Study Finds Average Cost of $1.3 Billion for Drug R&D

Drugmakers spend an average of $1.3 billion in research and development costs to get a new medicine to market, according to a new study reported in JAMA last week, a number considerably lower than a 2016 Tufts University report citing development costs of $2.8 billion.

The research team from the London School of Economics and Political Science, the London School of Hygiene and Topical Medicine, and the Leuven Institute of Healthcare Policy looked at 63 FDA-approved drugs developed by 47 pharma companies between 2009 and 2018 and estimated the R&D costs for each one based on data available to the public, finding an average cost of $1.3 billion. The 2016 Tufts report was based on 106 randomly selected new drugs from just 10 companies.

Per product, the median R&D cost was $985 million, including the expenses from failed trials, the researchers said. Investments ranged from $143 million for Jaguar Health’s Mytesi (crofelemer), an anti-diarrheal drug for HIV patients, to $6.4 billion for Sanofi and Regeneron’s blockbuster Dupixent (dupilumab), an eczema biologic.

Development costs differed widely depending on the drug’s therapeutic area. The median cost for nervous system drugs, for example, was $765.9 million, while the most expensive area, cancer, had a median cost of $2.7 billion.

Access the JAMA study here: https://bit.ly/2x6i3yF.

Complex Trial Design Pilot Has Seen 50 Percent Acceptance Rate, FDA Says

Four of eight complex trial designs submitted in the FDA’s complex innovative design (CID) pilot program have been accepted for review, the agency said Monday.

The five-year pilot has accepted CIDs in several therapeutic areas, including treatments for Duchenne muscular dystrophy, pediatric multiple sclerosis, systemic lupus erythematosus and multiple interventions across multiple pain conditions by sponsors Eli Lilly, Amgen and others.

The agency denied half of the submissions based on a lack of clarity on endpoints and a low level of innovation, said Dionne Price, director of the FDA’s Division of Biometrics IV, during a two-day Drug Information Association meeting held in Silver Spring, Md. Although the designs were rejected for the pilot program, sponsors may continue using other regulatory routes, she said.

Sponsors whose designs are accepted for review in the pilot that was launched in August 2018 get increased access to the agency, including two primary meetings and two alternate meeting requests per quarter. Prior to the meetings, the FDA and participants work out a disclosure agreement on what information the agency may include in public case studies.

Price hailed the program’s disclosure element as one of its greatest benefits because it allows for discussions to happen while pre-INDs are being investigated. “The disclosure piece allows us to talk about pre-INDs while the drugs are still under investigation, in an effort to advance science. That has been a key success from my perspective.”

Test Topical Skin Products for Risk of Contact Dermatitis, FDA Guidance Says

Clinical trials of drug products intended for topical application should assess the risk of contact dermatitis, a new FDA draft guidance says.

The guidance advises researchers to use static scales, such as current state and noncomparative methods, to evaluate signs of cutaneous issues such as erythema, edema and erosion.

Patient-reported outcome measures should be used to assess symptoms such as pruritus or burning.

Trials should plan the timing and frequency of assessments to identify anticipated reactions and characterize suspected adverse reactions using patch or photopatch testing with the active and excipient ingredients as well as the finished product.

The deadline for comments on the draft guidance is May 8.

Read the guidance here: https://bit.ly/3auJ7Gc.

Increase Representation of the Elderly in Cancer Trials, FDA Guidance Says

The FDA is encouraging cancer trials to pay greater attention to patients age 75 and older, according to a draft guidance issued last week.

Adults age 65 and older, especially those over 75, are underrepresented in clinical research on cancer despite comprising a growing portion of the cancer patient population, the agency said.

Sponsors need to discuss their enrollment goals with sites and investigators to achieve greater participation of elderly patients. The guidance also recommends recruiting more physicians specializing in geriatric oncology as investigators.

Because outcomes of cancer patients age 65 and older may vary by age, the guidance encourages sponsors to evaluate data by subgroups, for example, patients age 65 to 74 vs. patients age 75 or older.

Comments on the draft guidance are due May 6.

Read the guidance here: https://bit.ly/2ww9MDZ.

Pharma Industry Outpaces NIH, Academia in Sponsoring Gene Therapy Trials

Pharma companies outstrip all other sponsors of gene therapy trials, running 40 percent compared to 25 percent by hospitals, 25 percent by universities and 10 percent by the NIH.

Sponsorship varied by trial type and phase, according to a new study published in JAMA, which tracked 341 gene therapy trials posted to clinicaltrials.gov.

Industry sponsored 61 percent of in vivo trials, 61 percent of trials for therapies correcting genetic defects, 63 percent of noncancer trials, 27 percent of ex vivo trials, 30 percent of trials for therapies not correcting genetic defects and 27 percent of cancer trials. Industry sponsored 22 percent of phase 1 trials, 55 percent of phase 2 trials and 100 percent of phase 3 trials.

The study, which came from authors at the University of Pittsburgh School of Medicine and Brigham and Women’s Hospital in Boston, also looked at the funding of gene therapy trials.

Pharma companies were the sole funder for 36 percent of gene therapy trials compared to 50 percent for NIH and academia, with the remaining 14 percent coming from collaborations among the three funding groups.

To read the study, click here: https://bit.ly/2TpGxf2.

FDA Releases Request for Applications for Funding of Rare Disease Trials

The FDA will begin accepting applications for grants for 2020 beginning this August for trials of drugs that address unserved needs in rare diseases.

The agency’s Office of Orphan Products Development awards $15 million a year — $10 million to ongoing trials and $5 million to new trials. In 2019, the FDA awarded grants to 12 applicants out of 89 received.

The FDA encourages applicants to apply early to allow adequate time to make corrections. The deadline for 2020 applications is Oct. 6, and the deadline for next year is Oct. 5, 2021.

To read the FDA’s request for applications, click here: https://bit.ly/2POBliw.

Study Identifies Disparities in Clinical Trial Access in U.S.

A lack of geographically convenient trial sites hinders study participation, especially for rural residents.

A JAMA study published last week concluded that unequal distribution of trial sites relative to population may impede patients’ participation in trials and contribute to the well-documented low representation of minorities in many trials.

Data on population, median household income, racial demographics, funding and number of U.S. inpatient hospital beds used in trials was collected.

Factors related to trial recruitment and retention, not the location of trial sites, are most likely responsible for racial and socioeconomic inequities in trial access and participation, the study concluded.

The study was conducted by authors from Pharmagellan, Boston University School of Public Health and Columbia University.

To read the study, click here: https://bit.ly/2xeEPVj.

New Illinois Law Ensures All Patients with Cancer Can Participate in Clinical Trials

A new law in Illinois will allow low-income patients with cancer from underserved communities to be reimbursed for clinical trials within 30 days.

The Cancer Clinical Trial Participation Act will reimburse patients for out-of-pocket expenses, including costs associated with traveling to sites. Only patients who meet certain criteria on a sliding scale of income will receive financial assistance.

Laws similar to the Illinois law are in effect in California, Pennsylvania and Texas. Similar legislation is being considered in Florida, Massachusetts, New Mexico and Wisconsin.

Financing Round Secures $14 Million for Inato’s Clinical Trials Matching Platform

French company Inato has secured $14 million for its platform that matches clinical trials with sponsors and sites. The new funds will help the organization improve the predictive capabilities of its platform and also help develop a global clinician network.

The funding comes from Obvious Ventures, Cathay Innovation, Serena and Fly Ventures. Prior to this financing round, Inato has already used its platform for 50 clinical trials across 2,000 global research sites.

Owkin and University of Pittsburgh to Launch Pilot on AI

Startup Owkin is collaborating with the University of Pittsburgh to launch a pilot program that marries its artificial intelligence and Pitt’s trial datasets and research findings to come up with better trial designs, more effective drug development strategies and biomarker discovery.

Owkin’s federated network produces research from data in clinical trials at U.S. and European academic medical centers.