Life Science Compliance Update

June 17, 2016

PhRMA Members Invested $58.8 Billion in R&D in 2015

In 2015, PhRMA member companies invested $58.8 billion in research and development, up 10.3% from 2014. The new R&D data is based on findings from the 2016 PhRMA annual member survey released in the 2016 Biopharmaceutical Research Industry Profile and the corresponding industry chart pack, Biopharmaceuticals in Perspective, which highlighted the wide-reaching impact of PhRMA member companies on the economy and biopharmaceutical innovation.

In the United States, the biopharmaceutical industry is a driver of economic growth and global competitiveness, and is the most research-intensive sector of the economy. The biopharmaceutical industry invests an average of six times more in R&D as a percentage of sales than all other manufacturing industries. The sector also accounted for approximately 17% of all business R&D spending by U.S. businesses. Overall, PhRMA member companies represented the majority of all biopharmaceutical R&D spending in the United States.

According to Stephen J. Ubl, president and CEO of PhRMA,

Investing more than half a trillion dollars in R&D since 2000, our member companies remain tireless in their commitment to driving innovation and delivering greater value than ever before. It is through this increased R&D that the U.S. biopharmaceutical industry continues to lead the world in the development of new medicines to address unmet medical needs of patients.

The increase in long-term R&D investments made by the biopharmaceutical industry have led to more medicines in clinical development than ever before, more than 7,000 medicines globally. As a sign of how increased R&D can really help, from 2000 to 2015, more than 550 new medicines were approved by the United States Food and Drug Administration (FDA) – including 56 new medicines in 2015 alone. Since only 12% of medicines in clinical trials make it to patients, it is critical that there are pro-innovation policies in place that can help sustain the long-term investments needed to develop tomorrow's cures.

As noted in the recently-released "Medicines in Development for Rare Diseases," the biopharmaceutical industry is currently developing more than 560 medicines for patient with rare diseases. The industry is also working to find cures and other treatments for Alzheimer's, cancer and heart disease, and other devastating conditions. This progress once again makes clear that public policies are needed that maintain a health care system that recognizes the value of medicines and incentivizes researchers to continue to develop new treatments and cures for patients.

Of those 560 medicines currently in development for rare diseases, 151 are for rare cancers and 82 are for rare blood cancers; 1468 are for generic disorders, including cystic fibrosis and spinal muscular atrophy; 38 for neurological disorders, including ALS and seizures; 31 for infectious diseases, including rare bacterial infections and hepatitis; and 25 for autoimmune diseases, including systemic sclerosis and juvenile arthritis.

ALS (also known as Lou Gehrig's disease), is notorious for being a rare disease with no cure. However, new therapies that are currently under development, such as antisense technology against SOD1, are a step toward helping patients and their families manage the disease.

These figures and anecdotes show that the biopharmaceutical industry is delivering true value to Americans by transforming patient's lives, lowering projected health care costs, strengthening the United States economy, and helping to improve the drug review and approval process, all while continuing to invest for the long term health of our country.

May 26, 2016

NIH Asks for Input on NCAB Cancer Moonshot Blue Ribbon Panel

Recently, the National Institutes of Health (NIH) released a Request for Information (RFI), asking for both public and cancer research community input on the National Cancer Advisory Board (NCAB) Blue Ribbon Panel. The Blue Ribbon Panel is part of the National Cancer Moonshot Initiative, which is led by Vice President Joe Biden and aims to make more cancer therapies available, while continuing to improve cancer prevention and early detection. Additional details of the National Cancer Moonshot Initiative can be found at

The purpose of the NCAB is to ensure that the National Moonshot Initiative's goals and approaches are grounded in solid science. The Blue Ribbon panel is a panel of experts assembled to guide the NCAB's work and is composed of leading experts from a broad range of scientific areas including biology, immunology, genomics, diagnostics, bioinformatics, and cancer prevention and treatment. The panel does not just include researchers, however, it also includes clinicians and nurses, as well as representatives of cancer advocacy organizations and the pharmaceutical and biotechnology industries.

NIH is seeking community input to help "enable the Blue Ribbon Panel to consider a wide range of input from researchers, scientists, physicians, advocates, students, data scientists, and members of the public." They are requesting input in the following specific areas: expanding clinical trials; enhanced data sharing; cancer immunology and prevention; implementation sciences; pediatric cancer; precision, prevention, and early detection; and tumor evolution and progression.

The request for information is for planning purposes only and should not be construed as a solicitation for applications or proposals, or as an obligation on the part of the United States federal government.

If you have any questions about this particular RFI, you can contact Kelli Marciel at the National Cancer Institute at 9609 Medical Center Drive, Bethesda, MD 20892-9760,, or (301) 594-3330.

NIH is asking for responses to be submitted to either the National Cancer Institute (NCI) or NIH before July 1, 2016 at 5:00 pm EST. Responses can be submitted electronically through the online platform or via email to While electronic responses are preferred, non-electronic responses can be accepted by calling the NCI Cancer Information Service at (800) 422-6237, or by mail to Blue Ribbon Panel, National Cancer Moonshot Initiative, National Cancer Institute, 9609 Medical Center Drive, Bethesda, MD 20892-9760.


February 29, 2016

FDA Guidance: Determining the Extent of Safety Data Collection Needed in Late-Stage Premarket and Postapproval Clinical Investigations

The FDA finalized a guidance it originally released in 2012 that is aimed to help industry collect safety data in late-stage premarket and postapproval clinical investigations. The FDA says in the guidance that selective safety data collection may be possible for some late-stage premarket and postapproval clinical investigations because "certain aspects of a drug's safety profile will be sufficiently well-established and comprehensive data collection is not needed."

Safety Databases

Safety databases collect information to accurately assess and characterize the risks of a new drug. A sponsor collects safety-related data during the course of drug development and knowledge of a drug's safety profile can change as more information comes in through its lifecycle. However, FDA notes that encouraging selective safety data collection in late-stage premarket and postapproval clinical investigations is consistent with the agency's approach to safety assessment which is focused on information that is useful and adds to current knowledge.

FDA outlines the growing interest in larger, simpler trials to obtain outcome data, data on long-term effects of drugs, and data on comparative effectiveness and safety. According to the agency, excessive safety data collection may discourage the conduct of these types of trials by increasing the resources needed to perform them and be a disincentive to investigator and patient participation in clinical trials. Therefore, FDA believes its guidance will facilitate the conduct of larger trials without compromising the integrity and the validity of trial results or losing important information, facilitate investigators' and patients' participation in clinical trials, and help contain costs by making more-efficient use of clinical trial resources.

Selective Safety Data Collection

FDA issues several examples of selective safety data collection, including no collection of certain safety data, less-frequent collection of certain safety data, and collection of certain safety data from only a fraction of the total trial enrollment. The agency writes that in general, selective data may be appropriate for certain types of safety data if:

  • The number of patients and their characteristics, the duration of exposure, and the dose range used in previous clinical investigations are sufficient to adequately characterize the safety profile of the drug for common, non-serious adverse events.
  • The occurrence of common, non-serious adverse events has been generally similar across multiple clinical investigations.
  • The drug's safety profile is established to the extent that it is reasonable to conclude that the occurrence of common, non-serious adverse events in the population to be studied will be similar to rates observed in previously conducted clinical investigations.

FDA also clarifies when selective safety data collection may be appropriate, noting the following types of clinical investigations:

  • Clinical investigations of new indications of approved drugs, if an existing safety database is relevant to such investigations.
  • Postapproval clinical studies and trials conducted to fulfill postmarketing requirements and postmarketing commitments. For example, when the study population in these types of studies is generally the same as or similar to the population from which the premarket safety database was derived, safety data collection can often be limited to the primary safety endpoint and other endpoints of interest.
  • Late-stage premarket and postapproval outcome clinical trials.
  • Premarket clinical investigations for some original applications, unless sufficient safety data already exist to adequately characterize the safety profile of a drug, comprehensive safety data collection is expected.
  • Postapproval clinical investigations in a different patient population or with different doses or other conditions of use. Selective safety data collection generally may not be appropriate in clinical investigations of marketed drugs in which there are important differences in the patient population, dose, dosage regimen, duration of use or route of administration compared with the conditions of use for the marketed indications.

Additionally, when selective safety data collection is suitable, it may be appropriate to limit collection of data or stop collection of some data, such as:

  • Non-serious adverse events not associated with dose modification, drug discontinuation, or withdrawal from the trial.
  • Routine laboratory monitoring as in many cases it may be possible to eliminate routine laboratory monitoring or to decrease the frequency of monitoring certain laboratory parameters.
  • Information on concomitant medications; if existing data satisfactorily characterize all anticipated drug-drug interactions and metabolic pathways, additional detailed information on concomitant medications may be of limited use, particularly for drugs used only short term.
  • Patient history and physical exams, where less-detailed histories and less-frequent physical exams for patients may be appropriate in some circumstances, especially in outcome clinical trials.


FDA writes that other considerations for safety data collection including the collection of complete safety data in population subsets and collection of complete safety data that identifies risk factors. The guidance also specifies that sponsors should be aware of data types that are generally not appropriate for selective safety data collection and should always be collected. This includes:

  • Data on all serious adverse events.
  • Data on non-serious adverse events that lead to dose modification, drug discontinuation, or withdrawal from the trial.
  • Data on unscheduled study visits, hospitalizations, and accidental injuries because these events may reflect serious adverse events of the drug.
  • In an oncology setting, data from all Grade 3 and Grade 4 adverse events, as well as Grade 2 adverse events that affect vital organs (e.g., heart, liver).
  • In development programs for rare disease indications, complete safety data should be collected, because these trials generally have limited patient populations and are unlikely to meet the recommendations for selective safety data collection.

For these types of safety data, it is generally important to collect information on all occurrences to better understand the following:

  • Causality;
  • Incidence;
  • Severity of adverse events;
  • Populations that are at risk;
  • Dose-response;
  • Other factors that contribute to our understanding of the nature of the event and who is at risk.

In its final section, FDA added language titled, "Agreement between sponsors and FDA on a plan for selective safety data collection." FDA writes that sponsors should discuss its specified plan with the relevant FDA review division or divisions at the appropriate time (e.g., at the end-of-phase II meeting for selective safety data collection for a phase III trial). The agreement reached should be incorporated into the procedures for safety data collection in the protocol, the monitoring plan, and other appropriate trial documents, according to the agency.


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