The Future of Orphan Therapy: Regulation, Technology, Accessibility

At the second plenary session of the II CIS Orphan Forum, experts discussed issues of availability, localization and regulation of orphan drugs. Representatives of the Russian Ministry of Health and the Ministry of Industry and Trade presented a comprehensive analysis of the current situation and steps needed for sustainable development in this area.

Elena Anatolyevna Maksimkina, Director of the Federal Center for Planning and Organization of Drug Provision for Citizens of the Russian Ministry of Health:

Despite the fact that we often hear from the pharmaceutical industry about the low investment attractiveness of the orphan drug segment, in fact, the trends in registering drugs with orphan status in international jurisdictions - taking into account the choice of targeting targets, even in relation to diseases that do not have orphan status (for example, oncological diseases) - indicate the opposite.

Elena Anatolyevna also added that

The price per unit of most orphan drugs is tens of times higher than the cost of non-orphan drugs. In these conditions, the main challenge of drug provision for patients with orphan status for healthcare systems is ensuring the financial availability of pathogenetic therapy, taking into account the comparability of therapy costs with the achievement of the clinical effect of preserving physical and mental functions.

Dmitry Sergeevich Galkin, Director of the Department for the Development of the Pharmaceutical and Medical Industry of the Ministry of Industry and Trade of Russia:

The creation of the Circle of Good fund was a breakthrough not only for providing patients, but also for the entire pharmaceutical industry. This gave manufacturers a clear signal: the state is ready to invest in the treatment of orphan diseases.

Dmitry Sergeevich cited statistics:

• 87 registered orphan drugs in the Russian Federation;
• more than 30 are already being produced in the country;
• about 20 are in active development;
• 13 out of 19 current clinical trials in the orphan direction are conducted by Russian companies.

It is important that the development vector covers not only import substitution, but also platform innovations - adeno-associated viruses, mRNA, CAR-T. We see an increase in technological capabilities, and are ready to transfer solutions to the CIS and EAEU countries.

Anna Viktorovna Frolova, Deputy Director of the Department for Regulation of Circulation of Medicines and Medical Devices of the Russian Ministry of Health:

The centralized procurement of orphan drugs covers more than 290 thousand patients, of which 33 thousand have confirmed orphan status. Moreover, half of them are adults. It is important that 20 of the 26 names in this program are already produced in Russia.

Anna Viktorovna also noted that:

• 45.8 billion rubles were spent on orphan patients in 2024
• 25 billion of which were spent on adults, 20 billion on children
• about 60% of the total amount was spent on hemophilia treatment

The geography of speeches in the second plenary session covered the CIS, BRICS and the Middle East. Representatives of India, Oman, the UAE, Kazakhstan, Kyrgyzstan, Uzbekistan and Belarus shared their experience and priorities in the field of orphan therapy.

The discussion included key points about the development of gene therapy and CAR-T, the role of health technology assessment (HTA), the importance of registries and international cooperation. The tools for sustainable financing, localization of production and involvement of patient communities were discussed.

The first day of the Forum became an important platform for dialogue between developers, regulators, clinicians and patients, and the exchange of practices and strategies outlined the vectors for the development of orphan care.

Tomorrow is the second day of the II CIS Orphan Forum! A rich program of thematic sessions lies ahead - don't miss it!