APR Nov/Dec 2022 - 50

»
BIOPHARMACEUTICALS
»
Source: PhRMA.org. A Decade of Innovation in Rare Diseases. Available at
http://phrma-docs.phrma.org/sites/default/files/pdf/PhRMA-Decade-ofInnovation-Rare-Diseases.pdf
Figure
3.
there is currently minimal patient follow-up data to know whether
these products will be cures or if the disease may return.
Adverse reactions are also a major issue, as serious adverse events10
(AEs) associated with these therapies are garnering more attention and
the mitigation of these AEs represent an unmet need in this emerging
field.11
This points to the need for recommendations regarding the
design of long term follow-up studies12 for the collection of data on
delayed adverse events following administration of a GT product.
Furthermore, many clinical trials have been put on hold to evaluate
adverse reactions amid troublesome reports13
disadvantages to using the Adenovirus, the most commonly used
vector in gene therapy clinical trials. Risks include non-integration,
immunogenicity, replication competence, no targeting, and small
insert size.
Other key areas of concern that include:
* Hepatotoxicity: liver damage
* Neurotoxicity: toxic substances alter normal activity of the
nervous system.
* Various types of cancer including acute myeloid leukemia, often
based upon animal models with tumor development.
* Autoimmune response: body attacks and damages its
own tissues.
* Use in immunocompromised patients and viral load concerns:
can mean the person is more infectious, such as breakthrough
infections of COVID-19.
50 |
| November/December 2022
Specialty Pharmacy Optimizes
Management of Gene Therapies
The evolving, critical role of Specialty Pharmacy (SP) in the
management of gene therapies cannot be understated. Ideally, they
maintain direct relationships with product manufacturers and offer
end-to-end solutions to facilitate payment reimbursement, support
processes and implement 'white glove' services with a high-touch
patient centered model that is focused on patient outcomes. SPs that
offer a full range of programs and services across the product lifecycle,
from pre-commercialization and market access, distribution, fulfilment,
and Hub services to compliance and monitoring for enhanced patient
outcomes, are best positioned to fulfill manufacturer expectations
for optimized product adoption, increased patient engagement and
better outcomes as well as streamlined communications between
prescriber, patient and pharmacy that decrease time to fill.
By employing a consultative approach to seamless new product
launches that provide early insights into market conditions, as well as
experienced teams to negotiate with payers, SPs ensure streamlined
access to therapies and advantageous pricing. Progressive SPs are
pioneering innovative technology-based suites of unique financial
solutions, including loan-based programs for cell/gene therapies to
help offset the high cost of curative medications and copay advisory
services to monitor and track manufacturer copay funds, ensuring that
allotted funds are utilized to help offset costs of expensive therapies for
patients. This also includes alternative funding programs which means
of access to philanthropic organizations, grants or other foundational
programs that support access to high-cost therapies and shift the cost
away from the patient and payer.
regarding the
Utilization Management Approaches to Lower the
Cost of Care
SPs apply programs such as step care therapy, quantity limits, partial
fill programs and other initiatives to control costs. The development of
criteria for prior authorization, for example, ensures appropriate use
and enhances opportunity for positive outcomes. Typically, criteria is
based upon clinical study inclusion and exclusion criteria, interpretation
of clinical study endpoints, national guidelines and CMS/Medicare
policies. Additional use of benefit maximum and deductible programs
and application of rebate management programs impact the cost of
care. Finally, by identifying alternative Site of Care programs, SPs can
direct patients to lower cost sites of quality care that can provide cost
savings to the patient and payer while making it more convenient for
patients, caregivers and support team members to access care.
Cell and gene therapies are at the forefront of innovation and
transforming how we treat certain diseases. Over the next few
years, more than 50 new in vivo and ex vivo gene-therapy launches
are already planned.14
with pharmaceutical
As the urgent need for research continues,
to map the genes
companies continuing
responsible for rare diseases and develop gene and cell therapies,
these manufacturers will want to identify and collaborate with the SP
community for optimal product market access.

http://phrma-docs.phrma.org/sites/default/files/pdf/PhRMA-Decade-of-Innovation-Rare-Diseases.pdf

APR Nov/Dec 2022

Table of Contents for the Digital Edition of APR Nov/Dec 2022