December 12, 2014
A review published in Clinical Pharmacology & Therapeutics outlines regulatory activities geared towards accelerated access and reimbursement benefits for patients in need across four regulatory agencies. The authors drew up two tables providing comprehensive information on the existing, emerging and new regulatory access and reimbursement policies implemented in the United States, Europe, Singapore and Canada to help pharmaceutical companies bring innovative medicines to the market.
The paper describes how sponsors can obtain accelerated access, conditions under which it is provided and how the assessment takes place. While orphan drug and pediatric drug initiatives were not designed specifically to accelerate patient access to drugs, these initiatives are frequently used to bring innovative drugs to the market. In the US, programs such as Accelerated Access and Priority Review help sponsors benefit from shortened clinical development and shortened review, respectively. The FDA’s Fast Track and Breakthrough Therapy regulatory programs also focus on bringing innovative medicines to patients in need. The EU’s Conditional Marketing Authorisation and Approval Under Exceptional Circumstances programs allow sponsors to benefit from shortened development time and Accelerated Assessment accelerates the review process. The EU and the US also offer parallel scientific advice between the EMA and the FDA, whereby they align advice and reduce developmental complexity. In Canada, the orphan drug framework is an emerging regulatory process being developed for orphan drug regulation. In Singapore, the process of abridged evaluation and verification has shortened processing time.
Greater diversity is observed in medicines’ reimbursement approaches across the four jurisdictions. In the US, Coverage with Evidence Development, used by the Center for Medicare and Medicaid Services (CMS), provides early reimbursement of emerging technologies. A new Center for Devices and Radiological Health program, in coordination with the CMS, helps shorten overall development time by collecting data needed by the CMS early during development. The emerging Managed-Access program in the US can be used by private payers to control post-market access to new medicines. The EU has recently developed a similar managed entry program. However, in the EU, “payers rely [increasingly] on Health Technology Assessment (HTA) bodies to provide information and analysis of the clinical, patient, organisational and economic implications of new medicines, recognising that regulators are generally prohibited by law from taking economic considerations into their decision making”. Canada is working on programs with the Institut national d’excellence en santé et en services sociaux to shorten reimbursement time. In Singapore, apart from the Standard Drug List of drugs to be subsidized at public health-care institutions and a group purchasing office that procures health products in bulk, the pricing of medicines is largely left to open market forces.