Standard regulatory factors also need to be taken into account when formulating the overall Asia Pacific regulatory strategy. Some countries have relatively low governmental fees (e.g. Vietnam) whilst others are expensive (e.g. Taiwan and China) and this needs to be put into context of
- The number of patients with the disease,
- Their likelihood of receiving reimbursement, and/or
- Their ability to self fund treatment.
It’s important therefore to have a good handle on the overall commercial viability before setting out on an exhaustive filing process.
Standard regulatory complications include the multitude of file formats required as there remains considerable variation across the region e.g. filings can be ICH, CTD, ACTD, or Chinese FDA depending on the country. There is also considerable variation in stability data requirements where zone 2 stability data may be acceptable for registration in New Zealand, Hong Kong and China for instance. In contrast zone 3 stability data will be necessary in some other countries and as of the adoption of the ASEAN guidelines all SE Asia countries are moving (at different paces) to the considerably more challenging zone 4b requirements of 30C:75% RH.
In the short term it is possible to file in some countries without all the necessary data being available - as long as specific assurances can be given. In other territories this is becoming impossible. It’s important for new entrants to be guided by partners that can advise across the whole region – taking a piece meal approach to registration can lead not only to missed patient and commercial opportunities but to soured relationships between individual distributors. Innovative solutions are possible where regulatory strategy is matched to secure supply chains. This gives reassurance to the orphan originator that their orphan arrives in the physician’s hands in perfect condition.
There are numerous options available for any Orphan Drug company – if you would like to learn more and make a conscious decision which benefits all parties please contact AFT Orphan via giles@aftpharm.com.
