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From the pages of Medical Marketing & Media:
European regulators to publish clinical trials data
The head of the European Medicines Agency has said it's a matter of how, not if, the body will mandate publication of clinical trials data – and that has pharmas on both sides of the Atlantic sweating the potential implications for global competitiveness.
EMA executive director Guido Rasi said last week “The European Medicines Agency is committed to proactive publication of clinical trial data, once the marketing authorization process has ended. We are not here to decide if we publish clinical trial data, but how.”
His remarks came at the start of a daylong workshop in London on data transparency around clinical trials. The agency has convened several advisory groups, including some representation from industry, to grapple with thorny issues including patient confidentiality, data formats, rules of engagement, good analysis practice and legal aspects. They will start work in early 2013 and are expected to deliver their recommendations by the end of April, with mandatory publication of trials data taking effect on January 1, 2014.
“The implications are rather significant relative to the continued protection of intellectual property rights,” said Peter Pitts of the Center for Medicine in the Public Interest, “and continued incentives—or disincentives—to invest in innovation.”
The full article can be found here.
Gulf News
Experts stress need for innovation in healthcare
Regulation will ensure adequate supplies of medicinal stocks, says ministry
By Carolina D’Souza, Staff Reporter
November 28, 2012
Dubai: The UAE is intensifying its efforts to increase availability of medical technologies and drugs. This key message was delivered by the UAE Ministry of Health (MoH) during the opening of a two-day conference on Wednesday.
The Ministry believes that the UAE is one of the leading countries in the region to provide innovative medicine.
The conference titled ‘Competitiveness Forum: Health Care and Access to Innovative Medicines in the UAE’ brought together senior officials from the MoH, healthcare authorities, Ministry of Economy and GCC regulatory bodies as well as representatives from the private pharmaceutical sector.
Speaking to Gulf News, professor Peter Pitts, president of the Centre for Medicine in the Public Interest (CMPI), said, “The UAE has smartly figured out that by partnering with the pharmaceutical industry, it can bring innovation to the country and make it a hub of innovation.”
He said that the UAE is recognised for its speed in marketing new medicines and providing patients with the newest medical technologies. Stressing on the need for innovation in medicine, he explained that incremental innovation is more common — than one-time innovation — through which companies enhance a product or a technology.
Extending lives
“Stakeholders, governments and regulators should support innovation in healthcare — it extends peoples’ lives,” said Pitts.
Dr Amin Hussain Al Amiri, assistant undersecretary for Medical Practices and Licensing at the MoH, said that innovative medicines are key drivers for growth in the healthcare sector. “The UAE is one of the leading countries, providing its residents with all kinds of medicine, including life-saving drugs. Through regulations, we will ensure that new medicines are introduced and adequate stocks are maintained for all healthcare needs.”
Dr Ola Al Ahdab, pharmaceutical consultant for registration and drug control at the MoH, told Gulf News that access to timely and advanced medicine is a top priority for the UAE government.
Chair of the conference organising committee Dr. Yaqoub Haddad added, through this conference, the pharmaceutical companies and stakeholders will be able to identify strengths and weaknesses in the healthcare sectors to continue to provide access to innovative medicine.
At Barnes-Jewish Hospital, Dr. Lynch said physicians from all over the Midwest referred their sickest heart patients to his facility for transplants and other major interventions. But those patients can skew his hospital’s readmissions numbers, he said: “The weaker your heart, the more advanced your emphysema, the more likely you are to be readmitted to the hospital.”
Dr. Lynch said Barnes-Jewish set up follow-up appointments for patients who didn’t have their own doctors. But about half of the patients never showed up, he said, even after the hospital made reminder phone calls and arranged for free rides. Sending nurses to see patients at home did not significantly reduce readmission rates either, he said.
“Many of us have been working on this for other reasons than a penalty for many years, and we’ve found it’s very hard to move,” Dr. Lynch said. He said the penalties were unfair to hospitals with the double burden of caring for very sick and very poor patients.
“For us, it’s not a readmissions penalty,” he said. “It’s a mission penalty.”
Read the full New York Times piece here.
The first step towards solving a problem is to identify that a problem exists.
The breakthrough therapy provision in FDASIA is aimed at shortening the development time for drugs intended to treat serious or life-threatening conditions and for which preliminary clinical evidence suggests a substantial improvement over existing therapies. But the devil is in the developmental details.
But the promise of speeding innovative medicines to market could be blunted unless issues related to manufacturing, companion diagnostics and international harmonization are adequately addressed.
That was the message from a multi-stakeholder panel discussion at the November 14th Conference on Clinical Cancer Research, co-sponsored by the Friends of Cancer Research and the Engelberg Center for Health Care Reform at Brookings.
Panelists and FDA representatives agreed that if a drug is ushered through the development process, hold-ups will arise from other areas. As a result, discussions surrounding chemistry, manufacturing and control issues for new drugs and biologics will need to take place much earlier in the clinical development process than currently happens to avoid a situation where a breakthrough therapy is approved but the manufacturing processes are not in place to support commercial marketing.
Discovery is one thing. Development and commercialization are another. Nobody said it was going to be easy.
Breakthrough treatments that require companion diagnostics also could be potentially problematic, as the development, validation and approval of the diagnostic may not be able to keep pace with the drug’s abbreviated clinical development program.
Under FDASIA, sponsors must request that their product be designated as a breakthrough therapy, and FDA must respond within 60 days. For drugs receiving the designation, the agency must work in a variety of ways to expedite the products’ development and review, including providing timely advice to the sponsor to ensure the development program is as efficient as possible and involving senior managers and experienced review staff in a collaborative cross-disciplinary review.
Under the law, FDA must issue draft guidance by January 2014 on the process and criteria for making designations and actions the agency will take to expedite development and review.
What about international harmonization? During the development of the FDASIA legislation, Woodcock cited the need for FDA and EMA to “be on the same page” about abbreviated development programs for breakthrough therapies. And, according to a conference issues brief, absent an equivalent development pathway in the EU, “drugs that receive breakthrough designation in the U.S. may still be required to go through the traditional drug development pathway for EMA approval. These differing requirements may make the breakthrough pathway an unattractive option for drug sponsors.’
Richard Pazdur raised the question as to what happens when subsequent studies of a breakthrough-designated product fail to confirm the encouraging efficacy signal seen early in development. Bob Temple suggested the agency’s guidance on breakthrough designations would account for such a scenario. “The concept clearly includes the possibility that it looked really good at first and then now it doesn’t and so it’s designation could go away,” he said. “I’m sure there will be a provision for that.”
That's one thing we can most certainly be sure of. Caveat emptor.
“PR” does not stand for “Post Regulatory” – nor should it.
Via an untitled letter concerning a pitch letter, the FDA sent out an important holiday reminder that press materials are, indeed, regulated speech.
The target of the October 31st letter was Cornerstone Therapeutics. Their press release for its Curosurf (indicated for rescue treatment of premature infants experiencing Respiratory Distress Syndrome) was, according to the FDA, false and misleading due to omission of risk info and presentation of unsubstantiated superiority claims. A pretty standard objection, and an obvious mistake that should have been avoided by the Cornerstone communications staff.
Communications departments and PR agencies beware – play by the rules or suffer the consequences.
The Problem of Fake and Useless Drugs
Global health authorities are making only fitful progress toward eliminating fake or substandard drugs that cause widespread suffering and death in the developing world. Delegates from 76 member countries of the World Health Organization met this week in Buenos Aires to lay the groundwork for more forceful action.
No one knows precisely how much fraudulent or substandard medicine is sold around the world, but the fragmentary data are alarming. In poor countries, half of the medicines used to treat some deadly diseases have been found to be fakes that had little or no active ingredient; worse yet, some contained toxic substances.
One estimate by experts is that at least 100,000 people die every year from substandard and fake medicines for cancer, heart disease, infectious diseases and other ailments. Fake malaria drugs pose a real risk of hampering the international effort to curb the disease. In wealthy nations, substandard or fraudulent drugs have caused thousands of adverse reactions and some deaths.
The United States, despite having a strong regulatory agency, is hardly immune to this problem. In recent years, fake doses of Avastin sold to physician groups for cancer treatment lacked the active ingredient; a weight-loss medicine contained undeclared ingredients that posed a health risk; and compounding pharmacies have produced contaminated drugs that caused meningitis and deaths.
An analysis by health experts published in the British Medical Journal last week lamented the lack of progress in addressing this scourge. The group called for a global treaty backed by governments, drug companies and nongovernmental groups to ensure a safe drug supply.
Crackdowns by international police and national regulatory agencies have made progress in shutting down dangerous online pharmacies. But, in poor countries, the greater problem is keeping bad products out of the supply chain that serves pharmacies and health care provider groups. In Kenya, even Doctors Without Borders, a smart operation, was duped into buying fake antiretroviral drugs to combat AIDS, which it gave to thousands of patients before the fraud was discovered.
There is an urgent need for a more vigorous international effort. That might include stronger surveillance and research to determine the extent of the problem, clearer definitions for bad drugs (substandard, counterfeit, falsely labeled, falsified or fraudulent drugs, for example) and new international laws to make it a crime to traffic in such medicines.HHS has issues a proposed rule that would increase the number of drugs eligible for reimbursement by insurers under the Affordable Care Act's essential health benefits requirements. In a previous bulletin, HHS said it planned to require that essential health benefits cover only one drug in each therapeutic class included in benchmark plans put forward by each state. The new rule essentially changes the language from 'only one' to 'at least one' drug, requiring that insurers cover at least one drug per therapeutic area or the same number of drugs in each category and class as specified in the state benchmark plan, whichever is greater.
BioCentury reports that the proposed rule also states that a plan "must have procedures in place that allow an enrollee to request clinically appropriate drugs not covered by the health plan." Comments on the proposed rule are due Dec. 26. Previous comments on HHS' bulletin said a one-drug requirement would have provided insufficient access to medications for some conditions. Medicare Part D requires that drug plans provide access to "all or substantially all" medications in six therapeutic classes.
HHS also released a proposed rule that prohibits health insurance companies from discriminating because of a preexisting or chronic condition, as well as a proposed rule on implementing and expanding employment-based wellness programs.
When it comes to jurisdiction over compounding pharmacies, Senator Lamar Alexander (R, TN), wants someone “on the flag pole” responsible for ensuring that non-traditional compounders meet set federal standards.
Alexander proposed allowing FDA to designate state boards of pharmacy as the primary regulator, if they are willing and able. If the state regulators proved unable or made mistakes, FDA would have the ability to take over regulation.
FDA Commissioner Hamburg gave that idea a positive nod, saying it was one model that could be explored, but she still wants federal regulations to assure consistency.
“I think it should be made explicit,” she said. “I think for non-traditional compounding there should be federal standards that apply to certain types of practices that go to issues of quality and safety.”
FDA said those facilities should be required to submit to inspections, report adverse events and register with the agency.
On the House side, Tom Harkin (D, IA), suggested that compounding facilities shipping across state lines should come under FDA scrutiny. Other ideas included using a volume threshold and prescription records.
If the FDA gets more responsibility – which is looking increasingly likely -- they will also need more funding. Otherwise this is all just C-SPAN rhetoric.
Did somebody say “sequestration?”
The election may be over, but the malarkey continues.
The most recent Marlarkey Alert comes to us courtesy of the New York Times editorial page.
In defense of the IPAB, the Gray Lady editorializes:
The board, known as the Independent Payment Advisory Board, has been the subject of false attacks over the past few years by Republicans who claim that it will ration care, disrupt doctor-patient relationships, and tell patients what treatments they can receive. That is an outlandish way to describe a board that is prohibited by law from making any recommendations to ration care, raise premiums, increase cost-sharing, restrict benefits or limit eligibility.
That’s malarkey.
Medicare spending is expected to be $575.7 billion this year, jumping to over $1 trillion by 2022, as the country ages. Over the next 75 years, the program is projected to accumulate a $38 trillion budget shortfall.
Much of this enormous price tag goes towards financing Medicare Parts A (hospital insurance) and B (medical services -- including diagnostic tests and doctor visits). And yet, IPAB has no authority over either. And the board can’t make any substantive structural changes. Neither the fee-for-service structure nor enrollee premiums and fees can be altered.
The board’s only viable option is to further ratchet down reimbursement rates for Medicare providers, especially doctors, who are already losing money on Medicare patients. Indeed, the financial burden of too-low payments under Medicare has driven 17 percent of doctors and 31 percent of primary care physicians across the country out of the Medicare program altogether, according to a study from the American Medical Association.
If rates fall any lower, seniors will have an increasingly difficult time securing doctor appointments. Visits will be cut short to squeeze in patients and care compromised.
Lawmakers must cast aside IPAB’s flawed approach (and the media malarkey surrounding it) and focus instead on innovative initiatives that address the program’s real cost-drivers while protecting seniors’ access to care.
Center for Medicine in the Public Interest is a nonprofit, non-partisan organization promoting innovative solutions that advance medical progress, reduce health disparities, extend life and make health care more affordable, preventive and patient-centered. CMPI also provides the public, policymakers and the media a reliable source of independent scientific analysis on issues ranging from personalized medicine, food and drug safety, health care reform and comparative effectiveness.
