Regulatory Environment for Cell and Gene Therapies

By Poonam Balani

Cell and gene therapies include experimental methodologies using cellular/genetic material for the treatment of life-threatening diseases without available treatment options. The innovation in this rapidly expanding area was spearheaded primarily by academia and small biotech players. The field now attracts investment from big pharma companies owing to mounting scientific evidence of successful proof-of-concept for cell and gene therapy methods.

Globalization of the medical industry poses a significant challenge for the pharmaceutical companies as drug approval is often delayed and is cost intensive due to the diversity in the regulatory requirements of different regions/countries. Furthermore, lack of clear regulatory requirements in the developing countries, which are potentially large markets for such drugs, might delay the approval process.

The international regulatory forum on human cell therapy and gene therapy products has called for regulatory convergence, to facilitate alignment of regulatory requirements across regions/countries as they gradually adopt the international guidelines for reduced approval time of cell and gene therapeutics. Additionally, a ‘minimum consensus package’ was also proposed as a common internationally applicable technical requirement[1]. In order to realize the full potential of the promises held by cell and gene therapies, there remains an urgent unmet need of accelerated programs to fast-track the innovation and approval, relying on the realistic risk assessments regarding scalability, safety and potency of this new technology.

The US landscape

The FDA launched its Advancing Regulatory Science Initiative in Feb 2010, overseen by the Office of Regulatory Science and Innovation. FDA my grant regulators relief for regenerative medicine (RM) depending on the product characterization. In the US, stem cell–based therapies are classified as ‘‘human cells, tissues, or cellular or tissue-based products’’ (HCT/Ps). These are defined as ‘‘articles containing or consisting of human cells or tissues that are intended for implantation, transplantation, infusion, or transfer into a human recipient.’’[2] This category is subject to regulatory requirements for HCT/Ps i.e. establishment registration and product listing, donor eligibility, and current Good Tissue Practice. On the other hand, biological cell therapeutics are subject to both, the Public Health Service Act and meet the definition of drugs under the Federal Food, Drug and Cosmetic Act (FDC Act).

Despite the most favorable environment in the US for cell and gene therapeutics, the first approved trial of human embryonic stem cells was abandoned by Geron Corporation in 2011, citing high research costs and regulatory complexities. The trial was resumed only after a fresh round of funding as Geron entered into Asset Contribution Agreement with BioTime, and is scheduled to be completed by 2018.

Japanese regulatory environment

The Japanese government has taken several proactive steps to accelerate innovation and commercialization of cell and gene therapeutics by clearing new legislations i.e. the Act on the Safety of Regenerative Medicine”(ASRM)[3] and the Pharmaceuticals, Medical Devices and other therapeutic products Act (PMDA)[4], effective since November 2014. PMDA identifies the need for collaboration between medical research institutes and the medical industry and introduced a new fast-track option for conditional, time-limited approval of “regenerative medical products”. ASRM charts the guidelines ensuring safety of stem cell therapies, regulates practices and clinical studies related to regenerative medicine as well as the manufacturing and quality requirements for cell and gene

It is believed that these regulatory changes in Japan would enable approval of new therapeutics in approximately 3 years as against 7 to 10 years before the implementation of PMDA and ASRM[5].

Advanced Therapy Medicinal Products (ATMPS) in the EU

ATMPs are classified into four categories as

  1. Gene therapy medicinal products (GTMP),
  2. Somatic cell therapy medicinal products (sCTMP),
  3. Tissue engineered products (TEP), and
  4. Combined ATMPs, which contain a medical device as an integral part of a cell- or tissue-based product.

EU allows centralized authorization of ATMPs and defines the technical standards and also provisions incentives for small- and medium-sized enterprises. ATMPs must demonstrate safety, efficacy and quality before they can be marketed. Another essential requirement is the traceability of all raw materials used in the development of ATMPs. The current marketing authorization procedure in the EU for ATMPs is considered to be extremely challenging for prospective applicants. The regulators also agree that necessary changes need to be made to the regulatory procedures to attract new investment, carefully balancing the risks and benefits on an individual basis.


Success of cell and gene therapeutics is dependent on the harmonization of the regulatory requirements, easing the burden on the stakeholders. Japan has been able to attract large investments and partnerships with foreign players after passing the new legislations to accelerate approvals of new therapeutics. The regulatory authorities in the US and Europe are also looking into ways to expedite approvals and adopt less risk averse pathways for market authorization. A predictive regulatory support and harmonization of regulatory requirements is expected to boost investments in this emerging technology.




[4]  5

[5] New Japanese initiatives on stem cell therapies. Cell Stem Cell. 2015;16:350-352.

Tags: cell therapy , gene therapy , biotech , regulatory environment , pharma , pharmaceutical

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