Therapeutics
more traditional small molecule pharmaceutical company that recognised the growth potential of this field. Other pharmaceutical companies are making similar strategic investments, which sug- gests that as regenerative medicine companies con- tinue to progress, there could be multiple options for strategic partnerships downstream. Despite these positive developments, the tradi- tional venture community has been slow to invest in regenerative medicine. They still view the sector as extremely risky with uncertain regulatory and reimbursement prospects and very long develop- ment timelines. Moreover, the VC community is facing its own challenges as many of them are addressing underperforming portfolios, anxious limited partners and limited prospects for raising new funds10. Therefore, the cost of funding dis- covery and early clinical development of regenera- tive medicine products is being born dispropor- tionately by small-mid size companies funded pri- marily by federal and private research grants and a small amount of private investment capital. Pharmaceutical firms are beginning to invest more in this field, but it is still early and the amount of funding available has thus far been crowded out by their near-term revenue priorities. If the industry is to sustain the progress it is making in regenerative medicine, careful consideration should be given to new funding models and tax incentives that will attract new sources of capital to this vitally impor- tant sector.
Getting the regulatory house in order Despite the growing market and impressive pipeline for regenerative medicine technologies, tools and therapies, there are still many regulato- ry roadblocks that need to be addressed to ensure that safe and effective products reach patients quickly. A key component to the industry’s broad- er commercialisation strategy is the reduction of regulatory risk.
The current regulatory barriers to commerciali- sation are recognised by both industry and regula- tors. Both groups understand the need for a pre- dictable, efficient and swift review and approval process and are working together to address these issues. These efforts require engagement and co- ordination among many groups, including the FDA and NIH; Congress; industry stakeholders; research institutions and organisations; and the patient advocacy community. The Alliance for Regenerative Medicine (the Alliance) is bringing these stakeholders together to define and achieve actionable steps toward improvements in the regu- latory process.
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The Alliance is currently working with the FDA and others to address the key regulatory issues cur- rently impacting regulatory risk. Key issues for dis- cussion are: FDA processes; development of stan- dards and consensus best practices in the regenera- tive medicine community; the need for cross- agency co-ordination; and improving the regulato- ry science base at the FDA.
FDA processes
Unclear regulatory pathways are one of key issues that affect the risk perception for regenerative medicine companies, both from a product devel- opment and investment viewpoint. Clearer rules regarding co-ordination between CBER and CDRH on product review are needed. Even after a product is assigned to one division to lead the review, questions remain as to how the divisions will co-ordinate on key issues. This creates uncer- tainty for companies as they work toward a regu- latory filing in terms of what data will be needed for endpoints, understanding of the timetable for review and other matters. This is a particular chal- lenge in the cross-disciplinary field of regenerative medicine as many products are comprised of both biologic and medical device components and may require expert review from multiple FDA divi- sions. Discussions with agency regulators earlier on in the development process could help ease some of this uncertainty by providing clarification on agency viewpoints of complex products, espe- cially those that can be considered medical devices or biologics. Additionally, regulatory approval of regenera- tive medicine products is impeded by the lack of consensus standards surrounding important sci- entific issues related to the regulatory process for regenerative medicine products including: cell characterisation; quality control; and animal models. The lack of standards makes compara- bility across studies (and products) difficult, slows the regulatory process, and contributes to uncertainty in the development process. Development of standards, especially on pre-clin- ical topics such as those listed above is critical to inform regulatory assessments and analyses of new products. The Alliance has convened an expert working group of scientists from industry, research organisations, and universities to begin work on these standardisation issues with an ini- tial focus on cell potency assay development. Workshops with participation from all these organisations as well as patient advocacy groups and FDA will also be important information sharing opportunities.
Drug Discovery World Spring 2011
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