ENSURING REGULATORY COMPLIANCE
Drugs and other products manufactured for human consumption must comply with governmental or industry-spe-cific regulations. For pharmaceuticals, it is the current Good
Manufacturing Practices (cGMP) of the FDA. Food grade and
kosher regulations may apply to food and nutritional products.
During the R&D phase, companies can minimize expenditures by producing test quantities using non-compliant
batch production methods. However, converting these
processes to meet regulatory requirements for scaled-up
commercial production can be extremely time-consuming
and costly. Frequently a change in facilities is also needed,
further complicating matters.
In the production of pharmaceutical products, cGMP
regulations, for example, require that all commercially produced drugs and pharmaceutical products meet stringent
assay, quality, and purity requirements. Facilities must have
appropriate quality management systems in place that can
detect, investigate, and correct product quality deviations.
Investigational new drug (IND) submissions to the FDA can
easily be delayed and rejected by insufficient data, inadequate reporting or insufficient cGMP reference standards.
This may necessitate rapid preparation of clinical trial
batches and validation and/or production of GMP-grade material to serve as a reference standard itself.
Even if the custom compound is the active pharmaceutical ingredient (API), and therefore does not require cGMP
certification, the supply of specialized intermediates and
precursors for life science applications may necessitate specific ISO certification on the commercial scale. This is becoming increasingly relevant as medical device companies
request custom synthesis services for new excipients and
components for novel drug-device combinations.
SECURING ADEQUATE FUNDING FOR PRODUCT
DEVELOPMENT AND MANUFACTURING
While there are many potential sources of funding for
product development, obtaining funding is nonetheless
highly competitive, and each investor or funding organization will have different requirements. Funding sources
include venture capital (VC) groups, angel investor consortiums, and grant opportunities such as Small Business
Innovation Research (SBIR) available through governmental agencies such as the National Institutes of Health.
Identifying the proper grant options for the technology in
question, as well as employing experts with grant-writing
expertise, is of paramount importance. It is vital for start-up
organizations to “get in front” of VC and angel boards to
make a pitch for their novel technologies. External vendors
and partners with existing relationships with such funding
organizations are attractive options for young companies in
need of capital.
In addition, companies can also license their technology
PROTECTING INTELLECTUAL PROPERTY
to commercial partners with synergistic or complementary
technologies. Big Pharma typically leverage their resources
in this way to bolster R&D pipelines. In order to do this,
however, proof-of-concept work, data collection, and anal-
ysis must be conducted to convince potential investors to
fund its product development activities. This is often one
of the most expensive and difficult steps in the life of a
start-up. While these fledgling companies typically confirm
the bioactivity of a drug candidate on their own, the ability
to prepare a comprehensive technical package suitable for
licensing or transfer often remains beyond their internal
capabilities. Thus, it is important for these outfits to identify
external resources capable of handling synthesis, testing,
and formulation work at all scales.
Companies must balance the need to avoid any patent
infringements or protect their own intellectual property (IP),
and safely share their confidential process information with
development partners. IP should be cross-referenced against
existing patents and then protected during development
and technology transfer. While this is typically conducted
internally by legal staff or through a contracted external law
firm, any perceived gaps may need to be addressed through
additional laboratory work. For instance, a start-up may need
to prepare additional patent example compounds, quickly
synthesize competitive samples, perform analytical mea-