A week after the FDA finalized its guidance on enhancing diversity in clinical trials, the Pharmaceutical Research and Manufacturers of America (PhRMA) released the first industrywide principles on trial diversity Nov. 17.

Included as a new chapter to PhRMA’s Principles on Conduct of Clinical Trials and Communication of Clinical Trial Results, the diversity principles focus on four main areas: building trust and acknowledging the historic mistrust of clinical trials within Black and ethnic communities, reducing barriers to clinical trial access, using real-world data to enhance information on diverse populations beyond product approval, and enhancing information about diversity and inclusion in clinical trial participation.

In announcing the voluntary principles, which will go into effect in April, PhRMA President and CEO Stephen Ubl said, “We are addressing issues of mistrust and working to reduce systemic issues that deter communities of color from participating in clinical trials, so that those patients who want to participate can.”

The principles recognize both the medical and societal benefits of enhancing trial diversity. For instance, having meaningful representation of diverse participants will provide information about a drug’s safety and efficacy in populations that are historically under-represented and under-studied, PhRMA said.

Lack of participation in trials also can limit patients’ early access to potential therapies, especially in areas of unmet medical need. That lack of access can further health disparities. Then there is the ethical matter in which the clinical development of drugs should serve the needs of those who are affected by the disease or condition, PhRMA said.

Going forward, the trade organization said it is committed to continuing to work with patients, patient advocacy groups, regulators, health care professionals, academics and policymakers to “define the systematic and impactful approaches that we will take to enhance the diversity of clinical trial participants and help reduce healthcare disparities.”

Some of those efforts will look at ways to make trial participation less burdensome, design trials with the needs of diverse populations in mind, and broaden eligibility criteria when scientifically and clinically appropriate.

In many ways, the new industry principles reflect the FDA’s final guidance. However, PhRMA’s principles specifically reference only demographic diversity – race, ethnicity, sex/gender and age – whereas the FDA’s guidance also stresses the need for diversity in terms of non-demographic characteristics such as organ dysfunction, co-morbid conditions, rare diseases, disabilities and weight.

The FDA has been pushing for greater trial diversity for several years. But despite those efforts, challenges continue, the agency said in its guidance. Part of the continuing lag in trial diversity could be attributable to the fact that nearly two-thirds of drug trials conducted in the past five years were outside the U.S., many of them in countries with less diverse populations.

CDER, CBER promise EUA transparency

With emergency use authorization (EUA) requests in the offing for COVID-19 vaccines, the FDA’s Center for Drug Evaluation and Research (CDER) and the Center for Biologics Evaluation and Research (CBER) are taking additional steps to promote public confidence in their reviews of the data and the products they authorize for emergency use.

The two centers said that, in the future when EUAs are granted, revised or revoked, they intend to publicly post, to the extent legally permissible, their reviews of the data and information supporting the regulatory decisions. The reviews will be posted with other EUA-related materials on the FDA website.

“Today’s transparency action is just one of a number of steps we are taking to ensure public confidence in our EUA review process for drugs and biological products, especially any potential COVID-19 vaccines,” FDA Commissioner Stephen Hahn said Nov. 17.

Hahn also reiterated his promise to schedule a meeting of the Vaccines and Related Biological Products Advisory Committee upon submission of an EUA request for a COVID-19 vaccine to discuss whether the available safety and effectiveness data support an EUA.

FDA plasma guidance updated, again

The FDA this week once again updated its guidance on COVID-19 convalescent plasma – this time to include a recommendation that convalescent plasma meeting the requirements of the EUA should not be collected from people who have received an investigational COVID-19 vaccine.

The update also gives blood establishments more time, until Feb. 28, 2021, to develop the necessary operating procedures to manufacture the plasma consistent with the EUA, including the testing of plasma donations for anti-SARS-CoV-2 antibodies using the Ortho Vitros SARS-CoV-2 IgG and then qualifying units as high titer or low titer based on that testing.

In a September update to the guidance, the agency had given blood establishments 90 days to adopt the new procedures. If they were unable to do so by then, they were to be limited to administering investigational convalescent plasma under an investigational new drug application.

“This extension will allow continued access to convalescent plasma for the treatment of hospitalized COVID-19 patients” while the blood establishments work to meet the EUA manufacturing requirements, the FDA said.

WHO targets cervical cancer

It sounds like an audacious goal, but the World Health Organization (WHO) thinks it’s doable: eliminate cervical cancer worldwide.

WHO launched its global strategy Nov. 17 to accelerate the elimination of cervical cancer, with the goal of reducing more than 40% of new cases of the disease and 5 million related deaths by 2050 through vaccination, screening and treatment.

The organization called the launch a historic milestone as it marked the first time that 194 countries have committed to eliminating cancer.

“Eliminating any cancer would have once seemed an impossible dream, but we now have the cost-effective, evidence-based tools to make that dream a reality,” WHO Director-General Tedros Adhanom Ghebreyesus said. “But we can only eliminate cervical cancer as a public health problem if we match the power of the tools we have with unrelenting determination to scale up their use globally.”

WHO’s strategy calls for meeting the following targets by 2030:

  • 90% of girls fully vaccinated with the HPV vaccine by 15 years of age;
  • 70% of women screened using a high-performance test by age 35 and again by 45;
  • 90% of women identified with cervical disease receiving treatment (90% of women with pre-cancer treated and 90% of women with invasive cancer managed).

However, if no action is taken, the annual number of new cases of cervical cancer is expected to increase from 570,000 in 2018 to 700,000 by 2030, WHO said, while the annual number of deaths is projected to rise from 311,000 to 400,000. The biggest toll would be in low- and middle-income countries where the incidence of cervical cancer is nearly twice as high and its death rates three times as high as those in high-income countries.

Draft guidance to help FDA respond to 2012 citizen petition

The FDA issued a draft product-specific guidance to help sponsors develop generics of Boehringer Ingelheim GmbH’s Spiriva Respimat (tiotropium bromide inhalation spray).

Like other product-specific guidances, the draft addresses bioequivalency issues. But in this instance, the FDA said it also is looking to comments about the draft to help it respond to a citizen petition Boehringer submitted in 2012 in which it asked the agency to adopt and apply certain requirements in its review of any proposed generics referencing Spiriva Handihaler or subsequent Boehringer oral inhalation products containing tiotropium bromide. (Spiriva Respimat was approved in 2015.)

Comments on the draft guidance should be submitted to Docket No. FDA-2007-D-0369 by Jan. 17, according to a notice to be published in the Nov. 18 Federal Register.

More time to comment on instituting PTAB trials

The U.S. Patent and Trademark Office (USPTO) is extending its request for public input on the agency’s discretion in instituting inter partes reviews and other trials before the Patent Trial and Appeal Board (PTAB).

As it looks at codifying, through the government rulemaking process, its policies and practices for instituting PTAB trials, the USPTO said it wants comments on its current approach, as well as alternative approaches.

The comment period, which was to end Nov. 19, has been extended to Dec. 3. Comments should be submitted to Docket No. PTO-C-2020-0055.

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