We've seen an unprecedented amount of Late Stage-related news, guidance and updates published in the past two months, suggesting that 2016 is going to be a very busy year for us all.

I've provided you with an overview of the most recent news below and truncated links to the rest...there really was just too much for one email blast.

Enjoy and best wishes,


Stuart McCully, PhD
Founder & Owner of CHCUK Ltd
CHCUK: 2015 Country-Specific NIS Reports
 2015 version of 38 country-specific NIS reports now available on-line for just £50 each - This is a tine-limited offer!

Country-specific NIS reports are currently available for:

Argentina, Australia, Austria, Belarus, Belgium, Bulgaria, Canada, Czech Republic, Denmark, Finland, France, Germany, Greece, Hungary, India, Ireland, Israel, Italy, Latvia, Lebanon, Lithuania, Malaysia, Netherlands, Norway, Peru, Poland, Portugal, Romania, Russia, Saudi Arabia, South Korea, Spain, Sweden, Switzerland, Turkey, UK, USA and Vietnam

We'll be adding NIS reports for a further 20 countries in the very near future
  Browse the CHCUK NIS Reports »
China: Tougher Criminal Provisions Governing Data Privacy
Article 253 of the Criminal Law has been amended, as of 1 November 2015, to permit criminal prosecutions relating to a broader range of acts involving the unlawful disclosure or the unlawful procurement of personal information.

Article 253 of the Criminal Law as currently in effect imposes criminal liability on employees of government institutions and companies in the financial, telecoms, transportation, educational and medical sectors who sell or otherwise unlawfully provide to third parties any citizen's personal information that has been obtained in the  course of employment or provision of services where the associated circumstances are "serious". It also imposes criminal liability on any person who obtains that information by means of theft or other unlawful means-again, if the associated circumstances are "serious". Punishment for breach of Article 253 as currently in effect includes prison terms or criminal detention of up to three years and/or a fine.

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USA: Drug and Device Companies May Soon Face Less Burdensome FDA Approval Process
On July 10, 2015 Congress passed the 21st Century Cures Act.  The bill seeks to make the U.S. Food and Drug Administration (FDA) drug and device approval process less onerous to expedite patient access to new treatments and cures.

During the review of a new drug, the bill requires the FDA to consider patient experience data in the drug's benefits and risks assessment.

Medical device companies could also benefit from this bill. The bill clarifies that for FDA approval of medical devices, evidence such as registry data, studies published in peer review journals, and data collected in countries other than the United States can be considered under certain circumstances.

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USA: Delivering Health Care Services to Consumers Through ehealth Devices
Smart medical devices that can deliver health care services are increasingly consumer friendly. Using the latest technologies and sophisticated algorithms, some devices are capable of collecting data directly from consumers then providing them with diagnoses and prescriptions for treatment, equipment and products. Companies producing these devices are eager to market and deliver their products and services directly to defined demographic consumer groups for certain low-risk health conditions. The marketing campaigns and business models they use for national expansion, including how licensed practitioners are involved, depend upon the application of state professional practice laws.

Professional practice laws govern who can collect patient data, administer tests, make diagnoses, determine treatment plans and issue prescriptions, among other things. These laws vary by state and are based on product-by-product and professional license-by-license categories. The laws may dictate the relationship between the device and licensed practitioners and ultimately determine a company's organizational structure. Further, professional practice laws may define the content of advertising for the medical devices and the health conditions they address. Typically, violations of these professional practice laws are classified as misdemeanors.

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EU: EMA Guidance - Questions and Answers on Post-Authorisation Efficacy Studies
The EMA has produced lists questions (and answers) that marketing-authorisation holders (MAHs) may have on post-authorisation efficacy studies (PAESs). It provides an overview of the European Medicines Agency's (EMA) position on issues that are typically addressed in discussions or meetings with MAHs in the post-authorisation phase.
  1. What is a PAES imposed in accordance with the Commission Delegated Regulation? 
  2. How and where the PAES imposed in accordance with the Commission Delegated Regulation will be reflected in the marketing authorisation? 
  3. Following which procedure will my imposed PAES protocol be assessed? 
  4. When should I submit my imposed PAES protocol? 
  5. In which timeframe will my imposed PAES protocol be evaluated (timetable)? 
  6. What are the possible outcomes of the evaluation of an imposed PAES protocol? 
  7. Do I have to submit interim results? 
  8. Do I have to submit the final results of my imposed PAES? 
  9. Do I have to pay fees for the protocol and final study results submission? 
  10. How is a PAES enforced? 
  11. Will there be any publication on the outcome of my PAES protocol and final study results assessment? 
  12. Who should I contact if I have a question when preparing my application?
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EU: Draft Scientific Guidance on Post-Authorisation Efficacy Studies - Open for Comments
The EMA has developed, in cooperation with national competent authorities and other interested parties, a draft scientific guideline on PAES which outlines how companies should design these studies in order to support regulatory decision making in the European Union.

This guidance is intended to provide scientific guidance for marketing-authorisation holders and national competent authorities on post-authorisation efficacy studies with regard to methodological considerations and the potential role of particular study designs. This guidance does not include procedural aspects, which are dealt with in the human post-marketing authorisation regulatory and procedural guidance.

First published06/11/2015
Last updated06/11/2015
Consultation start date06/11/2015
Consultation end date31/01/2016

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USA: HIPAA Lessons from the Warner Chilcott Settlement
Last week, the US Attorney's Office in Boston announced that drug company Warner Chilcott agreed to plead guilty to health care fraud and pay $125 million to resolve criminal and civil liability arising out of allegations involving the promotion of the company's drugs. Continuing its focus on individuals, the former President of the Company has been charged with conspiring to pay kickbacks to physicians. The government interest in prosecuting illegal drug promotion activities and illegal payments to physicians has been a longstanding priority.  However, in a new twist that should be of great interest to the health care community, the government has brought criminal charges under the Health Insurance Portability and Accountability Act (HIPAA) against company employees
as well as the physician practice owner for the alleged unlawful access and disclosure of  patient medical records. These HIPAA violations could result in prison sentences, significant fines and exclusion from the Medicare program.

According to the Information filed last week, district managers allegedly encouraged their sales representatives to flag patient medical records with brochures about the company's drug.  The sales reps also were accused of filling out the required prior authorization forms for the physician office and, in some instances, taking patient records home in order to complete those prior authorizations.  In order to conduct such activities, the sales representatives would have had to access the patients' medical records.

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UK: A Bitter Pill - Hard Lessons Learnt by Online Pharmacy Fined for Selling Customer Data
The Information Commissioner has issued a monetary penalty notice (MPN) of £130,000 to Pharmacy2U, the UK's largest NHS approved online pharmacy, after it sold the details of 21,500 customers to third-parties through an online marketing company.

Initially uncovered by a Daily Mail investigation, the Commissioner found that Pharmacy2U had advertised more than 100,000 customers' details - at £130 per 1000 customers - for rental through a marketing company, Alchemy Direct Media (UK) Ltd.  In late 2014, Alchemy supplied a total of 21,500 Pharmacy2U customer names and addresses for use by a health supplement company, an Australian lottery company and a charity.

The first data protection principle, as outlined in paragraph 1, Schedule 1 of the DPA, requires  personal data to be processed fairly and lawfully and in accordance with at least one of the conditions set out in Schedule 2 of the DPA, one of which is the provision of informed consent by the customer. The Commissioner found that Pharmacy2u had breached this principle, and given the severity and negligent nature of the breach, as well as the distress caused, issued a substantial fine. The decision notice can be read in full here.

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EU: OECD Report Demonstrates the Need for Outcomes-Based Approach to Healthcare in Europe
EFPIA acknowledges the publication of the OECD report, "Health at a Glance 2015", noting its contribution to the debate on the sustainability of health systems. Its content lends weight to the argument that an outcomes-based approach is part of the solution to the many challenges faced by European healthcare systems. A view the pharmaceutical industry supports.

The report acknowledges the variance in patient outcomes between countries at a similar development stage. This supports the contention that healthcare expenditure in total is not the issue, but rather how and where resources are allocated.  Europe needs systems that focus on improving patients' health in a holistic and evidence-based way. Systems that allocate resources towards those interventions that deliver the best possible outcomes and away from those that don't, these systems improve quality which is often less expensive in the long-term and thus more sustainable than the current transaction-oriented approach to healthcare.

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US: Reform of Clinical Research Regulations, Finally
Proposed Changes to the Common Rule
The proposed changes, though imperfect, are a significant step forward. Six aspects deserve special attention; some would enhance protections, while others would improve efficiency - and in turn enhance protections by focusing resources and attention on studies posing the most serious risks and ethical challenges.
  1. The proposal applies protections to all clinical trials conducted at U.S. institutions receiving federal funding for human-subjects research.
    1. Currently, protections apply only to research funded by departments and agencies that have adopted the Common Rule.
  2. The regulations aim to enhance and streamline the informed-consent process, in part by shortening and focusing informed-consent documents on "essential information that a reasonable person would want to know," with additional details provided in an appendix.
  3. Secondary research on biospecimens and identifiable private information originally collected for research, clinical, or other purposes would require informed consent.
    1. In keeping with extensive empirical research, one-time, general, open-ended consent could be obtained at the time of collection. This approach allows people to decide whether they want their specimens and data used for research but obviates the need for subsequent consent for each project.
  4. The proposal defines four types of regulatory oversight - delineating the types of research that are excluded (a new category) or exempt, types qualifying for expedited review by a single IRB member because they pose minimal risk, and types requiring full-IRB review.
  5. Continuing-review requirements would change.
    1. Excluded and exempt research would still require no continuing review.
    2. The default for expedited research would be no continuing review - a change - but a study's reviewer could make a case for continuing review.
    3. For greater-than-minimal-risk research, a change would mean continuing review could cease once recruitment and experimental interventions were completed and only clinical monitoring of participants and analysis and reporting of research results remained.
  6. Instead of protocols for multicenter projects being reviewed by each institution's IRB, all participating U.S. sites would rely on a single IRB review.
    1. The only exceptions would be for multicenter projects that require multiple IRB reviews by law (e.g., for FDA-regulated device research), research at non-U.S. sites, and cases in which a funding agency chooses to require multiple reviews.

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AU: Draft Guidance - Collecting Health Information for Research Without Consent
This business resource explains the requirements under the Privacy Act 1988 (Cth) (Privacy Act) for private sector health service providers or other private sector organisations who are seeking to collect, use or disclose health information without consent for research or statistical purposes relevant to public health or public safety. This resource is part of a series that outlines what private sector health service providers need to know about handling their patients' health information. Some of the key health privacy terms used are explained in Business resource - Key health privacy concepts, while other terms are explained in the  Australian Privacy Principles Guidelines.

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EU: EMA Initiative for Patient Registries - Strategy and Pilot Phase
Problem Statement
Regulators may require marketing authorisation applicants/holders (MAAs/MAHs) to establish a registry in order to measure the safety or efficacy of individual products in routine clinical practice. Various challenges with regard to registries currently exist, including a lack of sustainability and a lack of harmonised protocols and data structures. In addition, existing disease registries or other data sources established at national or international levels by physicians' associations or national agencies are not fully utilised, a situation that may lead to duplication of efforts and inefficiencies. Therefore, the need has been identified to explore opportunities to improve this situation by making better use of existing registries and facilitating the establishment and utility of new registries as a source of high- quality post-authorisation data for regulatory decision-making.

Objective of the Initiative
The main objective of the initiative for patient registries is to facilitate the use of existing patient registries and facilitating the establishment and utility of new registries if none are available or adequate, in order to collect and analyse high quality data informing regulatory decisions.
The initiative for patient registries includes two components: a strategy on registries and a pilot phase to test whether this strategy better supports MAAs/MAHs to meet regulators' (and potentially other stakeholders') needs for data and information.

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US: FDA is Soliciting Input on the Use of Technology and Innovation in Clinical Research
FDA is soliciting public input from a broad group of stakeholders regarding technologies and innovative methods for using technology to more efficiently conduct clinical research. FDA is interested in identifying new opportunities to study medical products, as well as receiving comments on barriers, challenges, and relevant considerations that may affect a medical product clinical investigation that uses these technologies and methods.
In addition to the general information requests in section II of this document, FDA is interested in obtaining information and public comment on the following specific issues:

1. What technologies, communication infrastructure, or innovative methods are being used to conduct clinical investigations?
2. What are ways FDA could encourage adoption of these technologies and innovative methods in the conduct of clinical investigations?
3. Identify any clinical, cultural, business, regulatory, or other barriers perceived by stakeholders that serve as a disincentive to the use of technology to facilitate the conduct of clinical investigations.
4. FDA is interested in obtaining information on potential trial participant acceptance, privacy, and human subject protection issues that may occur as a result of the use of technologies and innovative methods for the conduct of clinical investigations. In particular, FDA isinterested in assessing potential trial participants' interest, tolerance, concerns, and willingness to participate in clinical investigations that involve nontraditional settings or utilize new technologies. FDA is also interested in identifying the factors that affect trial participant awareness, acceptance, enrollment, and retention for these investigations.

Submit electronic or written comments by December 28, 2015

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Still More News...!!
Click on the links below for more news items:
Kylen, Craidarroch Drive
Contin, IV14 9EL, UK
+44 1997 423311  /  info@chcuk.co.uk  / www.chcuk.co.uk
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