Category Archives: Drug Approvals

21st Century Cures Act Seeks to Expedite Bench to Bedside New Treatments


Author: Louis P. Sintasath, MS, MBA
ResearchDx, Director, Business Development

Recently this year the U.S. House of Representatives resoundingly passed a bipartisan resolution, H.R. 6, also known as the 21st Century Cures Act. So what is the 21st Century Cures Act and what is it trying to do? In short, the bill would reauthorize the National Institutes of Health (NIH) through FY2018 and provide $8.75 billion in additional funding through FY2020.

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For proponents of the 21st Century Cures Act, the mission is to improve the lives of every American by bringing innovation to the health care infrastructure, by reducing the time new therapies gets to the clinic, by paving the way for novel discoveries and cures, and by ultimately saving lives.
Internationally recognized as the “contract diagnostic organization,” ResearchDx specializes in the development of companion diagnostics and in vitro diagnostics for the pharmaceutical,biotechnology, research, and diagnostic industries, and is instrumental in new drug development.
According to Philip Cotter, PhD, FACMG, FFSc (RCPA), co-founder and Principal at ResearchDx, an Irvine, California based life sciences company, “The 21 Century Cures Act has long been needed to introduce the growing discipline of Translational Medicine.” Dr. Cotter further explains, “Translational Medicine is a rapidly growing discipline in biomedical research and aims to expedite the discovery of new diagnostic tools and treatments by using a multi-disciplinary, highly collaborative, “bench-to-bedside” approach. Within public health, translational medicine is focused on ensuring that proven strategies for disease treatment and prevention are quickly implemented within the community.”
Title I of the bill focuses on innovation and discovery. It promotes strategic planning for clinical research at the NIH as well as increase accountability at the NIH, reduce administrative burdens, and standardize data in the clinical trial registry data bank. It encourages collaboration among researchers and aims to remove barriers that discourage it. It supports emerging young scientists and establishes an Innovation Fund. The provision also promotes pediatric research through the establishment of the National Pediatric Research Network (NPRN).
The large bulk of the proposed bill lies in Title II. Title II revamps the regulatory processes for innovative drugs and medical devices. This involves the “development and use of patient experience data to enhance structured risk-benefit assessment framework,” says Dr. Cotter. It would also establish a new drug development tool that includes “(1) a biomarker; (2) a clinical outcome assessment; and (3) any other method, material, or measure that the Secretary determines aids drug development regulatory review.” To help advanced qualified drugs, there would be an accelerated approval development plan that includes agreement on surrogate endpoints. This provision has met with some resistance as some have raised concerns that an expedited process may result in increased risk to safety and efficacy.

Title II also lays out a provision on precision medicine, calling for two guidance documents to be added: “General agency guidance and precision medicine” and “Precision medicine regarding orphan-drug and expedited-approval programs.” Modern clinical trial design and evidence development would be implemented as well as a streamlined data review process.

Lawmakers also recognize the need for new antibiotic drug development, inserting a provision that allow for approval of certain drugs for use in a limited population of patients. For breakthrough medical devices where no approved alternatives exist, there will be a new priority review process for premarket approvals (PMA) as well as 510(k) devices. There will also be a formation of a third-party quality system assessment process. There will also be an easing of regulatory burdens for certain Class I and Class II devices.
Title III targets the disparate landscape of electronic health information technology. Title III calls for interoperability standards of electronic health information technology. In addition, the provision also expands the eligibility of telehealth coverage under the Medicare program and calls for the continuing education for physicians. Issues of price transparency are also addressed, along with patient safety and drug abuse prevention under Medicare.

Title IV proposes changes to Medicare and Medicaid to help offset costs of the program. The provision will establish for the first time an upper limit to the payment for durable medical equipment (DME) under the Medicaid program. There will be an incentive to move away from traditional X-Ray imaging to digital radiography. A provision that benefits drug manufacturers involves the ability to exclude authorized generics from the calculation of average manufacturer price (AMP). Because removing the low price of generics from the AMP, the price would be higher and thus the rebate to manufacturers would be higher on brand name drugs. The majority of funding for the program will come from a key provision to drawdown the Strategic Petroleum Reserve (SPR).
H.R. 6 has recently come under intense scrutiny over potential safety and efficacy concerns as well as loosening of FDA standards in the approval process. As the controversies play out in the media and the floor of the Senate, we will have to wait and see what provisions ultimately makes its way into law.

For the full text of H.R. 6, please visit:

FDA-Ok’d Drugs W/ Biomarkers Growing

Targeted drugs, personalized medicine, stratified therapy–whatever you call it, using biomarkers to identify particular patients for particular drugs has been hailed as a boon for patients and a savvy strategy for pharma.

Advocates can talk up approval numbers, labeling changes and Phase III therapies. But it’s according to the 80/20 rule: A small number of pharma companies account for the lion’s share of targeted meds. And the star of personalized medicine is just the company you’d expect: Roche ($RHHBY), with its drug-plus-diagnostic approach to cancer R&D, its stable of blockbuster HER2-positive therapies, and a total of almost $20 billion in sales from its targeted drugs.

Just ask Diaceutics, the U.K. research firm, which keeps close tabs ondiagnostics-aided medicine. Yes, the number of FDA-approved drugs linked with a particular biomarker has leapt over the past three years, to more than 80 from just over 20. And while only 7 new drugs with companion diagnostics have made their market debuts, 53 others have new FDA labeling that flags safety-related biomarkers–bringing the percentage of targeted therapies on the market to 19% from 6% in 2010.

This blog post was originally written by Tracy Staton with the title Who are the stars of personalized meds? Roche, Novartis and J&J

New Drugs at FDA: CDER’s New Molecular Entities and New Therapeutic Biological Products

Innovation drives progress. When it comes to innovation in the development of new drugs and therapeutic biological products, FDA’s Center for Drug Evaluation and Research (CDER) supports the pharmaceutical industry at every step of the process. With its understanding of the science used to create new products, testing and manufacturing procedures, and the diseases and conditions that new products are designed to treat, FDA provides scientific and regulatory advice needed to bring new therapies to market.

Novel New Drugs
The availability of new drugs and biological products often means new treatment options for patients and advances in health care for the American public. For this reason, CDER supports innovation and plays a key role in helping to advance new drug development.
Each year, CDER approves a wide range of new drugs and biological products. Some of these products are innovative new products that never before have been used in clinical practice. Others are the same as, or related to, previously approved products, and they will compete with those products in the marketplace.
Certain drugs are classified as new molecular entities (“NMEs”) for purposes of FDA review. Many of these products contain active moieties that have not been approved by FDA previously, either as a single ingredient drug or as part of a combination product; these products frequently provide important new therapies for patients. Some drugs are characterized as NMEs for administrative purposes, but nonetheless contain active moieties that are closely related to active moieties in products that have previously been approved by FDA. For example, CDER classifies biological products submitted in an application under section 351(a) of the Public Health Service Act as NMEs for purposes of FDA review, regardless of whether the Agency previously has approved a related active moiety in a different product. FDA’s classification of a drug as an “NME” for review purposes is distinct from FDA’s determination of whether a drug product is a “new chemical entity” or “NCE” within the meaning of the Federal Food, Drug, and Cosmetic Act.