Life Science Compliance Update

July 28, 2017

FDA Orphan Drug Modernization Plan Released



In late June 2017, the U.S. Food and Drug Administration (“FDA”) unveiled a strategic plan to both eliminate the agency’s existing orphan designation request backlog and ensure timely responses to all new requests for designation with firm deadlines.


This Orphan Drug Modernization Plan comes hot on the heels of FDA Commissioner Scott Gottlieb’s testimony before a Senate subcommittee, where he made a commitment to (1) eliminate the current backlog within ninety days and (2) respond to all new requests for designation within ninety days of receipt.


Authorized under the Orphan Drug Act, the Orphan Drug Designation Program provides orphan status to drugs and biologics that are defined as “those intended for the safe and effective treatment, diagnosis or prevention of rare diseases,” which are generally defined as diseases that affect fewer than 200,000 people in the United States. Various incentives, including tax credits for clinical trial costs, relief from the prescription drug user fee, and eligibility for seven years of marketing exclusivity are some of the incentives for manufacturers to develop orphan drugs.


Currently, the FDA has roughly 200 orphan drug designation requests pending review. The number of orphan drug designation requests has steadily increased over the past five years: in 2016, the FDA’s Office of Orphan Products Development received 568 new requests for designation – more than double the number of requests received in 2012.


“People who suffer with rare diseases are too often faced with no, or limited, treatment options, and what treatment options they have may be quite expensive due in part to significant costs of developing therapies for smaller populations,” said FDA Commissioner Scott Gottlieb, M.D. “Congress gave us tools to incentivize the development of novel therapies for rare diseases and we intend to use these resources to their fullest extent in order to ensure Americans get the safe and effective medicines they need, and that the process for developing these innovations is as modern and efficient as possible.”


This is the first of several efforts the FDA plans to undertake under its “Medical Innovation Development Plan,” which is aimed at ensuring that the FDA’s regulatory tools and policies are modern, risk based, and efficient. The goal of the plan is to seek ways the FDA can help facilitate the development of safe, effective and transformative medical innovations that have the potential to significantly impact disease and reduce overall health care costs.


To ensure all future requests receive a response within 90 days of receipt, the agency will take a multifaceted approach. These efforts include, among other new steps: reorganizing the review staff to maximize expertise and improve workload efficiencies; better leveraging the expertise across the FDA’s medical product centers; and establishing a new FDA Orphan Products Council that will help address scientific and regulatory issues to ensure the agency is applying a consistent approach to regulating orphan drug products and reviewing designation requests.


The agency intends to communicate around the successful elimination of the backlog by mid-September and will soon provide more information about the Medical Innovation Development Plan. 

July 14, 2017

PhRMA Report Shows More than 240 Immuno-Oncology Treatments in Development

ACS CAN logoACS CAN logo

In early June 2017, the Pharmaceutical Research and Manufacturers of America (PhRMA) – in partnership with the American Cancer Society Cancer Action Network (ACS CAN) – released a report that found there are over 240 immuno-oncology medicines and vaccines currently in development.

Immuno-oncology treatments are found through research into the role of the body’s immune system in fighting cancer. New immuno-oncology treatment options are allowing the patient’s own immune system to fight cancer similar to the way it fights disease-causing viruses and bacteria. The treatments can help the patient’s own immune system to work against cancer, with the potential for lasting results.

According to the report, there is no single accepted definition of immuno-oncology. However, this report includes many of the most recognized classes: adoptive cell therapies (including CAR-T therapy), bi-specific antibodies, cytokines, immune checkpoint modulators, oncolytic virus therapies, and vaccines. Former President Jimmy Carter was a notable recipient to immunotherapy treatment in his battle to fight metastatic melanoma.

In addition to the medicines currently in development, researchers are working to understand the full potential of each individual medicine, seeking approval for new indications for currently-existing immunotherapies, as well as new uses in combination with other cancer medicines.

“Cancer continues to be one of the most complex and vexing diseases of our time and it will impact an estimated 1.6 million Americans who will be diagnosed this year,” said Stephen J. Ubl, president and chief executive officer of PhRMA. “As our understanding of the root causes of cancer grows, we are expanding the types of treatments we are able to bring to patients. The idea of harnessing the body’s own immune system to fight cancer is not new but recent breakthroughs are making it a reality, bringing hope to patients.”

“We are at a moment of tremendous opportunity when it comes to developing therapies that can address even the most vexing cancers we see today,” said Chris Hansen, president of ACS CAN. “To fully leverage our potential to reduce suffering and death from cancer, robust and sustained federal investment in basic research is critical to provide the necessary building blocks that together with privately-funded innovation lead to advances in immunotherapy and other targeted treatments.”

In 2016, then-president Barack Obama announced the creation of the Cancer Moonshot Initiative. Immunotherapy research was identified as a priority and a blue-ribbon panel of experts called for the creation of both adult and childhood immunotherapy clinical trial networks designed with the needs of cancer immunotherapy research in mind. As part of the Moonshot initiative, the American Cancer Society – the largest nongovernmental funder of cancer research in the United States – also committed to a goal of doubling its own research budget to help reach the national goal of accelerating progress.

Increases in federal research funding, like those advocated for by ACS CAN, enable continued growth in our understanding of cancer immunotherapy, and allows important investments in research infrastructure such as the Moonshot-recommended immunotherapy trial network. Those federal investments in basic research provide the necessary building blocks to fuel further privately-funded innovation.

The report was released as part of PhRMA’s GOBOLDY campaign, which showcases the bold advancements the industry is making in tackling some of the most complex and devastating health conditions through innovative research. While the biopharmaceutical industry is a leader in the development of innovative treatments, it also plays a critical role along with others in the health care ecosystem to help bring new medicines to patients.

June 29, 2017

Chicago Releases Pharmaceutical Representative Disclosure Log Draft


The City of Chicago recently released a draft of the disclosure log pharmaceutical representatives will be expected to use to keep track of the interactions they have with Chicago physicians. The form requires pharmaceutical representatives to log the following information with respect to any interactions they have with physicians within city limits: HCP first name; HCP middle initial/name; HCP last name; HCP name suffix (i.e., Jr.); HCP primary business address; HCP license type (i.e., MD, DO, etc.); HCP state license number; HCP NPI (if applicable); date of interaction; location of interaction; duration of interaction; pharmaceuticals promoted; whether drug samples were provided, and if so, the quantity of samples given and the value of such samples; whether pharmaceutical-related materials were given, if so, the value of the materials given; and whether any other items of value or compensation were given, if so, what type and the combined value of other items.

The log includes instructions, which state,

Use one line per interaction. An interaction is any instance in which you communicate with an HCP as part of your work as a pharmaceutical representative, whether in person, over the phone, via video conference, by email, or via another communications method, as well as any time you leave materials or samples for that HCP, even if you do not communicate personally. However, you do not need to report a telecommunication or written communication if it was done simply to set up a meeting or other communication with an HCP and no marketing or promotion took place. It is not necessary to include time spent in a waiting room before meeting an HCP when reporting the duration of the interaction. If the options in any of the dropdown menus do not provide a perfect description of the contact, select the closest option. However, if the "HCP license type" dropdown menu does not include the license type of the HCP with whom you interacted, you do not need to disclose the interaction at all. If you interact with multiple HCPs at one time, for example through a dinner or entertainment event with several doctors, include a line for each HCP. If you cannot precisely break down the number of items or amount of compensation that went to each HCP because you provided them as a set to multiple HCPs, please report the average amount per HCP by dividing the number of items and the compensation value by the total number of HCPs. For instance, if you give a box of 50 samples to two HCPs jointly, mark down 25 apiece. For group meals or other forms of compensation that you provide to HCPs and non-HCPs jointly — like a lunch for a medical office that includes four HCPs and one receptionist — figure out the per-person cost and report that for each HCP. For instance, if you were to provide that office (four HCPs, one receptionist) with a $100 lunch, you would report $20 in food and beverages for each HCP.

The instructions are quite interesting and actually create more questions than they answer. While the next box explains that only interactions that take place while both parties are within the Chicago city limits should be reported, does that mean licensed representatives in Chicago have to disclose emails to physicians if both parties are within city limits when the email chain is started? What if the emails continue, after one party exits city limits?

The proposed disclosure form is draconian and adds unnecessary requirements for Chicago pharmaceutical representatives and the physicians they interact with.


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