Latest Drugwonks' Blog
Congressional Quarterly reports that the Food and Drug Administration has yet to give final guidance on which of the growing number of mobile health apps it will regulate. Draft guidance indicates “enforcement discretion” will be taken toward consumer-oriented mobile apps, especially ones that are more high risk. The draft guidance also notes that clinical apps could “present a potential risk to patients if they do not work as intended.” Many health industry groups would like to see a wider regulatory framework on IT in place before the FDA issues specific mobile app guidance.
That sounds familiar.
For a more detailed look at this issue – with particular emphasis on the issue of enforcement discretion see, “A Regulatory App-ening.”
South Africa seeking mutual recognition with FDA and EMA.
Maybe a better first step is to establish a reference basket of maybe five to six countries. A good model is Singapore. Everyone’s favorite city-state reviews six countries—USA, Canada, Australia, NZ, Japan, Switzerland and the EMA. If any two of the five have approved, then approval is basically a formality.
Singapore doesn’t have mutual recognition with either the FDA or the EMA, but they unilaterally recognize the benefits of referencing to the “big regulatory dogs”, but did not demand that the FDA reference to their tiny agency.
New medicine control body a step closer
Tamar Kahn: Business Day
THE establishment of a new regulatory body for medicines is a step closer, after the Cabinet said yesterday that it had referred enabling legislation containing the Medicines and Related Substances Amendment Bill to Parliament.
The bill will replace the Medicines Control Council (MCC) with the South African Health Products Regulatory Agency (Sahpra), an entity with much wider scope.
The Cabinet said the bill sought to establish a strong, efficient and effective medicine regulatory authority. The Department of Health envisages Sahpra as being the solution to the extensive delays besetting the MCC, which takes much longer compared with US or European regulators to approve new medicines and clinical trials.
It is also expected to bring scrutiny to bear on aspects of the market that have largely gone unregulated, such as medical devices and complementary medicines. The new regulatory agency will also be responsible for foodstuffs, cosmetics, disinfectants and diagnostics.
To the frustration of researchers and the pharmaceutical industry, Sahpra has been stuck in the works for years. In 2008, Parliament passed amendments to the Medicines and Related Substances Act, which were not implemented. The bill was subsequently redrafted and published for comment in March. Since then it has taken more than a year to refine the bill and get it through the Cabinet.
Department of Health director-general Precious Matsoso said in June that one of the key changes made to the draft legislation was the inclusion of provisions for Sahpra to be a public entity with an independent board chaired by a CEO. It would also have a stronger governance structure than the previous draft, which had a CEO appointed by the Health Minister and gave final authority for the approval of new products to the Minister. The draft bill also included measures to shorten the registration time for medicines and medical devices by allowing mutual recognition agreements between Sahpra and other regulatory authorities such as the US Food and Drug Administration.
From the pages of Health Affairs:
Electronic Communication Improves Access, But Barriers To Its Widespread Adoption Remain
Abstract
Because electronic communication is quick, convenient, and inexpensive for most patients, care that is truly patient centered should promote the use of such communication between patients and providers, even using it as a substitute for office visits when clinically appropriate. Despite the potential benefits of electronic communication, fewer than 7 percent of providers used it in 2008. To learn from the experiences of providers that have widely incorporated electronic communication into patient care, we interviewed leaders of twenty-one medical groups that use it extensively with patients. We also interviewed staff in six of those groups. Electronic communication was widely perceived to be a safe, effective, and efficient means of communication that improves patient satisfaction and saves patients time but that increases the volume of physician work unless office visits are reduced. Practice redesign and new payment methods are likely necessary for electronic communication to be more widely used in patient care.
- 1Tara F. Bishop (tlfernan@med.cornell.edu) is an assistant professor in the Departments of Public Health and Medicine at Weill Cornell Medical College, in New York City.
- 2Matthew J. Press is an assistant professor in the Departments of Public Health and Medicine at Weill Cornell Medical College.
- 3Jayme L. Mendelsohn is a research coordinator in the Department of Public Health at Weill Cornell Medical College.
- 4Lawrence P. Casalino is the Livingston Farrand Associate Professor in the Department of Public Health at Weill Cornell Medical College.
- ↵*Corresponding author
From the pages of the South Florida Sun Sentinel …
Competition from insurers is benefit for consumers
How do you know a federal government program is working? When states start using it as a model for their own initiatives. That's what's happening with Medicare Part D, the prescription drug program for seniors. States are incorporating its unique market-based structure into their own healthcare programs to expand access and manage costs.
Under Medicare Part D, seniors select their prescription coverage from a host of private insurers. States are now applying that principle of free market competition to their Medicaid programs, and the results have been excellent.
Kansas, Louisiana, and Florida have received federal waivers that allow them to experiment with providing Medicaid services through private insurers. The states pay a fixed amount to insure each Medicaid enrollee, and then set minimum benefits that plans must provide.
But it's private insurers that actually supply the plans, and it's the enrollees themselves who decide which plan they would like. As a result, insurers can't provide the bare minimum — Medicaid enrollees can easily select a more appealing plan based on their own needs.
The results have been impressive. When Florida recently ran a pilot program, participating counties outperformed others 64 percent of the time on measured health outcomes. In Louisiana, where those on private insurance have the option of returning to the state's traditional Medicaid program, only one-third of 1 percent have chosen to do so. And because these states expect to save money, other states, including North Carolina, Texas, and Utah, are now considering similar policies.
And these programs don't just promise to save money, they feature various innovations to ensure quality. For example, in Florida, insurers are required to conduct customer-satisfaction surveys. While some states have increased the amount they will pay for coverage of high-risk patients, giving insurance companies an incentive to cover these patients and keep them healthy. This type of risk-based pricing helps patients receive more preventive care, according to a report last year by the Urban Institute.
Nationwide, 36 states and the District of Columbia provide at least some of their Medicaid services through private insurers — and these programs are expected to grow as the new health-care law expands access to Medicaid. States already spend up to one-third of their budgets on Medicaid, so an opportunity to save money while providing better service is an obvious win.
All of this is exciting — but it's not surprising, at least not to those who are familiar with the success of Medicare Part D. By letting seniors choose between private drug plans, Part D has cost the government about 45 percent less than initially projected when Congress enacted the program in 2003.
Out-of-pocket expenses for seniors are also lower than expected. And in a recent survey, 90 percent of Part D beneficiaries said they were satisfied with the program.
Given Medicare Part D's success in its own right and as a model for other healthcare programs, it's bizarre that the president and some in Congress would like to undermine the competition that makes Part D work.
The president's most recent budget, as well as bills introduced in Congress by Sen. Jay Rockefeller and Rep. Henry Waxman, would require drug companies to give "rebates" to the government for the drugs purchased for low-income seniors — known in technical parlance as "dual eligibles" because they qualify for both Medicare and Medicaid.
To put it simply, these politicians would substitute government price controls for the competitive marketplace that has been so effective in keeping costs down. As the Congressional Budget Office has reported, the private plans provided through Part D are already negotiating low prices for drugs. Requiring drug makers to, instead, sell their products for below-market prices will force manufacturers to raise prices on other consumers, including most seniors. Such rebates could increase seniors' premiums by 40 percent, according to a study by former CBO director Douglas Holtz-Eakin.
States may be the laboratories for innovation, but the big lesson from Part D is clear: Competition between private insurers reduces costs and encourages better health care. Some states are learning this lesson and applying it to their Medicaid programs. The federal government should be encouraging this market-based reform, not trying to undermine it.
Peter J. Pitts, a former FDA Associate Commissioner, is President of the Center for Medicine in the Public Interest.
Rationing Health Care in Oregon
BY: HOPE LANDSEM
Liberal states often preview health-care central planning before the same regulations go national, which ought to make an Oregon cost-control commission especially scary. On Thursday a state board could change Oregon's Medicaid program to deny costly care to poor patients who need it most.
Like most such panels, including the Affordable Care Act's Independent Payment Advisory Board, the Oregon Health Evidence Review Commission, or HERC, claims to be merely concerned with what supposedly works and what doesn't. Their real targets are usually advanced, costly treatments. That's why HERC, for example, proposed in May that Medicaid should not cover "treatment with intent to prolong survival" for cancer patients who likely have fewer than two years left to live. HERC presents an example to show their reasoning for such a decision: "In no instance can it be justified to spend $100,000 in public resources to increase an individual's expected survival by three months when hundreds of thousands of Oregonians are without any form of health insurance."
Amazingly enough, the Affordable Care Act quashed that one. The law says coverage decisions cannot discriminate against people because of their diagnoses and life expectancies.
So HERC changed a few words of its proposal. Before, the plan would have limited treatment based on life expectancy. Now, the plan will determine whether an individual continues to receive potentially life-saving treatment based on the severity of the illness, using ambiguous performance statuses, a scale that tries to quantify a cancer patient's well-being. Some difference.
This is a recurring theme in HERC's Medicaid overhaul. The commission also suggested guidelines that would limit to once a week the number of times some diabetics could check their blood sugar, down from the three tests a day the American Diabetes Association recommends now. Outrage from diabetics, not to mention medical experts, forced the commission to postpone that vote.
The public isn't receiving the cancer proposal any better. In a letter to HERC, a Willamette Valley Cancer Institute and Research Center patient navigator, a trained health-care worker responsible for educating cancer patients and guiding them through various treatment options, writes that "patients deserve treatment that is available based on the best evidence, not on a timeline." We'll learn today if HERC is willing to restrict potentially life-saving treatment in favor of the left's one-size-fits-all health approach.
A Capitol Hill Briefing
When: September 10, 2013 from 9:30AM – 2:15PM
Where: Rayburn House Office Building B338, Washington DC
RSVP: Mario Coluccio at mcoluccio@cmpi.org
BREAKFAST & LUNCH TO BE SERVED
AGENDA
9:30- 10AM: Breakfast and Registration
10AM: Welcome
Peter Pitts & Robert Goldberg, Center for Medicine in the Public Interest
10:15: Keynote: FDA Regulation and Responsible Access to Pain Medication
Douglas Throckmorton, Deputy Director, Regulatory Programs, CDER, FDA
11:00: Access to Pain Medications: The Role of Patients and Manufacturers
Moderator: Steve Usdin, BioCentury
Cindy Steinberg, US Pain Foundation
Bob Twillman, American Academy of Pain Management
Stuart Kim, Mallinckrodt Pharmaceuticals
11:45: Break and Lunch
12:00: Issue: Pain Medications: Two Reporters Views
Moderator: Peter Pitts, CMPI
Barry Meier, New York Times
Judy Foreman, Syndicated Columnist
1:30: Closing Keynote: Pain Medications and the Future of Personalized Medicine
Introduction: Bob Goldberg, CMPI
Charles Inturrisi, Weill Cornell Medical Center
2:15: Closing Remarks
Peter Pitts & Robert Goldberg, Center for Medicine in the Public Interest
RSVP: Mario Coluccio at mcoluccio@cmpi.org
BREAKFAST & LUNCH TO BE SERVED
From the pages of Forbes.com.
Medicare Budget Cuts Could Threaten Cancer Patients' Access To Drug TreatmentsBy Peter J. Pitts
Earlier this month, Forbes guest commentator John Wilson celebrated cuts planned for Medicare Part B as a result of sequestration and called for additional “savings” to be wrung out of the program.
Across-the-board federal budget trimming has forced the Centers for Medicare and Medicaid Services (CMS) to significantly reduce the reimbursement rate for healthcare providers that participate in Part B, which covers drugs that have to be administered under professional supervision.
Mr. Wilson thinks these rate cuts are a good start — and he wants more. He claims that “Part B drugs have a history of CMS overpayments” and suggests additional reimbursement reductions won’t have any ill effects.
He’s mistaken.
The model for Part B provider payments is working well to bring down long-term healthcare costs and ensure enrollees have access to needed medication. Additional reimbursement reductions will compromise care in communities throughout the country while doing little to curtail Medicare expenses.
Under Part B, doctors pay for medications on their own and are then reimbursed according to a formula: the average market price for that drug plus an add-on to cover administrative expenses. That additional compensation above the prevailing price is crucial. It helps participating healthcare providers finance other crucial, resource-intensive aspects of treatment, like drug acquisition and storage.
Under this unique, market-like setup, caregivers have an incentive to find the best treatment for the lowest price. Indeed, the Congressional Budget Office projected that this reimbursement system would generate $16 billion in savings over its first decade of operation. And yearly cost growth for the program has been below overall medical inflation.
One major study found that Part B’s reimbursement formula reduced drug spending by over seven percent during the program’s first year of operations. And it limited Part B’s average annual expense growth rate to just 2.4 percent, compared to nearly 11 percent for all of Medicare.
Community health clinics are major participants in Part B. These locally oriented operations tend to be significantly more cost-efficient than larger hospitals. By properly compensating clinics and encouraging them to treat Medicare enrollees, Part B saves the government money over the long-run. For instance, research from the consulting group Milliman has found that Medicare saves an average of $6,500 per year when a patient receives chemotherapy treatment at a clinic rather than a hospital.
All in all, Part B’s reimbursement formula has been working terrifically well. But as a result of sequestration, CMS will be cutting that administrative add-on by about two percentage points — from six to four percent. And some in Washington are looking to ratchet back this reimbursement even further, to closer to three percent.
Further cutting this rate would seriously threatens the financial viability of many of the community health clinics currently participating in Part B. These operations already run on exceedingly thin profit margins. They depend on proper compensation from Part B and other public programs to stay afloat. Reducing reimbursements would force many clinics to close and physicians to turn away enrollees.
New cuts would make a bad situation even worse, particularly in the realm of cancer treatment. According to the Community Oncology Alliance, over the last six years, 288 cancer clinics have closed. Another 469 have entered into a contractual relationship with a hospital or been acquired by a hospital. And 407 report they’re struggling financially.
The American Society of Clinical Oncology predicts that Part B cuts could force up to three-quarters of the remaining cancer clinics in the country to start redirecting Medicare patients to other caregivers.
Mr. Wilson also argues for scaling back the intellectual property protections currently afforded an advanced class of pharmaceutical drugs called biologics. These are highly complex treatments derived from living organisms. In addition to standard patent controls, biologics are also provided 12 years worth of data protection preventing generic competitors from accessing the original innovators research information.
The provision establishing these 12 years of data protection was included in the President’s 2010 health care reform law — and it was one of the few provisions that enjoyed broad bipartisan support from both chambers of Congress.
After noting that 12 years is “far longer than for most other drugs,” Mr. Wilson joins the chorus of misinformed critics calling for biologic data exclusivity to be scaled back to seven years. He thinks such a move would save the public health system billions in drug expenses by expanding the pool of low-cost generic alternatives.
But that 12 year set point isn’t arbitrary. Virtually all the research on this subject shows that that’s about as long as it takes for the average biologic to break even in sales. The average new drug costs on average $1.2 billion to research, develop and bring to market. And just two out of every ten new drugs ever turns a profit.
Cutting down the period of data protection to just seven years would flood the biologic market with generic competition well before most innovators have had time to get out of the red. Drug developers would be much less likely to invest in new products in the future and patients would be deprived of new breakthrough treatments.
Mr. Wilson and I are in agreement that public officials need to find effective ways of controlling Medicare costs without compromising enrollee care. But further cutting Part B reimbursements and reducing the protection of intellectual property for innovative drug companies doesn’t fit the bill — it will undermine community caregivers and choke off patient access to needed medicines.
Peter J. Pitts, a former FDA Associate Commissioner for External Affairs, is President of the Center for Medicine in the Public Interest.
Imagine if our immune systems could vanquish cancer in much the same way they take on the common cold. New research could turn such science fiction into fact.
Currently in the works are several new drugs that can order certain white blood cells - the immune system's warriors - to attack cancer cells. If they prove successful, a world free from cancer could be one step closer. Researchers are increasingly exploring how we can personalize the fight against cancer - harnessing the unique characteristics of our own bodies to beat the disease. Such personalized approaches offer our best shot at eradicating cancer - and should be at the heart of our battle plan against it. Medical science has already made progress.
Since 1990, new medicines have doubled the number of cancer survivors - from six million to 13 million. They have given patients collectively about 43 million years of additional life. These aren't years of pain and desperation. Cancer survivors add about $4.7 trillion in value to the economy just by living and working longer. Today, every dollar spent on new cancer medicines reduces spending on hospitals and doctors by $7. Yet the actual amount we spend on such treatments is small - about 1 percent of total health-care spending. Indeed, spending on innovative cancer research and therapies has already delivered a hefty return on investment. We should double down on that approach.
Step one is to put patients in charge of cancer research. How? Patients can use online communities to test treatments, design studies, and determine better ways to tackle their illnesses. They're already keeping tabs on their health with fitness monitors and tablets. Research should be shaped by these real-time, real-world experiences in combination with information about the particular genetic mechanisms that make their tumors tick. As genomics professor Eric Topol argues: "It is time for a jailbreak; it is time for the rise of the consumers to drive the future of medicine. It is their DNA, their medical data, their cellphones, and their own health at stake."
That jailbreak should include replacing one-size-fits-all research with personalized cancer studies. The Human Genome Project empowers researchers to do so. Personal genomes can now be sequenced in a few hours for under $500. Such sequencing can yield medicines that are truly personalized. Several cancer organizations, including the International Myeloma Foundation, StandUp2Cancer, and the Sarcoma Foundation of America, require researchers to look for genetic cues that could lead to cures. This should be the rule, not the exception.
These personalized approaches could also allow regulators to get new cancer medicines to market faster. Developing a new cancer medicine takes 8.8 years, on average - much longer than for other drugs. Most of this time and effort is spent testing medicines in people who researchers know won't benefit. But by focusing solely on patients and their specific cancer-causing genetic mutations, researchers could identify what therapies work early on. That could mean approving cancer therapies as fast as HIV medicines - in two to three years.
Government officials can also get personalized treatments into the hands of patients more quickly by requiring health plans to pay for them. Advances in cancer treatment are saving lives and cutting health-care costs. But many health-insurance plans haven't caught up with the times. Many cancer patients are forced to choose between a treatment that could save their lives - or one that's paid for. Insurers should instead pay for the right treatment for the right patient.
Under the health-care status quo, cancer treatment is divided up according to who gets paid. Innovations that save money are pitted against services that lose money. New "Charter Cancer Communities" can solve that problem by focusing specifically on the value of care. Like public charter schools, these communities would have greater flexibility to use and pay for the combination of treatments that deliver real value. And they'd be accountable to the member organizations and to the patients they serve. Thanks to recent advances in medical science, we're closer to a world free from cancer. If we ratchet up our investments in personalized medicine, that world can become a reality.
On Thursday I joined a select group of FDA and healthcare policy experts at the joint FDA/Engelberg Center for Health Care Reform (Brookings Institution) to discuss, debate, and digest the many issues surrounding the thorny opportunity known as Special Medical Use (SMU).
Expertly chaired by Mark McClellan (my former boss at the FDA and the hardest working man in healthcare policy), the day was filled with honesty, ideas, identification of roadblocks – and frustration.
But all to the good.
The meeting was also filled with many senior thinkers and heavy hitters from the agency (Rachel Sherman, John Jenkins/OND, Janet Woodcock, among others) as well as senior Hill staffers, officials from the biopharmaceutical industry, BIO, PhRMA, payers, patient organizations, and academia. The list of attendees can be found here.
First of all, permit me to recommend the short briefing paper that McClellan’s team developed in advance of the meeting, “Special Medical Use: Limited Use for Drugs Developed in an Expedited Manner to Meet an Unmet Medical Need.” Worthwhile reading both as a primer and a guide to many of the regulatory quandaries surrounding SMU.
The meeting began with McClellan commenting that we needed to “channel PCAST” and making the key distinction (regularly confused) that the difference between special medical use (from a labeling perspective) and REMS – is that REMS is for known safety risks.
And then came Janet.
Her remarks focused understanding SMU along the “safety and certainty” continuum. SMU medicines are for sub-segments of a patient population (with a serious and life-threatening disease) that are not effectively served by current therapies. “There’s a difference between headaches and HIV.” And that wasn’t a throwaway line. In fact, there was zero mention or acknowledgement that SMU should be a pathway exclusively for anti-infectives. Janet strongly affirmed that the FDA has no interest in interfering in the practice of medicine. To the contrary, she believes the best way to ensure that SMU products are used appropriately is through clear and distinct labeling – special logo, etc.
This makes perfect sense and is a logical extension of the agency’s Safe Use of Drugs initiative.
Janet also raised the issues of SMU products and industry promotional practices. Her feeling is that (as with other Breakthrough Designation programs) that pre-view of all marketing materials would be an SMU prerequisite. She also raised the interesting question of seeking to limit on-label communications in order to drive “appropriate messaging from launch because appropriate messaging leads to appropriate use.”
Tom Abrams, call your office.
Janet also noted the fear of many in industry that the FDA would “deem” an SMU designation. She made it clear that would not be the case.
After Dr. Woodcock’s opening keynote, the first panel of the event focused on “The Special Medical Use Pathway Proposal.” The panelists were Jim Greenwood (BIO), John Castellani (PhRMA), Jack Lasersohn (The Vertical Group), Margaret Anderson (FasterCures), and Jeff Allen (Friends of Cancer Research).
And, while there were no fireworks – there was a degree of discomfort – specifically on SMU and legislation, regulatory authority, and therapeutic areas of use.
Here’s how Steve Usdin at BioCentury saw it:
BIO, PhRMA split on special use pathway
The leaders of the Biotechnology Industry Organization and the Pharmaceutical Research and Manufacturers of America staked out separate positions on the creation of a Special Medical Use pathway proposed by the President's Council of Advisors on Science and Technology and senior FDA officials …
BIO President and CEO James Greenwood said the group's board "strongly approves" of SMU, endorses congressional action to create the pathway and feels it should be "broadly applied" to drugs for a wide range of diseases. John Castellani, PhRMA's president and CEO, declined to endorse creation of an SMU pathway and said FDA has sufficient authority to approve drugs for special populations, though he stressed the urgency of facilitating development of new anti-infective drugs. Members of Congress have indicated that enactment of legislation creating an SMU pathway is contingent on unanimous support from industry, so PhRMA's skepticism about the need for an SMU pathway could scuttle the concept.
While the general sense of Usdin’s comments is correct, it may not be quite so black and white.
Both Greenwood and Castellani support the concept of SMU. And that’s an important point of departure. Greenwood, while supporting the concept of legislation, believes that the FDA already has the regulatory authority to create a Special Medical Use pathway and the ensuing labeling for such products. He said that, rather than pushing for actual legislation, that a “sense of Congress” might be a better idea.
Dr. Woodcock, call your lawyers.
Castellani agreed that the agency already as the authority to move forward. So there’s important convergence there. But he was less clear on the issue of an SMU pathway that went beyond anti-infectives. I specifically asked him if he felt the SMU pathway should be limited to anti-infectives. His answer was to reiterate PhRMA’s concerns about “direct or indirect” impact on the practice of medicine and possible agency hindrance of appropriate promotional communication.
Both Margaret Anderson and Jeff Allen feel that the SMU pathway should be open to any appropriate therapeutic area. Jeff Allen made a key point, that the SMU pathway is possible because of advances in science. It sounds like a simple point – and it is – but it’s important to recognize that SMU is the next logical step towards personalized medicine.
Margaret Anderson also got real by pointing out that, minus additional budget, the FDA will only be able to go so far. “How much innovation,” she asked, “can the agency be expected to handle?”
Indeed.
The general consensus was that SMU should be piloted with anti-infectives – but proceed rapidly to where the need is the greatest. Bravo – but highly subjective.
John Castellani, call your office.
Panel Two operated under the moniker, “Implementation and Impact of Special Medical use Products in Clinical Practice” – but this was really the payer panel.
Sam Nussbaum (WellPoint) brought the urgent issue of patient outcomes into the conversation. His point was (per reimbursement) that if the SMU process works as designed (limited use with an enhanced risk/benefit profile in a defined sub-population) that they would be swiftly tiered for reimbursement.
Pursing the topic of the role payers have in outcomes, Ed Septimus (HCA Healthcare) stated that, “Stewardship is resource intensive.” True – but lack of stewardship is even more costly in terms of scarce dollars and clinical sense – and patient lives. Septimus also introduced the concept of mandatory patient registries.
I asked the panel if, as part of the regulatory process, the FDA should mandate patient registries for SMU medicines. There was an uncomfortable silence.
Gerald del Pan, call your office.
Panel III: “Postmarket Considerations to Promote Safe Use and Continued Evidence Development of Special Medical Use Products” opened with Preeti Pinto (former AZ compliance chief and now consultant extraordinaire to the regulatory stars) stating that, “Marketers will promote the product to the full extent of the label.” No surprise there, but a real issue when it comes to promoting safe use of SMU medicines. Can marketers self-regulate? Is that even feasible? Short of an OPDP SWAT team for SMU medicines (with the authority to apply harsh penalties) how can marketers (per Preeti’s prescience) be made to “do the right thing?” What are the appropriate tools for post-marketing surveillance and how can they be validated?
Rob Califf (Duke Translational Medical Institute and Duke University Medical Center) pointed out that the label has become nothing more than a tool for legal protection – dismissing any future utility. But, in a post Wyeth v. Levine world, with preemption off the table, perhaps now is precisely the right time to revisit the public health utility of the PI.
Perhaps, as part of the SMU pathway, the FDA should adopt the EPARS (European Public Assessment Reports) system. The European Medicines Agency (EMA) publishes an EPAR for every medicine granted a central marketing authorization by the European Commission. EPARs are full scientific assessment reports of medicines authorized at a European Union level.
Rachel Sherman, call your office.
An “almost” consensus of the meeting was the SMU pathway should proceed apace with anti-infectives as the pilot program and under existing regulatory authority. Almost. Maybe.
Yesterday I participated in the Brookings Institution/FDA meeting on Special Medical Use. I am gathering my notes and will have a more complete report ready for Monday.
In the meantime, here is an interesting take on the issue from the pages of Specialty Pharmacy Times.
FDA’s New Expedited Approval Program Provides Major Communication Breakthrough for Industry
Drugs that show a clear or substantial benefit to patients in the earliest stages of clinical trials can now reach the market more quickly, thanks to the FDA’s new Breakthrough Therapy designation. The expedited pathway, introduced by the Food and Drug Administration Safety and Innovation Act of 2012, has the potential to shave off nearly 75% (or by some accounts, nearly 2 years) of the time it typically takes the FDA to review a new drug application.
One of the major innovations of the expedited drug development process appears to be that drug companies now have direct access to something that previously eluded them: FDA officials. According to Reuters, efforts to bring promising therapies to market have been well-coordinated through the use of these pathways, and communications “that might typically take weeks and months” have been occurring more quickly, with fewer gaps between interactions.
The FDA began granting breakthrough designations in January 2013. To date, the agency has received 77 requests. Of these, the regulatory body has granted 25 designations and denied 24. Kalydeco (ivacaftor), Vertex’s cystic fibrosis drug, was approved under the breakthrough designation pathway earlier this year. Anticipated blockbusters such as Genentech’s obinutuzumab and Janssen/Pharmacyclics’ ibrutinib have snagged breakthrough designations as well. Obinutuzumab was granted the designation for the treatment of chronic lymphocytic leukemia (CLL), and ibrutinib was granted 2 breakthrough designations: one to treat patients with CLL and the other for patients with mantle cell lymphoma.
Although getting treatments to patients more quickly is definitely a good thing, a recent blog from Context Matters points out that, based on its calculations, the FDA’s previously established programs to expedite therapies (including Fast Track, Accelerated Approval, and Priority Review) have not produced lasting improvements in approval time. “Based on our preliminary analysis, in 2008 the average cycle time for ‘Priority’ was 10.1 months, versus the ‘Standard’ which was 21.2 months. In 2011, the average cycle time for ‘Priority’ was 19.5 months, versus ‘Standard’ which was 17.5 months,” the blog authors wrote. “What’s interesting here is that over time the fast lane has apparently become just another standard lane; in 2011 it was actually even slower.”
The group at Context Matters compared the process in the United States to that of the one used by the European Medicines Agency (EMA) and concluded that despite not having “fast” or “standard” designations, the EMA approval process took an average of just 3.5 months during 2012.
The FDA needs to have the proper infrastructure and support to successfully reduce approval times, says Peter Pitts, a former head of communications for the FDA who is a board member of Context Matters and co-founder of the Center for Medicine in the Public Interest. “Clearly, when you are looking at products that have less data behind them, you need more senior people who can devote greater resources to studying them, and that also takes time away from other programs that the agent needs to review,” Pitts told Specialty Pharmacy Times. “It’s one thing to give the FDA more authority, it’s another thing to give the FDA greater responsibility, but if you don’t give them the resources to get it done, to a large degree it is just rhetoric.”
Despite the improvements in communication and collaboration between drug developers and the FDA, manufacturers still must overcome many hurdles on the way to getting therapies approved by the agency. Sponsors must have more resources available in less time in order to push their therapies through the abbreviated regulatory process, and because many of the drugs being submitted for review under the new designation are targeted therapies, they frequently must be developed in concert with a companion diagnostic test. Such diagnostics would have to be approved separately by the FDA’s Center for Devices and Radiological Health, and guidance governing their development has been shaky so far.
Once therapies gain approval through the breakthrough therapy program, they still may face challenges in getting to patients as health plans may be reluctant to provide reimbursement for therapies with a limited amount of evidence regarding patient outcomes. Other issues that may thwart successful market penetration of drugs traveling through this pathway include timing of facility inspections and obtaining drug approval in other countries (where the breakthrough designation is not recognized or accepted). A lack of FDA resources may also affect the success of the breakthrough pathway in getting therapies to patients with few or no treatment options.
But, speed to market isn't the only metric that matters, Pitts points out. "Breakthrough designation doesn’t always mean the product gets approved...sometimes it means that because you fail faster, you don’t waste money on programs that don’t pan out."
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.
