cell and gene therapy commercialization innovations

Beth Schurman, Brian TorresHerspiegel Consulting

CELL AND GENE THERAPY COMMERCIALIZATION INNOVATIONSThe Best Path to C&GT Commercial Success

[email protected]

267.759.2130

www.HerspiegelConsulting.com

1040 Stony Hill Road, Suite 125

Yardley, PA 19067

mailto:[email protected]

https://www.herspiegelconsulting.com/

AbstractThe cell and gene therapy (C&GT) market is maturing and near-term growth is accelerating. There are over a dozen cell and

gene therapies already approved in the U.S. and more than 350 additional treatments in development.

Innovation in cell and gene therapy brings new commercial challenges. Each new commercialization of a cell and gene therapy

treatment has unique roadblocks as physicians, patients, and payers acclimate to the science of these new cell and gene

therapy entrants, as well as their evolving approaches to commercialization and distribution.

In this whitepaper you will discover:

1. The four most common challenges in cell and gene therapy commercialization

2. Methods to address common product launch challenges

3. Insights to accelerate and de-risk launch for cell and gene therapy treatments

Commercialization of cell and gene therapy treatments can provide significant value across advanced therapy medicine

products (ATMPs), driven by a combination of their clinical benefit and durability, as well as being able to treat the root cause

of disease and provide curative effects.

In such a nascent field, few organizations have experience to inform the best path to commercial success. Herspiegel

Consulting has supported numerous cell and gene therapy (C&GT) companies with their first commercialization efforts.

1 Cell and Gene Therapy Commercialization Innovations

Cell & Gene Therapy Overview The FDA anticipates receiving more than 200 INDs and approving 10 to 20 C&GTs for each of the

next 7 years. This market development path has been likened to the mainstreaming of monoclonal

antibodies in the 1990s.

The Alliance for Regenerative Medicine provides these helpful definitions of different types of ATMPs:

Commercial leaders of cell and gene therapy

should focus on four key areas of complexity:

1. Treatment logistics and customer experience

2. Driving patient referrals

3. Enabling access / managing customer financial risk

4. Establishing safety and clinical value

Gene Therapy

Seeks to modify or introduce genes

into a patient’s body with the goal

of durably treating, preventing, or

potentially even curing disease.

Currently studies focus on oncology,

viral diseases, and inherited

disorders.

Genome Editing

A technique by which DNA is

inserted, replaced, removed, or

modified at particular locations in

the human genome for therapeutic

benefit in order to treat cancer, rare

inherited disorders, HIV, or other

diseases.

Cell Therapy

The administration of viable, often

purified cells into a patient’s body

to grow, replace, or repair damaged

tissue for the treatment of a

disease. A variety of different types

of cells can be used in cell therapy.

2Cell and Gene Therapy Commercialization Innovations

Cell & Gene Therapy Challenges

Launch success hinges on robust

customer support for these complex

therapies. Herspiegel Consulting uses

benchmarks to define an optimized

customer experience for all stakeholders

involved and helps commercial teams

design and implement an optimal supply

chain and service structure.

Challenge 1: Treatment Logistics and Customer Experience

Cellular-based treatments often require precise manufacturing and

logistics, starting from therapy to first use in patients. The product

needs to arrive at exact moments in the patient journey. Chain of

custody and chain of identity requirements pose unique challenges

for C&GT, which traditional medical product supply chain logistics

aren’t prepared to handle. The cell therapy supply chain must be

carefully planned with scenario plans in place for disruptions along

the path.

Weather and travel restrictions can disrupt delivery or receipt of

cell therapies. When a severe storm hit the Northeast on the day

of a blood stem cell donor collection, the storm made it impossible

for the donor to reach the hospital, risking the entire logistical

timeline. When a hurricane along the Texas coast grounded air

traffic, a hospitalized patient was awaiting arrival of their therapy.

Contingencies of all sorts must be considered in planning for a

dynamic supply chain.


There is a critical need for a high level of synchronicity among various stakeholders and processes to ensure a

successful C&GT supply chain. Regional transport regulations make the process even more complex. In some

cases, long vein-to-vein time can impact patient eligibility. Once on-site and temperature acclimated, cells typically

must be used within a few hours, requiring healthcare providers, patients, and other treatment center staff to

coordinate very tight windows of time for therapy administration. Even for ATMPs with a centralized production

location, transport sensitivities, limited shelf life, and stakeholder coordination remain challenges.

Treatment center accreditation is expanding, but still limits the availability of cell therapy across the U.S. On

top of the supply chain logistics, commercial teams face two major challenges in this regard. The first relates to

geographic distribution of patients and their access to accredited treatment centers. The second is the complexity

of treatment and burden on centers and manufacturers in establishing protocols. Complex ordering systems and

staff training efforts may further limit true capacity.

Figure 1: Heatmap of the number of cities

with ≥1 FACT-accredited center

There are approximately 221 centers

accredited by the Foundation for

Accreditation of Cellular Therapy

(FACT) that are attached to major

health systems. These centers cater

to adult patients and would be eligible

for the Tumor Infiltrating Lymphocytes

(TIL) cell therapy.



Key questions to investigate:

• What are the characteristics of prescribers

willing to recommend C&GT to their patients?

• What criteria and process drive patient

identification?

• What impact might the referral have on the

prescribers’ practice?

• What information do the prescriber and other

stakeholders in the network need to know to

make the referral?

• What is the likely referral network among

target prescribers and treatment centers?

Referral decisions have heavy emotional components and are often irreversible. As such, patients will require

significant information for product selection. This will likely include talking to other patients, working with

advocacy companies, and detailed online searches. A comprehensive market development plan to support

education and referral network development should be adopted early in commercialization planning for C&GT.

As C&GT enters new therapeutic

areas, commercial leaders will need to

understand critical referral drivers and

map referral patterns to inform go-to-

market strategy. Herspiegel Consulting

leverages therapeutic area knowledge,

years of go-to-market planning, and

market shaping success patterns to direct

efforts for community and treatment

center engagement.

Challenge 2: Driving Patient Referrals

C&GT is a disruptive innovation that will shift clinical paradigms

from treatment to disease modifying or curative. It also disrupts

the traditional provider patterns, in many cases shifting care from

community providers to accredited treatment centers. Nearly

all current C&GTs are administered within inpatient settings at

accredited centers. In chronic disease cases, this dynamic will require

referral from community prescribers (which potentially disrupts their

existing business model). Early efforts to identify probable referral

patterns, and drive awareness and willingness to refer, will be critical

for commercial success.


Cell & Gene Therapy Challenges Early engagement with payers and treatment center stakeholders is critical to achieving access

and pricing goals. Success will be driven by efficacy data to support value, leveraging partnerships

to drive innovative models and policy changes. Patient advocacy groups can play a large role

in influencing standards of care. Manufacturers will benefit from approaching reimbursement

challenges in a collaborative, solution-oriented model with all the key stakeholders.

Challenge 3: Enabling Access / Managing Customer Financial Risk

The value proposition for C&GT is attractive, but therapy costs can top $1 million per patient. For example,

Novartis’ Zolgensma gene therapy for spinal muscular atrophy is currently the world’s most expensive drug to

date at $2.1 million per patient. Add to that the instances where some patients have had a failed treatment using

Zolgensma and had to add the costly Spinraza (Biogen, $600K) and payer frustrations are compounded. With gene

therapy being a one-time, curative solution, the pricing model has had to change from the historic acute or chronic

care medicine cost model. Further, as utilization is still low, and in some cases only targeting rare or ultra-rare

conditions, development costs must be recouped from a small number of treatments across the population.


The high costs and limited data sets combine as a difficult challenge in proving value to patients, payers, and the

treatment centers managing administration. Payers struggle with the high up-front costs for products that have

yet to demonstrate long-term outcomes. Treatment centers also balk at preparing patients who may not wind up

getting the treatment, and for follow on care for those that do. Some value-based agreements set efficacy points

early enough that non-responders would not yet be identifiable. In the inpatient setting, current diagnosis related

group (DRG) payments are insufficient to cover the cost of cell therapy related care. Even with outlier payments in

the new technology add-on payments (NTAP), hospitals expect to incur a loss on Medicare patients treated with

cell therapy. If C&GT shifts to outpatient care, Medicare Part B would reimburse using the average selling price

(ASP) plus model, supporting better cost recovery for providers.

Payers are largely evaluating the innovative therapies on a case-by-case process per patient. The process can be

burdensome on treatment centers working to demonstrate patient eligibility. Unfortunately, for patients with

aggressive disease, any delays can result in deterioration and impact eligibility to undergo the treatment. The

future may hold new direct-to-payer models instead of the traditional system of selling to hospitals under the

DRG.

Due to the significant up-front cost of therapies, innovative reimbursement models have come into play. Spark has

negotiated both short-term and long-term durability measures for Luxturna™, tied to the rebate payment model.

This poses a risk that it undercuts Medicaid best price rules. Policy has yet to catch up to this evolving market.

At launch, Novartis established a 30-day guarantee for Kymriah. Based on the overall 83% remission rate in

pivotal ELIANA trial, Novartis was likely to see full price in most cases. A relapse within the first year might occur,

but the 30-day guarantee wouldn’t address that.



“There are three primary characteristics that investors are seeking in their cell and gene therapy portfolios: strong management, solid technology, and existing financing. Management teams that know how to pivot, or adopt an agile mindset as the business evolves, are extremely attractive.”

– Datamonitor reporting on Alliance for Regenerative

Medicine’s Cell & Gene Therapy Investor Day 2019

Challenge 4: Establishing Safety and Clinical Value

There are still traditional elements within this innovative space, especially when establishing safety and clinical

value. Both short and long-term evidence generation will remain foundational for success. Providers, payers, and

patients want to be assured of the safety and efficacy of the treatments and the risk/benefit vs. standard of care

treatments. New data analytics tools may allow for efficient RWE tracking to fulfill post-approval commitments

for long-term safety and efficacy data.

A robust understanding of the patient journey from identification through treatment and treatment center

challenges will help inform commercial activities. Healthcare providers will need to be aware of and comfortable

with the treatment options, understand how to identify appropriate patients, and know how to refer. A large

variety of stakeholders in treatment centers will need to be trained and sometimes certified. Support systems

need to be in place to help patients and treatment centers navigate the process.


Organizations that are expert in

efficiently planned investment

and insight driven promotion

will establish themselves in

this growing market. A robust

marketing program designed to

raise awareness, address concerns,

and solve challenges for customers

and all treatment stakeholders

will help drive successful

commercialization and increase

confidence in safety and clinical

value.

A stakeholder benefit map can

be a useful framework when

considering the approach for go-to-market strategy and promotional planning. The stakeholder benefit map can

quickly assess which stakeholders will be the most influential in driving or restricting adoption. Investigation

through market research or key opinion leader engagements can fill in knowledge gaps to complete the

stakeholder benefit map. Commercial teams will need to define the value messages and resources needed per

stakeholder group. An experienced partner can help to manage these complex mappings and direct successful

marketing of C&GT.


ConclusionCell and gene therapy is poised to address significant healthcare needs and change the treatment dynamic for many rare

and not-so-rare conditions. The future may even hold promise for C&GT to cure large-scale diseases, such as diabetes, heart

disease, and autoimmune disorders.

Commercial leaders of C&GT need a clear understanding of the barriers to adoption in their market. Understanding and

planning for four key areas can support success:

1. Treatment logistics and customer experience

2. Driving patient referrals

3. Enabling access and addressing customer financial risks

4. Establishing the safety and clinical value of the treatment

In such a nascent field, few organizations have experience to inform the best path to commercial success. Herspiegel

Consulting has helped numerous C&GT companies with their first commercialization efforts. Our experience addressing the

critical strategic issues of product commercialization and innovation in C&GT positions Herspiegel Consulting as a premier

commercial partner for C&GT companies.

Services:• Product Launch Preparation

• Commercial and Marketing Excellence

• Medical Affairs

• Market Access Strategy

• Patient Services Strategy and Implementation

[email protected]

267.759.2130

www.HerspiegelConsulting.com

1040 Stony Hill Road, Suite 125

Yardley, PA 19067

About Herspiegel Consulting

Herspiegel Consulting is a premiere life science consulting firm specializing in pharmaceutical and biotech commercialization. We help

our clients define impactful strategic approaches, streamline pharmaceutical product launch and commercialization processes, enhance

business intelligence and optimize product performance. Herspiegel Consulting service offerings span new product planning, commercial

readiness, marketing, medical affairs, and market access.



http://www.herspiegelconsulting.com

Beth SchurmanPartner

Beth Schurman is a Senior Partner at Herspiegel

Consulting and an experienced marketing

professional with extensive experience in the

pharmaceutical industry. Beth has 12 years of

experience in strategy consulting and client-side

experience. Beth’s therapeutic areas are diverse

and include Diabetes, CNS, Dermatology,

Autoimmune Diseases, Virology, rare disease,

oncology, and medical devices.

Beth expertly blends marketing strategy,

innovative thinking, and rigorous structure

to establish market-changing strategies

and impactful execution for her clients. Her

commercial projects have spanned many critical

business areas including market assessments,

launch planning, go-to-market strategy,

distribution and patient services design, market

shaping strategy, business planning, scientific

platform development, lifecycle management

planning, and value story development.

Brian Torres is a Partner at Herspiegel

Consulting. His strong strategic and client

engagement skills enable him to lead complex

projects and support senior clients with their

most difficult business decisions. Brian practices

a team-centric approach, with a focus on

building strong, collaborative teams that can

quickly flex to deliver on pressing client needs.

While at Herspiegel Brian has led various

projects across a broad range of product types,

including C&GT, rare disease, oncology diabetes,

GI, and obesity, among others. These projects

have included product launch, patient services,

marketing, market access, digital health, sales,

and organizational design.

Prior to working at Herspiegel, Brian supported

the vaccine franchise at Merck Sharpe and

Dohme. He holds an MBA in pharmaceutical

management from Temple University and a BS in

biology from Saint Joseph’s University.

Brian TorresPartner

Want to know more?

For more information on how Herspiegel Consulting can help with your cell and gene therapy strategy, visit us at

HerspiegelConsulting.com or contact Beth Schurman at [email protected].


https://www.herspiegelconsulting.com/


cell and gene therapy commercialization innovations

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