DIA's Content Currents provides you with new and important global regulatory developments and their impact on pharmaceutical, biotechnology, and medical product development.
EDITION PUBLISHED: February 21, 2013
SECTION 1 FDA GUIDANCES & MAPPS
CDER 2014 New & Revised Draft Guidance Agenda
Guidance Agenda: Guidance Documents CBER is Planning to Publish During Calendar Year 2014 (PDF - 31KB)
On February 18, 2014, FDA announced a final order entitled ‘‘Medical Devices; Neurological Devices; Classification of the Neuropsychiatric Interpretive Electroencephalograph Assessment Aid.’’
FDA is classifying the neuropsychiatric interpretive electroencephalograph (EEG) assessment aid into class II (special controls) in order to provide a reasonable assurance of safety and effectiveness of the device. This order is effective March 20, 2014. Following the effective date of this final classification administrative order, any firm submitting a 510(k) premarket notification for a neuropsychiatric interpretive EEG assessment aid will need to comply with the special controls named in the final administrative order. The risks to health associated with this type of device identified by FDA and the measures required to mitigate these risks are listed in the rule. [Federal Register
On February 18, 2014, FDA announced the availability of the guidance entitled “Requests for Feedback on Medical Device Submissions: The Pre-Submission Program and Meetings With Food and Drug Administration Staff.”
The purpose of this guidance is to provide an overview of the mechanisms available to application sponsors through which to obtain FDA feedback regarding potential or planned medical device submissions reviewed in the Center for Devices and Radiological Health (CDRH) and the Center for Biologics Evaluation and Research (CBER), including the Pre-Submission program (formerly the pre-Investigational Device Exemption (pre-IDE) program). In addition, the guidance provides recommendations regarding information that should be included in a Pre-Submission Package. This guidance also describes the procedures that CDRH and CBER intend to follow when manufacturers, their representatives, or application sponsors request a meeting with review staff. [Federal Register
On February 18, 2014, FDA announced the availability of the report entitled “Report on the Performance of Drug and Biologics Firms in Conducting Postmarketing Requirements and Commitments.”
Under the Food and Drug Administration Modernization Act of 1997 (FDAMA), the Food and Drug Administration (FDA) is required to report annually in the Federal Register on the status of postmarketing requirements and commitments required of, or agreed upon by, holders of approved drug and biological products. This notice is the Agency’s report on the status of the studies and clinical trials that applicants have agreed to, or are required to, conduct. [Federal Register
On February 18, 2014, FDA announced the opportunity to comment on a proposed collection of information on “Disclosure Regarding Additional Risks in Direct-to-Consumer (DTC) Prescription Drug Television (TV) Advertisements (Ads).”
FDA is required to provide this 60-day comment period prior to submitting the collection of information to OMB for approval. The public is invited to comment on the proposed study, which will investigate the impact of limiting the risks presented in DTC prescription drug television ads to those that are serious and actionable, and including a disclosure to alert consumers that there are other product risks not disclosed in the ad. Currently, there are conflicting viewpoints on the optimal length and content of the disclosure statements for best consumer comprehension of the message. [Federal Register
On February 19, 2014, FDA published corrections to a final rule that appeared in the Federal Register of March 9, 1979, establishing “Administrative Detention”
procedures for devices intended for human use believed to be adulterated or misbranded. The document was published with a citation in the first column on page 13240 that subsequently was changed by the Nutrition Labeling and Education Act Amendments of 1993. In addition, the document was published with one typographical error in the first column on page 13241. This document corrects these errors. [Federal Register
On February 19, 2014, FDA announced that it is amending its regulation “Medical Devices’ Reports of Corrections and Removals,”
regarding reports of corrections to and removals of medical devices. The amendments address a minor change as a result of the enactment of the Food and Drug Administration Amendments Act of 2007 (FDAAA). This action is technical in nature and is intended to provide accuracy to the Agency’s regulation. [Federal Register
On February 20, 2014, FDA established a docket to receive information and comments on “Standards for the Interoperable Exchange of Information for Tracing of Human, Finished, Prescription Drugs, in Paper or Electronic Format”
to comply with new requirements in the Drug Supply Chain Security Act (DSCSA). We are seeking information from drug manufacturers, repackagers, wholesale distributors, dispensers (primarily pharmacies) and other drug supply chain stakeholders and interested parties, including standards organizations, State and Federal Agencies, and solution providers. stakeholders and other interested parties are requested to comment about the interoperable exchange of transaction information, transaction history, and transaction statements, in paper or electronic format, for each transfer of product in which a change of ownership occurs. Comments are due by April 21, 2014. [Federal Register
On February 21, FDA announced the availability of a guidance for industry entitled ‘‘E2B(R3) Electronic Transmission of Individual Case Safety Reports (ICSRs): Implementation Guide—Data Elements and Message Specification’’
(the E2B(R3) implementation guidance) and an appendix to the guidance entitled ‘‘ICSRs: Appendix to the Implementation Guide—Backwards and Forwards Compatibility’’
(the BFC appendix). The E2B(R3) implementation guidance is intended to revise the standards for submission of ICSRs and improve the inherent quality of the data, enabling improved handling and analysis of ICSR reports. The BFC appendix describes the relationship between data elements from the 2001 ICH E2B guidance and the E2B(R3) implementation guidance. [Federal Register
SECTION 2 FDA NOTES & RELATED NEWS
FDA and EMA strengthen collaboration in pharmacovigilance area
The FDA and the European Medicines Agency (EMA) have set-up a new 'cluster' on pharmacovigilance (medicine safety) topics. Clusters are regular collaborative meetings between the EMA and regulators outside of the European Union, which focus on specific topic areas that have been identified as requiring an intensified exchange of information and collaboration. Building on the experience of previous regular videoconferences between the FDA and the EMA in this area and on the recent creation of the EMA’s Pharmacovigilance Risk Assessment Committee, this cluster will provide a forum for a more systematic and focused exchange of information on the safety of medicines.
As part of the new cluster, discussions on shared pharmacovigilance issues will now take place between the agencies on a monthly basis by teleconference. This increased degree of interaction will allow the agencies to work swiftly in the area of the safety of medicines and to coordinate communication activities. The creation of this cluster is the latest step in the FDA’s and the EMA’s broader approach to expand and reinforce international collaboration.
Canadian and Japanese regulatory authorities will participate in the meetings of the cluster on pharmacovigilance as observers. The information exchange is covered by confidentiality arrangements between the FDA and the other participants. More at link above. (FDA.gov)
FDA is Committed to Determining Sex Differences in How Drugs Work
Following her appearance on 60 minutes, Dr. Sandra Kweder, Deputy Director of the Office of New Drugs at CDER, wrote in a February 14 FDA Voice Blog: “There’s a lot happening these days with regard to the personalization of medicine and how drugs work differently in people, particularly in men versus women. FDA has a long history in understanding and analyzing these effects.
“We’ve issued guidance to the pharmaceutical industry explaining in detail our expectations about analyzing clinical data for sex-related differences as well as differences according to other demographic groupings.” She went on to discuss FDA’s updating of the dosing recommendations for women for a currently marketed sleep medication based on observed variations in women’s response. She concluded: “Our staff, including those in our longstanding Office of Women’s Health, are dedicated to protecting and advancing women’s health through policy, science, and outreach. We’ll continue to advocate for the inclusion of women in clinical trials and for analyses of how their bodies process medications. Our recent zolpidem decision is an example of how science evolves – and shows the importance of using new information to review previous decisions when needed.” More at title link above. Link to list of guidances: Understanding Sex Differences (FDA.gov)
Link to 60 minutes segment on sex differences: 60 Minutes: Sex Matters
Regulatory Science Supports FDA's Regulatory Mission
Carolyn A. Wilson, Ph.D., Associate Director for Research at FDA’s Center for Biologics Evaluation and Research (CBER) wrote in her recent blog in the FDA Voice: FDA scientists, including those in CBER, perform research, and regulatory science helps to turn innovative medical research done at FDA and other places into life-saving or life-enhancing biological products.
Most of the discoveries made at CBER support the development of new or improved vaccines, blood and blood products, and tissue, gene and cell therapies. This research also helps CBER make very informed decisions about new products and policies. That’s because many of the same CBER scientists whose research puts them at the cutting edge of science also review potential new products, inspect commercial facilities that make products, and help develop new policies and guidance documents for industry. In the past year, discoveries that CBER scientists have published in research journals have contributed significantly to public health by addressing issues that affect the safety and effectiveness of vaccines, gene therapy, and a treatment for a serious blood disorder. More at link above. (US FDA)
FDA Policies and Procedures for Proposed Trial Design Aimed at Multiple Chronic Conditions
In a December 2013 staff memo , Dr. Robert Temple, Deputy Director for Clinical Science at the FDA's CDER, laid out a new policy in the Manual of Policies and Procedures (MAPP) that FDA staff should follow in reviewing proposed trial designs. This is part of the Department of HHS Initiative on Multiple Chronic Conditions. In his memo, Dr. Temple stressed FDA's interest in encouraging a broad population sample in the development of new drugs.
He writes: "This is reflected in the required (by regulation) analyses of safety and effectiveness by demographic and other relevant subgroups in marketing applications and the recognized concern with possible interactions between treatments and coexisting illnesses and concomitant treatments. FDA and ICH guidance on inclusion of both genders and the elderly also reflects this concern and the documents urge broad patient inclusion. During the interactions that take place between FDA and the sponsor during drug development, FDA should actively encourage the conduct of at least one trial in a broad population that closely resembles the people who will use the drug if it is approved and should discourage unnecessary exclusions." More at link above. (Policy and Medicine)
Drug Quality Concerns Spur New U.S. FDA Oversight Effort
Drug quality concerns, such as those that have banned U.S. sales of generic medicines from several Indian manufacturing plants, have spurred regulators to create a new unit to sharpen their oversight. FDA is establishing an Office of Pharmaceutical Quality to improve the agency’s scrutiny of brand-name, generic and over-the-counter drugs, Janet Woodcock, director of the Food and Drug Administration’s Center for Drug Evaluation and Research, said today.
The FDA is talking with the industry to develop data that may signal which manufacturing plants are straying from standards and need inspection, she said. The agency now collects such information only during inspections. The thrust of the effort would be to head off potential concerns before the agency wields penalties such as banning products from troubled factories, Woodcock said.
Lawmakers in Congress are scheduled to hear from doctors, researchers and patient advocates Feb. 26 in a briefing on whether substandard generic drugs are reaching the U.S. medical system from overseas. More at link above. (Bloomberg)
FDA Strengthens Regulatory Presence in India
FDA has disclosed it will make efforts to enhance its regulatory role in India, as the agency looks to stem rising fears about quality control problems in plants manufacturing medicines. In conversations with reporters in India, FDA Commissioner Margaret Hamburg disclosed plans to raise the number of FDA investigators in that country to 19 from 12. “We need the same level of oversight whether it is within our borders or outside,” she said. (Wall Street Journal)
FDA to Shift 1000 Workers to White Flint
The FDA plans to temporarily move about 1,000 workers into Three White Flint from various other locations in Montgomery County, FDA spokeswoman Heidi Rebello said. The FDA will move into four floors in the building this summer and plans to take another four floors at 11601 Landsdowne St. in North Bethesda in the spring of 2015.
The moves are an interim step for the FDA while work on its consolidated White Oak campus in Silver Spring is completed. Rebello said the exact details, including which buildings those workers will move from, are still being worked out in collaboration with the General Services Administration. More at link above. (Washington Business Journal)
SECTION 3 AGENCY AND ADVISORY COMMITTEE MEETINGS
Patient-Focused Drug Development: Disease Area Meetings Planned for FY2013-2015
Postponed: Public Advisory Committee Meeting. Risk Communication Advisory Committee. February 3-4, 2014.
On January 23, FDA announced the postponement of this meeting due to unavoidable operational changes. The committee was scheduled to discuss methods for identifying the impact and increasing the reach of communications on topics of interest to consumers. The discussion was to address how FDA can evaluate whether its “Consumer Updates'' are reaching the targeted population, and whether they are increasing awareness and understanding of the key risk messages. The discussion was also to assess whether the communications are having the intended impact on knowledge, behaviors, or outcomes. (Federal Register
Public Workshop. IOM Workshop: “Characterizing and Communicating Uncertainty in the Assessment of Benefits and Risks in Drug Regulatory Decision-Making.” February 12-13, 2014.
The purpose of the workshop is twofold: To explore potential approaches to addressing and communicating uncertainty and to identify key considerations on developing, evaluating, and incorporating potential approaches for addressing uncertainty into the assessment of benefits and risks in the human drug review process. The format of the meeting consists of a series of presentations on topics related to uncertainty in the assessment of benefits and risks, followed by a discussion on those topics with invited panelists and audience members. This workshop satisfies an FDA commitment that is part of the fifth authorization of the Prescription Drug User Fee Act (PDUFA V). Federal Register Note: Due to impending inclement weather, DAY 2 of this meeting, February 13th, focusing on communication of uncertainty, will be postponed and rescheduled for the spring (tentatively April or May). The new workshop date will be announced via email and will be posted on the FDA and IOM meeting websites.
Public Workshop. “Biofilms, Medical Devices, and Anti-Biofilm Technology—Challenges and Opportunities.” February 20, 2014. Silver Spring, MD.
The purpose of the public workshop is to initiate dialogue between academia, industry, and U.S. Government scientists on the science of developing products to address biofilm formation. Topics of discussion include current scientific and medical research on biofilms, their impact on medical devices, and biofilm prevention strategies and their public health impact. The Center for Biofilm Engineering of Montana State University is co-sponsoring the workshop.
Public Advisory Committee Meeting. Cellular, Tissue, and Gene Therapies Advisory Committee. February 25-26, 2014.
The committee will discuss oocyte modification in assisted reproduction for the prevention of transmission of mitochondrial disease or treatment of infertility; hear updates on guidance documents issued from the Office of Cellular, Tissue, and Gene Therapies, CBER, FDA; and discuss considerations for the design of early-phase clinical trials of cellular and gene therapy products. CBER published guidance on this topic in July 2013. (Federal Register
Public Advisory Committee Meeting. Nonprescription Drugs Advisory Committee. February 26, 2014.
The committee will meet to discuss whether over-the-counter (OTC) bronchodilators administered by hand-held rubber bulb nebulizers for the temporary relief of mild symptoms of intermittent asthma (shortness of breath, tightness of chest, and wheezing) should be removed from the monograph. Specific drugs to be discussed include epinephrine, epinephrine bitartrate, and racepinephrine hydrochloride (21 CFR 341.16). (Federal Register
New: Public Meetings on Proposed Rule. Focused Mitigation Strategies To Protect Food Against Intentional Adulteration. February 27, 2014 (Chicago, IL); March 13, 2014 (Anaheim, CA).
FDA is announcing two public meetings to discuss the proposed rule to require domestic and foreign food facilities that are required to register under the Federal Food, Drug, and Cosmetic Act (the FD&C Act) to address hazards that may be intentionally introduced by acts of terrorism. FDA proposed these requirements as part of our implementation of the FDA Food Safety Modernization Act (FSMA). [Federal Register
Public Advisory Committee Meeting. Vaccines and Related Biological Products Advisory Committee. February 28, 2014.
The committee will meet in open session to hear an overview of the research program in the Laboratory of Respiratory Viral Diseases, Division of Viral Products, Office of Vaccines Research and Review, Center for Biologics Evaluation and Research, FDA. The committee will then discuss and make recommendations on the selection of strains to be included in the influenza virus vaccine for the 2014 to 2015 influenza season. [Federal Register
New: Public Workship. Advancing the Development of Pediatric Therapeutics: Pediatric Bone Health. March 4, 2014.
This 1-day public workshop entitled ‘‘Advancing the Development of Pediatric Therapeutics (ADEPT): Pediatric Bone Health,’’ will be conducted by the Pediatric and Maternal Health Staff in the Center for Drug Evaluation and Research and the Office of Pediatric Therapeutics. The purpose of this initial workshop is to provide a forum to consider issues related to advancing pediatric regulatory science in the evaluation of bone health in pediatric patients. [Federal Register
New: Public Workshop. Application of Physiologically-Based Pharmacokinetic Modeling To Support Dose Selection. March 10, 2014.
The purpose of the workshop is to obtain input on scientific approaches for the conduct and assessment of physiologically-based pharmacokinetic (PBPK) modeling within the framework of drug development and regulatory decisionmaking. The input from the workshop may be used to refine FDA’s thinking on the various applications of PBPK. Preliminary elements of a draft concept paper will be presented to facilitate discussion at this public workshop. [Federal Register
New: Public Conference. Serious Drug-Induced Liver Injury: Who Gets It? Who Doesn’t? Why? March 19-20, 2014. Hyattsville, MD.
This conference will be cosponsored with the Critical Path Institute (C-Path) and the Pharmaceutical Research and Manufacturers of America. Its purpose is to discuss, debate, and share views among stakeholders in the pharmaceutical industry, academia, health care providers, patient groups, and regulatory bodies on how best to detect and assess the severity, extent, and likelihood of drug causation of liver injury and dysfunction in people using drugs for any medical purpose. [Federal Register
New: Public Advisory Committee Meeting. Vaccines and Related Biological Products Advisory Committee. March 20, 2014. Rockville, MD.
(The public is welcome to attend the meeting at the specified location where a speakerphone will be provided.) the committee will meet in open session to hear updates of the research programs in the Laboratory of Respiratory and Special Pathogens, Division of Bacterial, Parasitic and Allergenic Products, and in the Laboratory of Hepatitis Viruses, Division of Viral Products, CBER, FDA. At least one portion of the meeting will be closed to the public. [Federal Register
New: Public Hearing. Over the Counter Drug Review. March 25-26, 2014. White Oak Campus, Silver Spring, MD.
The Agency would like input on how to improve or alter the current OTC Monograph Process for reviewing nonprescription drugs (sometimes referred to as OTC drugs) marketed under the OTC Drug Review. This public hearing is being held to obtain information and comments from the public on the strengths and weaknesses of the current OTC Monograph Process, and to obtain and discuss ideas about modifications or alternatives to this process. [Federal Register
Rescheduled Date. Public Meeting. FDA Patient Focused Drug Development: Fibromyalgia. March 26, 2014.
The public meeting is intended to allow FDA to obtain patients’ perspectives on the impact of fibromyalgia on daily life as well as the available therapies for fibromyalgia. [Federal Register
Stakeholders Meeting. International Medical Device Regulators Forum (IMDRF). March 26, 2014. San Francisco, CA.
All interested parties are invited to attend this full day open session. Meeting participants will be invited to discuss how they contribute to continuous improvements of products safety and performance, and how they face the emerging challenges. Stakeholders’ input is important to effective progress on harmonization issues. [FDA Meeting Notice
Rescheduled: Public Workshop: Synergizing Efforts in Standards Development for Cellular Therapies and Regenerative Medicine Products. March 31, 2014. FDA White Oak Campus. Silver Spring, MD.
The purpose of the public workshop is to bring together a broad range of stakeholders to discuss current and future standards development activities involving cellular therapies and regenerative medicine products. This public workshop was postponed in October 2013 due to the government shutdown. [Federal Register
New: Public Meeting. Study Approaches and Methods to Evaluate the Safety of Drugs and Biological Products During Pregnancy in the Post Approval Setting. May 28-29, 2014. FDA White Oak Campus, Silver Spring, MD.
The purpose of the public meeting is to engage in constructive dialogue and information sharing among regulators, researchers, the pharmaceutical industry, public health agencies, health care providers, and the general public concerning challenges in designing and implementing pregnancy registries and other methods of evaluating the post-approval safety profile of drugs and biological products in pregnant women. The input from this meeting and public docket will be used to support the revision of a guidance for industry on establishing pregnancy exposure registries. [Federal Register
Public Meeting. Meeting for Software Developers on the Common Formats for Patient Safety Data Collection and Event Reporting. April 25, 2014. Rockville, MD. Convened by AHRQ, this meeting is designed as an interactive forum where Patient Safety Organizations (PSOs – formed under the Patient Safety and Quality Improvement Act) and software developers can provide input on the formats. AHRQ especially requests participation by and input from those entities which have used AHRQ's technical specifications and implemented, or plan to implement, the formats electronically. (Federal Register)
New: Public Meeting on Patient-Focused Drug Development: Pulmonary Arterial Hypertension. May 13, 2014. FDA White Oak Campus, Silver Spring, MD.
Patient-Focused Drug Development is part of FDA’s performance commitments in the fifth authorization of the Prescription Drug User Fee Act (PDUFA V). The public meeting is intended to allow FDA to obtain patients’ perspectives on the impact of pulmonary arterial hypertension on daily life, as well as their perspectives on the available therapies for pulmonary arterial hypertension.
New: Symposium. 2014 Medical Countermeasures Initiative Regulatory Science Symposium. June 2-3, 2014. FDA White Oak Campus, Silver Spring, MD.
The symposium is intended to provide a forum for the exchange of scientific ideas for medical countermeasure development and evaluation, communicate progress on regulatory science efforts related to the development and advancement of medical countermeasures, facilitate innovative directions, and inform stakeholders on medical countermeasure-related scientific progress and accomplishments. [Federal Register
SECTION 4 OTHER REGULATORY AUTHORITIES & ORGANIZATIONS
Refining Processes for the Co-Development of Genome-Based Therapeutics and Companion Diagnostic Tests - Workshop Summary
Many drug developers have examined new strategies for creating efficiencies in their development processes, including the adoption of genomics-based approaches. Genomic data can be used to identify new drug targets for both common and rare diseases, can predict which patients are likely to respond to a specific treatment, and have the potential to significantly reduce the cost of clinical trials.
Key is the ability to identify the population of patients who will benefit from treatment, which is largely hinged on the co-development and co-submission to the U.S. FDA of a companion diagnostic test. The co-development process has led to a major alteration in the way that drugs are developed with pharmaceutical and diagnostic companies working in close collaboration.
This report summarizes the February 2013 IOM workshop which aimed to examine challenges and potential solutions for the co-development of targeted therapeutics and companion molecular tests for the prediction of drug response. More at title link above. (IOM)
Victor J. Dzau, M.D., to Be Next Institute of Medicine President
Victor J. Dzau, M.D., has been named the next president of the Institute of Medicine, the National Academy of Sciences announced today. Currently chancellor for health affairs at Duke University, and president and CEO for Duke University Health System, Dzau will begin his term on July 1, 2014, succeeding Harvey V. Fineberg.
Dzau is highly regarded as a trailblazer in translational research, health innovation, and global health care strategy and delivery. He was the guiding force in establishing the Duke Translational Medicine Institute, Duke Global Health Institute, Duke-NUS Medical School in Singapore, and Duke Institute for Health Innovation.
Dzau's own seminal research laid the foundation for the development of angiotensin-converting-enzyme (ACE) inhibitors, which are used globally for the treatment of high blood pressure and congestive heart failure. He pioneered gene therapy for vascular disease, being the first to introduce DNA decoy molecules to block transcriptions as gene therapy in humans. More at link above. (IOM)
Patient Consent to Research Not Always Necessary Bioethicists Say
Under the right conditions, full informed consent is not ethically required for some types of health research, according to leading bioethics experts. The experts focus in particular on the comparison of common treatments in the February 20 issue of the New England Journal of Medicine, arguing that in some cases a time-consuming consent process is not only unnecessary from the standpoint of protecting patients, but also potentially harmful to patients when it functions as an obstacle to gaining new knowledge that can improve the quality of the care patients receive.
“In a mature learning healthcare system with ethically robust oversight policies and practices, some randomized CER studies may justifiably proceed with a streamlined consent process and others may not require patient consent at all,” the commentary states.
However, the authors note that no such system exists today with fully developed ethical safeguards, including patient engagement, to allow a study impacting patient care to be ethically acceptable. More at title link above. (Johns Hopkins Berman Institute of Bioethics Press Release)
Pharma Use of Big Data Limited-Firms Need Dedicated Teams Consultant Urges
Cutting Edge Information recommends that pharma firms have dedicated big data teams; develop prospective big data strategies for using health outcomes, patient-reported outcomes and real-world data; and build research teams around individuals with industry and analytics expertise. (Pink Sheet 2/17 – DIA can access this for payment article. This was from the Big Data conference at the University of Maryland on 2/11)
NHS England Delays Patient Data Roll-out
The giant NHS database that could allow pharma firms to buy into patient records will now not be launched until the autumn, as NHS England admits it needs more time to convince the public of its merits. Due to start in April the system will now be delayed by around six months. An NHS England spokesperson said the agency had been told ‘very clearly’ by the public that more time was needed to understand the benefits of sharing information and their right to object.
The central database which will be called care.data, is to be built up from records from GP practices and links them with hospital records. The data will be managed by the Health and Social Care Information Centre (HSCIC). Researchers say it will enable them to assess diseases, examine new drugs on the market and identify infection outbreaks as well as monitor the performance of the NHS. More at link above. (Pharma File)
Dutch Startup Offers Fast Lane Late Stage Drugs for Patients with Dwindling Options
A Dutch life science startup is collaborating with biotechnology companies to make it easier for patients with advanced chronic conditions and unable to get into clinical trials to access potentially life-saving drugs before they are cleared by regulators. MyTomorrows positions itself as a patient-focused company and recently raised $2.2 million from friends and family to expand the treatments available for this program to include amyotrophic lateral sclerosis or Lou Gehrig’s disease, multiple sclerosis and Parkinson’s disease, among others.
The company conforms to each country’s compassionate-use provision that spells out circumstances in which patients who are low on options can gain access to drugs not yet officially approved by regulators by working with their physician.
MyTomorrows co-founder and CFO Erdem Yavuz told MedCity News that so far it has signed deals with five biotech companies and expects to add 10 to 12 more this year. More at link above. (MedCity News)
Indian regulator says his Agency will follow own quality standards
During Dr. Margaret Hamburg’s recent visit to India, she and ministry officials signed a statement of intent to achieve, among other things, "convergence in regulations in keeping with international standards". The agreement provides for U.S. and Indian regulators to inform each other before inspecting drugmakers' plants, so host-country inspectors can join as observers.
The Drug Controller General of India on Monday told Reuters that he sees scope for India's Central Drugs Standard Control Organization (CDSCO) working with the FDA and improving regulatory practice, adding that the Indian regulator will continue to follow its own quality standards.
"We don't recognise and are not bound by what the U.S. is doing and is inspecting," G.N. Singh said. "The FDA may regulate its country, but it can't regulate India on how India has to behave or how to deliver." Singh said his agency inspects manufacturing facilities in India regularly and that it plans to raise the number of inspectors to 5,000 in 3-5 years from the current 1,500.
Industry officials in India say that weak domestic regulatory oversight and a lax approach to quality control by some drugmakers means that a large number of sub-standard drugs reach the market undetected. More at link above. (Reuters)
SECTION 5 LEGAL, POLICY, AND COMPLIANCE NEWS
House Subcommittee to Discuss Proposed Part D Changes
The health subcommittee of the U.S. House of Representatives Energy and Commerce Committee is holding a hearing on Feb. 26 to discuss changes to Medicare Part D proposed by CMS in January. The proposed rule would eliminate two of the six protected classes for which plans must provide "substantially all" approved drugs and would limit the number of plans a given insurance company can offer in a single region. The proposed rule also would allow CMS to intervene in negotiations between payers and pharmacies.
Last week, patient groups told BioCentury the proposed changes to protected drug classes would reduce access to necessary medications for transplant and mental illness. Healthcare policy specialists and consultants said they are worried the proposed changes would reduce competition and eliminate preferred pharmacy networks and the discounts they provide. Comments on the agency's proposed rule are due March 7. More at link above. (BioCentury)
CMS Draft Cal Letter Silent on Protected Classes
A draft of CMS's annual call letter informing insurers of plan requirements for 2015 does not address a proposed change to Medicare Part D that would eliminate antidepressants and immunosuppressants from the list of protected classes for which plans must provide "substantially all" approved drugs.
Legislators continue to pressure CMS to withdraw the proposed changes. On Wednesday, Rep. Dave Camp (R-Mich.), chair of the U.S. House of Representatives Ways and Means Committee; Rep. Fred Upton (R-Mich.), chair of the House Energy and Commerce Committee; and Sen. Orrin Hatch (R-Utah), ranking member of the Senate Finance Committee sent a letter
to CMS Administrator Marilyn Tavenner and HHS Secretary Kathleen Sebelius. The legislators called the proposed changes "a bureaucratic overreach into a highly functioning marketplace that will undermine the success of the Part D program." More at link above. (BioCentury)
SECTION 6 SOURCES REVIEWED FOR THIS NEWSLETTER
A partial listing of sources reviewed for this newsletter: AdvaMed Smartbrief; AHRQ Newsletter; Alzheimers Association; Alzheimers Research Forum Newsletter; BioCentury; Biopharma Reporter; BIOtechNow; CDISC Monthly Newsletter; CER Daily Newsfeed (NPC); Daily Dose (Becker); DIA Daily; Drug Daily Bulletin; EMA website; EP Vantage; Evaluate Pharma; Eye on FDA; FDA.gov; FDA Law Blog; Federal Register Table of Contents; Fierce Medical Devices; Fierce Pharma; Fierce Vaccines; FDLI Smartbrief; Genomeweb; Health Industry Washington Watch; Government Health IT; Health IT Security; Institute of Medicine News; MedCityNews; Medical Device Daily; Medical Device & Diagnostic Industry; MedPage Today; NPC Bulletin; Nutra Ingredients USA; Pharmabiz; Pharmafile; Pharma IQ; PharmaTimes; PhRMA website; PM Live; Policy and Medicine (newsletter); Regulatory Focus; RegLink News; US FDA Daily Digest Bulletin