December 22, 2014
In Canada, the Children’s Hospital of Eastern Ontario is challenging gene patents held by companies in the Federal Court of Canada. The hospital is specifically challenging the patent held on a genetic test for long QT syndrome – a rare genetic disorder with fatal consequences. Testing for the gene in people with a family history of the disorder can help doctors prevent disease-related incidents in high-risk patients. The test for long QT syndrome costs CAD 4,500 and, when incidentally discovered in a patient, cannot be revealed to the patient. The doctors can be prevented from learning anything further due to the patents held by a US company – Transgenomic North America. This violation of patient rights and incorrect usage of patenting has been taken to the Federal Court on medical and ethical grounds, citing barriers that gene patents present in providing tests for genetic diseases.
December 17, 2014
The Linked2Safety project, funded under the FP7 scheme of the European Commission, was launched to advance clinical practice and accelerate medical research by providing pharmaceutical companies, healthcare professionals and patients with an “innovative semantic interoperability framework, facilitating efficient and homogenised access to distributed Electronic Health Records (EHRs)”.
Electronic Health Records (EHRs) contain an increasing wealth of medical information and have the potential to signiﬁcantly advance medical research, as well as improve health policies. However, the European healthcare information space is fragmented due to the lack of legal and technical standards, cost effective platforms and sustainable business models. Linked2Safety is a platform for analysing EHRs from multiple institutions, while strictly adhering to the legal and ethical requirements deﬁned by each data provider at EU level.
Benefits for this 36-month Linked2Safety project include facilitating data analysis in EHRs, including genetic, environmental and medical histories of subjects exhibiting adverse events during clinical trials. This will help provide genotype-phenotype associations and a platform to identify and select patients for clinical trials by linking EHR repositories.
December 15, 2014
An article published in the Annals of Oncology discusses clinical trial endpoints acceptable to the Japanese Pharmaceuticals and Medical Devices Agency (PMDA) to approve oncology drugs. The authors highlight that acceptance of surrogate endpoints is essential to accelerate drug approval. However, following guideline revisions in Japan, the number of accepted surrogate endpoints has decreased. While Japan’s regulatory system proposes many expedited programs, including orphan drug designation, priority review, public knowledge-based application and a special committee on unapproved drugs, the country is lagging behind the US and the EU in terms of drug approvals. The authors believe that an accelerated approval program and surrogate endpoint discussions are necessary to bring oncology drugs to patients faster..
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.