The classic “one product, one regulatory track” model that worked for decades for small molecules has become economically unsustainable for innovative biomedicine, where development costs exceed $2 billion. A new regulatory approach is needed – one that allows reuse of quality, stability and preclinical data for a reproducible, well-understood technology, and permits simplified registration of new biologics if they are built on a validated platform. Such an approach is already used in Japan, the United States, China and Europe.
The traditional model – taking seven to ten years to develop a single product – is no longer economically viable, especially in gene therapy, genome editing and personalised medicine, Oleg Lavrov, adviser to the director general of the Innopraktika Innovation Engineering Centre, told the Sirius.Biotech drug developer summit.
The platform approach has become the answer to this challenge in several countries, he said.
“A shift from bespoke development to conveyor-0belt mode is only possible when the regulatory system begins to trust the platform – accepting that data obtained for one product can be partially reused for subsequent products, of course while maintaining absolute patient safety. This is exactly what the U.S. Platform Technology Designation Program (PTD) is designed to do. In May 2024, the FDA issued detailed guidance, and by 2026 it had accumulated the first examples of both granting and – just as importantly – withdrawing platform technology status,” he stressed.
This experience is unique, the expert said, because for the first time in global practice it formalises an “overlap zone” – the space where technological maturity of a platform meets regulatory trust.
“It is at this point that maximum economic efficiency is achieved, because the cost of developing the second and subsequent products can fall by 30-50%, and time to market by 20-40%,” Lavrov said.
The expert stressed that analysing the PTD program is important for Russia, because under the national Bioeconomy project the country has set a goal of technological leadership, including the development of its own competitive biopharmaceutical products. Therefore, being part of the new global trend of platform-based pharmaceutical development will be an imperative for the next quarter-century. At the same time, implementing such an approach cannot be done in isolation.
“It requires the coordinated development of related industries: new materials (lipids for nanoparticles, polymers for delivery systems), microelectronics (sensors, organ-on-a-chip components), information technology and artificial intelligence, which can learn from standardised platform data to predict safety and efficacy profiles of new candidates,” Lavrov said.
Gene technologies can and should become platform-based, said Igor Samatoshenkov, a cardiovascular surgeon and co-founder of Angiolife.
“Without changing the vector itself – plasmid or virus – one could simply replace the cloned gene for a specific disease. This would significantly speed up market entry, bypassing repeat toxicity or immunogenicity studies,” the expert noted.
Apart from gene therapy, platform technologies could be applied in three other areas, explained Irina Alekseenko, head of the Genetic Immuno-Oncology Therapy Group at the Shemyakin-Ovchinnikov Institute of Bioorganic Chemistry.
Platform technologies can also be applied to mRNA drugs, she said.
“In Russia there is already a GMP production base at the Gamaleya Centre, strong scientific expertise there, as well as at the Institute of Bioorganic Chemistry and Sirius University. On this platform at least three product classes can potentially be developed: vaccines, cancer drugs including mRNA immunoadjuvants, and therapies for rare diseases,” she elaborated.
The second direction highlighted by the expert is the adeno-associated virus (AAV) platform.
“This is where the platform approach is especially justified: the basic safety of AAV is already well understood, while what changes from product to product is primarily the genetic insert. We already have promising developments for spinal muscular atrophy, retinal dystrophy, haemophilia and Duchenne muscular dystrophy. If such technologies are formalised as platform-based, it will significantly reduce costs for both the state and developers,” Alekseenko stressed.
The third direction, according to the expert, is cell technologies – above all CAR-T.
“Many groups are now creating CAR-T products for different targets, yet the same target can often be used for several diseases. For example, CD19 – for both oncological and autoimmune conditions. Nevertheless, developers are forced to go through almost the entire process again each time – conducting preclinical studies de novo, then repeating the clinical track – even though the vector is already known and the safety profile is largely understood. When moving from, say, oncology indications to autoimmune diseases, fundamentally new risks for the patient may not arise, but time and money are still spent again,” she noted.
However, surgeon Igor Samatoshenkov believes that platform solutions are harder to apply to cell technologies. He argued that each batch of harvested or modified stem cells can vary significantly in biological terms and in efficacy.
Vladimir Andrianov, partner relations director at R-Pharm, said he is confident that Russia today has everything it needs – technological groundwork, an understanding of global trends and strategic targets – to make the platform approach a real tool for achieving technological leadership in Russian biopharmaceuticals.
“The R-Pharm platform launched in Pushchino is an example of exactly this approach: a single technological base that allows us to create up to 200 genetic constructs per year and bring new molecules for cancer and autoimmune diseases into pilot preclinical trials. But today, for each subsequent drug on this platform, we are forced to prove its safety and efficacy to the regulator largely from scratch – even though the FDA experience shows that another way is possible,” he added.
Olga Fedorova, vice-rector for research and postgraduate training at Siberian State Medical University, agreed that introducing platform technology status in Russia is not simply a transfer of foreign experience, but an investment in the future that will give patients access to innovative drugs and strengthen the position of Russian drug developers and manufacturers in the global biotech market. At the same time, she said, the approach will require restructuring all stages of the drug development lifecycle and, to a large extent, the drug supply system – launching new activities and necessitating adaptation of regulatory approaches.