Cell and gene therapies are designed to halt a disease in its tracks or reverse its progress rather than simply manage symptoms. They are often one-time treatments that may alleviate the underlying cause of a disease and they have the potential to cure certain conditions. Due to their potentially curative nature, cell and gene therapies can save significant costs for the healthcare system and restore productivity of the patient with economic benefits for the society overall. In contrast, many conventional medicines must be taken continually for weeks, months, or even for life.

The great potential of cell and gene therapies has triggered development of policies that establish dedicated regulatory frameworks to tackle challenges and specific needs around these advanced therapies. In particular in China, lots of movement in this area can be observed with many clinical studies in the field of cell and gene therapies, an increasing number of encouraging policies and special business zones that allow for collaboration between multiple stakeholders that contribute with complementary ideas and skills.

To further progress in this field and to make the country a major power for promising novel treatments, it is suggested to the Chinese government to continue on their way to open up for engagement in the area of advanced therapies.

This document continues the discussion about suggestions for an upgraded policy environment related to cell and gene therapies that builds on the suggestions made for the 2019 China Development Forum. This time’s focus lays on three areas, (1) Promoting R&D, (2) Optimizing Medical Infrastructure and (3) Enabling Patient Access.

Promoting R&D: the pharmaceutical industry welcomes and supports the intensive efforts of the various government administrations to regulate and promote R&D in rare diseases and particularly in advanced therapies. We recommend to pay additional attention to aligning regulatory and industrial policies and to upgrade the Foreign Investment Legislation in line with technological progress. In addition, fully implemented and enforced Regulatory Data Protection will support and incentivize the continued research and development of new medicines, in particular in the field of cell and gene therapy.

Optimizing Medical Infrastructure: High quality and safe patient treatment with advanced therapies requires an appropriate infrastructure to administer and investment in ongoing supportive care and patient monitoring. Building a world-class infrastructure could position China as a hub in the region, possibly not only serving Chinese patients, but also from other countries. Therefore, Novartis is working in a joint project with the China Medical Biotech Association in Beijing to draft a certification guidance that is in line with international standards and that is mandatory for all medical institutions carrying out cell and gene therapy.

Enabling Patient Access: The significant advances in the development of cell and gene therapy offer transformative benefits to patients, but also present a challenge to constrained healthcare systems. Therefore, mechanisms are needed to supplement the existing funding system by expanding the fiscal space of the healthcare system by a combination of additional components and increasing efficiency in using existing resources. It is suggested to the Chinese government to consider the establishment of specialized healthcare funds, promotion of supplemental commercial insurances, and expansion of upgrading the Basic Medical Insurance. Options for applicable pricing and payment models include outcomes-based payments and over-time payments.

The multinational companies, including Novartis, are happy to share its experiences gained in many places of the world and to constructively work together with the Chinese government to make cell and gene therapies a success for patients and the medical society and the country’s economy.

Download the full report：Special considerations to make cell and gene therapies a success in China