug companies develop and optimize LCM plans provides access to the industry’s best practices without significant training costs for permanent staff.  Outside partners with expertise in LCM know what has worked for other companies and what hasn’t, and can use this knowledge to effectively navigate your LCM challenges.

“By hiring externally, companies can instantly gain specific skills for certain projects, get an outsider's perspective without emotional investment to the business and augment their team to give them more resources for time-dependent tasks,” writes Ben Judah at Entrepreneur. Experienced outside partners have also seen the mistakes of other companies and know how to avoid those mistakes.  This experience is especially crucial in today’s market that penalizes any commercial delay.

2. Proprietary Frameworks and Models

An experienced outside partner will have proprietary LCM frameworks and models that have been developed, tested, and improved upon by real-world application.  Working with a partner that has these models in place avoids the trial-and-error process that can waste precious internal time and resources. Access to your external partner’s frameworks also helps ensure that your LCM is complete and spans the lifecycle of your product.

3.  Consistent LCM Process

For pharma companies with multiple brands, putting in place an effective process is critical for developing LCM plans for each brand.  A comprehensive life cycle process that is objectively and consistently executed allows companies to thoroughly explore potential concepts, foster collaboration between departments, and uncover more opportunities for innovation.

An external partner with experience developing life cycle processes can help your company establish its own process for LCM planning.  Outside participation also helps avoid the perception of functional over-reach, ensures broader organization alignment, and better integrates input from multiple departments and stakeholders.

4. Cross-Functional Engagement

An experienced external partner has the tools and resources necessary to engage cross-functional experts from clinical, commercial, medical, manufacturing, tech-ops, and regulatory departments.  This is important for idea generation and organizational alignment. 

Additionally, an outside partner can help key stakeholders within an organization understand the importance of LCM and provide them the framework to help ensure successful plan development and execution.

5. Integrated Solutions

External partners with diverse expertise offer the best choice for partnerships because they bring to the table comprehensive industry knowledge and a team of experienced experts. “Working with a...partner that can deliver integrated solutions across a broad set of specialized areas can drive enhanced value, increased synergies, [and] stronger patient relationships,” notes Joe DePinto, Specialty Pharmacy Times.  This is especially true for LCM which requires extensive understanding of the pharma landscape.

External partners can help bring all the key components of LCM together, from the initial planning stages through to implementation.

About RxC International

RxC International, a life sciences management consulting firm with deep operational and strategic expertise, has collaborated with many drug companies to successfully develop and improve LCM plans as well as internal life cycle planning processes. 

We leverage our experience to apply best practices and proprietary frameworks to every client engagement and are particularly experienced at assisting companies with developing innovative LCM strategies and performing in-depth analysis of existing LCM plans to identify areas for improvement.

To see how we can help your company develop new LCM plans or optimize existing plans, please contact us or email Frank Koos, Head of Business Development, at  To receive updates from RxC International, follow us on LinkedIn.  Visit our LCM Resource Center for other posts, case studies, and resources on optimizing life cycle plans.

About the Authors

Philip Vorhies is a Strategy Partner at RxC International with over 20 years of biopharmaceutical experience. Philip has deep expertise in new product strategy and commercial excellence. He has successfully assisted numerous clients in the areas of product launch strategy, brand and portfolio strategy, and life cycle planning.

Nick DeSanctis is an Executive Partner at RxC International with over 25 years of leadership in the pharmaceutical industry. Nick is an expert in new product planning and driving portfolio value through portfolio management, life cycle management, target value profile development, and therapeutic area strategy.

The 8 Most Common (and Costly!) Life Cycle Management Pitfalls (and How to Avoid Them)

The 8 Most Common (and Costly!) Life Cycle Management Pitfalls (and How to Avoid Them)

Understanding the critical success factors of Life Cycle Management (LCM) planning helps pharmaceutical companies avoid common life cycle pitfalls that can lead to sub-optimal life cycle plans or ineffective implementations.  Companies that don’t optimize the life cycle management process and develop innovative strategies to meet the demands of today’s pharmaceutical landscape generally experience lower returns than their more proactive counterparts.

9 Common Challenges for Existing Co-Promotions

9 Common Challenges for Existing Co-Promotions

Successfully optimizing co-promotional agreements relies upon governance, brand management, and performance management. Yet, despite good intentions from each party, challenges can arise in a co-promotional agreement that threaten its longevity and overall success.  In our experience, preventing or overcoming challenges in existing co-promotions usually requires assistance from a third party like RxC International.  Below are the nine most common challenges we have encountered during our extensive experience with co-promotion programs.

How to Avoid Pharmaceutical Value Erosion: 5 Critical Components of a Highly Effective Life Cycle Management Process

How to Avoid Pharmaceutical Value Erosion: 5 Critical Components of a Highly Effective Life Cycle Management Process

A comprehensive Life Cycle Management (LCM) process is essential to developing pharmaceutical life cycle plans that deliver maximum value over the life of pipeline and marketed products. Ensuring that the life cycle process is objectively and consistently executed enables organizations to fully explore all potential concepts, foster collaborative thinking, and uncover more innovative opportunities. Life sciences companies with multiple products across several therapeutic areas can benefit from a consistent approach that allows management to make effective comparisons across the organizational portfolio and allocate resources accordingly.

The Importance of Early Life Cycle Management

RxC BioPharma Life Cycle Management

This post is the first in a series that distills RxC International’s robust experience in LCM strategy into best practices.  Please subscribe at our LCM Resource Center to receive future posts.

Companies must implement effective Life Cycle Management (LCM) strategies to build more value into products while they are still in development.  LCM includes identifying and prioritizing multiple potential indications and formulations while strategically planning their development and launch timing and sequence.  Competitive companies must also direct sufficient resources to a strong LCM process for their pipeline and marketed products.

Implementing an effective LCM process is critical to:

  • Meeting burgeoning R&D expenses
  • Off-setting impact of generic erosion on key brands
  • Combating encroaching competition
  • Maximizing product value from launch to loss of exclusivity

Winning in Life Cycle Management:  Having the Right Team

Developing innovative life cycle plans and ensuring their technical success requires a cross-functional approach across clinical, commercial, medical, manufacturing, and regulatory areas.  Regional participation in this approach is also important. In addition to assessing concept feasibility, cross-functional participation also increases key stakeholder engagement and ensures broad organizational support during the development and launch of critical LCM strategies.

When to Start LCM: Timing is Everything

The goal of LCM is to maximize a product’s value through its entire life cycle.  LCM planning, then, should start during the pre-clinical phase when the organization is in the process of selecting the lead indication.  During this time a designated LCM team can identify potential indications based on the product’s mechanism of action as well as assess clinical and regulatory feasibility before completing and prioritizing commercial valuations.  This process enables the company to best determine the lead indication as well as strategically plan the launch timing of follow-on indications or formulations.

If a product’s life cycle has passed pre-IND timing, the next best time to begin LCM planning is when proof of concept has been achieved in phase 2 for the lead indication.  This provides greater assurance that there’s a path forward for the product, freeing the company to focus resources on identifying LCM strategies and initiating early planning to realize the most value over the product’s life cycle.

The justification for developing LCM strategies prior to approval and ideally early in development is clear.  Research has indicated that sales trajectories are often established within the first 12 weeks of launch.  The chart below illustrates the significant increase in revenue generated with early LCM planning compared with more traditional LCM planning approaches.

RxC Life Cycle Management

The Critical Role of a Third-Party Partnership for Successful LCM

Biopharma companies that embrace the importance of effective LCM planning and recognize the value it injects into their product portfolio often rely upon an experienced third-party partner for assistance with developing and implementing LCM plans.  RxC International has significant expertise in LCM and has successfully worked with clients to optimize their existing LCM processes or to create and implement best practice LCM processes.  We have also partnered with clients to develop product-specific LCM plans to ensure that full product potential is realized.

For a complimentary evaluation of your LCM process or a specific product LCM plan, please contact us or email Frank Koos, Head of Business Development.  To be notified of future releases of LCM information, please subscribe on our website.

Optimizing Co-Promotion Effectiveness

Optimizing Co-Promotion Effectiveness

As the complexity and investment for successfully marketing a prescription drug or biologic rises, many companies seek to de-risk their commercialization effort by joining forces with a partner biopharma company.  Co-promotions, or the formal relationship established between two companies to combine resources for a more effective promotion of a product, are becoming increasingly common in the biopharma industry.  While co-promotions are structured with the right intent, these agreements require careful consideration of several key strategic and operational issues in order to achieve optimal effectiveness.

Three Key Co-Promotion Drivers

A structured approach early-on in the formation of a co-promotion agreement can significantly reduce pitfalls and difficulties later on in the process.  To facilitate a successful co-promotion structure, three key co-promotion drivers must be optimized:

  1. Governance: Structuring a Winning Co-Promotion

  2. Brand Management: Optimizing Brand Planning and Execution

  3. Performance Management: Ensuring Optimal Performance

Successful co-promotions have several key factors in common: they are very focused, they have shared objectives, and their team members are committed to the process and outcome.


A successful co-promotion requires the governing body to fully support the initiative and foster a culture that reflects the shared values of both companies.  Governance is the key to ensuring that co-promotion teams work together effectively to provide the framework for undertaking the brand lifecycle management and performance management processes.

Brand Management

Brand strategic planning is a way to develop and communicate brand strategies across affiliated co-promoting companies.  A well-executed brand planning process provides alignment among brand teams, clear guidance on the brand objectives (near-term vs. long-term), supporting strategies/tactics, resource allocation, performance targets, and the leads for each tactical initiative.  While each company will have its own planning timetables and formats, the co-promotion should accommodate each partner’s own timeline.

Performance Management

To effectively oversee co-promotion, governance teams must establish objectives and supporting performance management processes to hold teams accountable.  Incentives should be designed to increase accountability and drive results at every level of the co-promotion.  The governing body must facilitate strategic issues, day-to-day operational challenges, mechanisms for monitoring performance, and the timely resolution of issues. Performance management must also include evaluating team effectiveness, team dynamics, and collaborative behaviors.

Achieving Optimal Performance and RxC International Assistance

The optimization of the above three key drivers ensures that supporting processes are in place, strategic brand life cycle management processes are consistent, and performance targets are clearly defined.  Often, achieving successful performance outcomes requires the expert evaluation and insight of a third party.  At RxC International, we have extensive experience optimizing both new and existing co-promotions.  

For companies experiencing challenges in a current co-promotion arrangement, we have developed a co-promotion effectiveness diagnostic framework.  The framework helps us work with companies to identify areas that need realignment and generate an action plan for co-promotion optimization.  If you would like more information about RxC International’s co-promotion capabilities,  if you would like to request a complimentary evaluation of your co-promotion partnership, or a copy of our latest whitepaper on this topic, please contact us today or email Frank Koos.