ell, maybe not in the immediate future, but the end may indeed come.

FDA’s press release announcing this approval states: “A one-time intravenous administration of Zolgensma results in expression of the SMN protein in a child’s motor neurons, which improves muscle movement and function, and survival of a child with SMA.”

A single dose apparently delivers a cure. This is transformative.

t FDA, there is a focus on evaluating and considering valuable novel approaches and tools that can support our regulatory decision-making. The dramatic growth and incorporation of digital technologies into daily life has afforded new opportunities to use these tools to understand patients’ functioning and how it is affected by different diseases and treatments.

Former FDA Commissioner Scott Gottlieb has made statements regarding advances in the field of digital health innovation, including launching the Agency’s Digital Health Innovation Action Plan. Likewise, the US President’s budget includes a proposal to create a Center of Excellence for Digital Health. In the current landscape, the Agency is receptive to innovative, technology-derived, study endpoints in various therapeutic areas.

atient engagement in medical product development has become a priority across patient groups, regulators, and industry. Presentations and communications proliferate about what patient preferences are and why it is important to understand them. But patient preference research needs practical steps to realize its goal; namely, the systematic inclusion of patient preference information and data into medical product decision-making. The Innovative Medicines Initiative (IMI) project Patient Preferences in Benefit-Risk Assessments during the Drug Life Cycle (PREFER) is helping in several major ways.

Background and Scope

egulatory approval (marketing authorization) is necessary for drug launch but is not sufficient to guarantee patient access to a licensed medicine. There is a time lag between a marketing authorization and patient access. The question of how long that lag is matters a great deal to patients. EFPIA sought to answer this question with empirical data.

• The rate of availability, measured by the number of medicines available to patients in European countries.
  • For most countries, this is the point at which the product gains access to the reimbursement list (see exceptions/explanations in callout box below).
  • In some countries, products are available with special reimbursement conditions (see exceptions/explanations in callout box below).
  • The rate of availability in a country does not necessarily indicate medicine uptake, as some medicines may be available in a market with no uptake (no sales or no volume).
• The average time between marketing authorization and patient access, measured by the number of days elapsed from the date of EU marketing authorization (or effective marketing authorization in non-EEA (European Economic Area countries) to the day of completion of post-marketing authorization administrative processes. Waiting times reflected in the Patients W.A.I.T. Indicator include any delay, whether attributable to companies or to competent authorities.

The analysis is based on information gathered from EFPIA member associations, who either refer to information available from official sources or gather this information directly from member companies.

s Canada heads into a federal election in October 2019, the first half of 2019 has been a busy time for pharmaceutical policy-related announcements. Issues that have been topics of discussion for decades have finally risen to the top of the political agenda, impacting areas such as rare diseases, national pharmacare, and drug pricing, to name a few. These developments are particularly relevant to reimbursement policies.

n March 19, 2019, the Union Ministry for Health and Family Welfare published the new Drugs and Clinical Trials Rules, 2019 (New Rules) which is expected to build a robust regulatory and clinical research ecosystem in the country and promote the conduct of ethical and quality clinical trials which, in turn, will benefit patients in India by providing early access to medicines for unmet medical needs.

These New Rules are applicable to all stakeholders like pharmaceutical companies, study sponsors, investigators, academic research institutions, and ethics committees who are involved in conduct of clinical trials, bioequivalence and bioavailability studies, and academic health research.

It is interesting to note that the word “oversight” appears several times in the New Rules. Although the number of mentions may not be that significant, it is the spirit of the oversight of research activities by stakeholders that gets a lot of attention in the New Rules.

What is a Specialty Drug?

pecialty drugs are used to treat complex and chronic conditions. Though there is no standard definition, there are two main considerations for determining whether a product is a specialty medication: complexity and cost.

• Complexity may be specific to the handling or administration of the product itself, such as an injectable for the management of psoriasis; it may also be due to the complexities of managing the disease state, such as an oral oncolytic with specific monitoring parameters.
• High cost of most specialty products is in part due to the complexities of manufacturing the product. For instance, some specialty products are biologics which are manufactured or synthesized from a biological source. These specialty products may require cold chain management and subcutaneous administration. As a result, patients who are utilizing specialty medications also require a higher level of clinical management and specific drug and disease support services.